Podcasts about Spark Therapeutics

This CME program provides information on best practices to manage children with lysosomal disorders who have been identified by newborn screening. WIth the wide range of symptoms and severities that present for these rare conditions, it is not always certain when the best time to start treatment is in these patients.Continuing Education InformationThis continuing education activity is provided by AffinityCE and the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC). This activity provides continuing education credit for physicians. A statement of participation is available to other attendees.To obtain credit, visit https://checkrare.com/learning/p-transforming-clinical-outcomes-with-early-treatment-of-lysosomal-disorders/ Faculty and DisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Ozlem Goker-Alpan MDFounder and CMO, Lysosomal & Rare Disorders Research & Treatment CentersDr. Goker-Alpan is on the Advisory Board/Consultant for Chiesi, Takeda, Sanofi, Prevail/Lilly, Sparks Therapeutics, Uniqure, Exegenesis, Astellas, Freeline, Team Sanfilippo. She receives grants/research support from Chiesi, Sanofi, Takeda, Prevail/Lilly, Spark Therapeutics, Amicus, Freeline, Sangamo, Cyclo, Odorsia, DMT, Homology, Protaliz. She is on the speaker bureau for Sanofi, Takeda, Amicus, ChiesiDavid F. Kronn MDAssociate Professor of Pathology and Pediatrics                                                New York Medical CollegeDr. Kronn is on the Advisory Board for Sanofi. He is also on the speaker bureau for Sanofi. He receives research funding from Sanofi.Uma Ramaswami FRCPCH, MDRoyal Free London Hospitals & Genetics and Genomic Medicine, University College LondonDr. Ramaswami is on the Advisory Board for Amicus, Chiesi, Sanofi and Takeda. She receives research grants from Chiesi and Intabio.Liz Jalazo MDAssistant Professor of Pediatrics and GeneticsUniversity of North Carolina at Chapel HillDr. Jalazo is on the Advisory Board for Sanofi and Ionis. Lindsay Torrice MSN, CPNP-PC MDAssistant Professor of PediatricsUniversity of North Carolina at Chapel HillMs. Torrice has no financial relationships to disclose.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible companies. All relevant financial relationships for faculty were mitigated by the peer review of content by non-conflicted reviewers before the commencement of the activity.Learning ObjectivesAt the end of this activity, participants should be able to:•     Cite the importance of early diagnosis and treatment of lysosomal storage disorders•     List the guidelines for the early treatment of LDs and enhanced integration of newborn screening programs•     Identify key research gaps and priorities and strengthen collaboration among researchers and healthcare professionals•     List the educational resources and support programs for familiesPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other healthcare professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity.Commercial SupportThis activity was supported by educational grants from Takeda, Sanofi, and Chiesi.Participation CostsThere is no cost to participate in this activity. CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.com.Send customer support requests to cds_support+ldrtc@affinityced.com.

Send us a textWe continue our conversation with Chynna Broxton, an analytical developmental scientist at Spark Therapeutics on her journey to this point in her career. In the early days of her education Chynna vividly remembers the difficulty she had learning how to read; this setback didn't deter her from doing well generally in her classes. In high school, however, something clicked in her brain in a way that felt very different compared to the struggles she had previously with reading. It was a big wow moment for Chynna as she effortlessly absorbed mathematical theorems and chemical equations in her high school STEM classes. She had an even bigger wow moment in her undergraduate biochemistry course at Saint Elizabeth University–understanding how molecules and compounds are applied to treat diseases. During the summer of her junior year, Chynna was encouraged by a mentor to attend the National Science Foundation (NSF) Research Experiences for Undergraduates program, which allowed her to spend time at Syracuse University doing research. Chynna remains thankful to the mentor who encouraged her to apply for this foundational program and opened her eyes to the possibility of becoming a scientist. As she looks back, she had many thoughtful mentors from her early days in middle and high school to managers in the workplace who had faith in her and nurtured her abilities. These countless mentors have helped Chynna to tackle a continual battle with imposter syndrome. She could not believe her acceptance to Johns Hopkins Bloomberg School of Public Health, initially, and even more recently had doubts about leading a three-hour workshop. Through the doubts, however, Chynna has really learned to persevere. The best advice she gives for handling imposter syndrome is, “Even if you don't believe in yourself, you have to listen to the people who invited you there. They invited you there for a reason.” Reflecting back on her career journey, Chynna also realizes that her vision of success has changed over time as she has grown and evolved from the time she was a postdoc and defined by her research project, to now, as an analytical developmental scientist. Tune into this episode to hear about Chynna's career journey and:What influences from her childhood encouraged her to pursue science, despite difficulties How the realization that college professors with Ph.D.'s are also called Dr. opened a door for exploring career options The impact of mentors in guiding her career journeyWhat does it mean to “make more space for [yourself], make more space for rest, and make more space for reflection” Also check out her YouTube channel: @ChynnaNicoleplansInstagram: @planning_phd and LinkedIn: https://www.linkedin.com/in/chynna-broxton/If you enjoyed this episode, check out one more:Ep 61: The Love Language of Organic Chemistry - The Journey Support the Show: WeLoveSciencePodcast.com Reach out to Fatu:www.linkedin.com/in/fatubmInstagram: @thee_fatu_band LoveSciencePodcast@gmail.com Reach out to Shekerah:www.linkedin.com/in/shekerah-primus and LoveSciencePodcast@gmail.com Music from Pixabay: Future Artificial Intelligence Technology 130 by TimMoorMusic from https://freemusicarchive.org/music/Scott_Holmes: Hotshot by ScottHolmesMusic

In this episode of Moving Forward, Giving Back, four of our incredible donors and life sciences leaders sit down for a roundtable discussion about their involvement in our program Life Science Shares. This campaign is a way for companies or individual leaders to contribute to Life Science Cares if and when they reach financial milestones or other positive events by pledging a percentage of a future transaction. Joining the discussion are host Jeff Marrazzo, the co-founder and former CEO of Spark Therapeutics, and guests Sarah Boyce, CEO and President of Avidity Bio, Robert Blum, President and CEO of Cytokinetics and Ron Cooper, Chairman of C4 Therapeutics and former CEO of Albireo Pharma. During this dynamic conversation Jeff, Sarah, Robert and Ron explain their reasons for getting involved with the Life Science Shares initiative and the benefits the decision has had for them personally and for their companies. Learn more about LSC and Life Science Shares at lifesciencecares.org.

Send us a textIn this episode, we sit down with Dr. Chynna Broxton, an Analytical Development Scientist at Spark Therapeutics, to uncover the realities of working in the biotech industry. Chynna dispels common myths about transitioning to industry, addressing concerns about losing scientific engagement and sharing how she's grown significantly at Spark. She gives a broad overview of her role, which involves developing methods to characterize gene therapy products for inherited genetic disorders. Comparing Academia to Industry, Chynna breaks down the pros and cons of working in both, touching on such themes as flexibility, goals, compensation, and the tangible impact of your work. Drawing from her experiences working at a CRO (Contract Research Organization) and her current role at Spark, she highlights the main difference between these industry settings. Not all industry jobs are the same, she explains. There are a wide variety of roles, and while some mirror the academic experience others are unique to the industry landscape.For anyone considering a move from academia to industry, Chynna offers encouragement, and advice on finding your best fit. Join us to learn from Chynna what it takes to thrive in the biotech industry.Tune in to Learn:What skills learned during her academic career directly transfer to this roleWhat surprised her about working in industryThe pros and cons of working in industry compared to academia Her key tip for finding a role in industry that's a good fit for youHow her graduate and postdoctoral work prepared her for her current positionWhat she loves about doing science other than the discoveries.Reach out to Chynna:LinkedIn: https://www.linkedin.com/in/chynna-broxton/Email: cbroxton@steu.eduIf you enjoyed this episode, you'll also enjoy:Ep 60: Arianne Hunter's experience - senior scientist at Abbvie  Ep 50: Clinical trials to cure genetic diseases with CRISPR technologyEp 15: Joseph Iacona's experience - scientist at Janssen Support the Show: WeLoveSciencePodcast.com Reach out to Fatu:www.linkedin.com/in/fatubmTwitter: @thee_fatu_band LoveSciencePodcast@gmail.com Reach out to Shekerah:www.linkedin.com/in/shekerah-primus and LoveSciencePodcast@gmail.com Music from Pixabay: Future Artificial Intelligence Technology 130 by TimMoorMusic from https://freemusicarchive.org/music/Scott_Holmes: Hotshot by ScottHolmesMusic

VIsit Nascentmc.com for medical writing assistance. Visit learnamastyle.com for freebies on medical writing and editing and ChatGPT. - Fasenra Pediatric Asthma Expansion: No details provided for this update. - Beqvez for Hemophilia B: FDA approved gene therapy, Beqvez, for adults with moderate to severe hemophilia B, which enables the production of clotting protein factor IX. This one-time treatment by Pfizer, derived from Spark Therapeutics, aims to replace frequent infusion therapies, demonstrating superior efficacy in a late-stage trial. - Anktiva for Bladder Cancer: Anktiva received FDA approval for treating BCG-unresponsive non-muscle invasive bladder cancer, enhancing NK and T cell proliferation. Based on a trial with 77 patients, it showed a 62% complete response rate, surpassing international clinical benchmarks, leading to its designation as a breakthrough therapy. - Pivya for UTI: FDA approved pivmecillinam (Pivya) for treating uncomplicated urinary tract infections caused by specific bacterial strains. This marks the first new antibiotic for such infections in the U.S. in over 20 years, backed by effective outcomes in three clinical trials. - Tovorafenib for Pediatric Low-Grade Glioma: Tovorafenib was approved for pediatric low-grade glioma patients with specific BRAF alterations, showing a 67% response rate in the FIREFLY-1 trial. It has been designated for accelerated approval due to its potential in treating these brain tumors. - Entyvio Maintenance for Crohn's Disease: Vedolizumab (Entyvio) received approval for subcutaneous administration as Crohn's disease maintenance therapy after initial intravenous induction. Supported by the VISIBLE 2 Study, it proved effective in maintaining clinical remission at 52 weeks. - Alecensa for NSCLC: Alecensa was approved as an adjuvant treatment post-tumor resection for ALK-positive non-small cell lung cancer. In the ALINA trial, it significantly extended disease-free survival compared to chemotherapy, particularly in early-stage patients. - SPG601 for Fragile X Syndrome: The FDA cleared SPG601 for a phase 2a trial in Fragile X Syndrome, addressing synaptic function through BK channel activation. This marks an advance for treating the core symptoms of the most common inherited intellectual disability. - Lumisight for Visualizing Breast Cancer: The FDA approved Lumisight and the Lumicell Direct Visualization System for use during lumpectomy surgeries to detect residual cancer tissues. This system, shown in the INSITE trial, improves surgical outcomes by reducing the need for second operations. VIsit Nascentmc.com for medical writing assistance. Visit learnamastyle.com for freebies on medical writing and editing and ChatGPT.

In this month's episode of Trends from the Trenches, host Stan Gloss speaks with Joseph La Barge. La Barge trained as an attorney but has spent most of his career building biotech businesses and securing funding. He shepherded Spark Therapeutics through its acquisition by Roche and has since joined Apertura Gene Therapy as CEO. He and Stan talk about the entrepreneurship journey, gene expression and capsid engineering, and Apertura's future goals and projects.  Links from this episode:  Bio-IT World BioTeam Bio-IT World Europe Apertura Gene Therapy Trends from the Trenches boiler: Bio-IT World's Trends from the Trenches podcast delivers your insider's look at the science, technology, and executive trends driving the life sciences through conversations with industry leaders. BioTeam co-founder Stan Gloss brings years of industry experience in science, data, and technology to conversations exploring what is driving data and discovery, and what's coming next.

As you will hear in this episode, Kristin Smedley grew up and lived her first thirty years or so as a list-maker and planner. She literally planned everything and she was successful at it. Well, she was until literally one day everything changed. In January 2000 she gave birth to her first son, Michael. When he was eight months old she asked a nurse friend/Michael's babysitter about the fact that Michael's eyes seemed not to be focused when he was lying on his back. After examinations, she got the news that Michael was blind. All the plans she had for herself and him “crashed to the floor”.   We get to hear Kristin's story with not one blind son, but a second one, Mitch who was born two years later. Kristin will tell you that she refused to adopt the attitude that these two blind kids could not grow up and do anything. She will tell us how both sons played baseball in grammar school. You will hear how Kristin's incredible positive attitude about blindness helped her family discover and learn that blindness does not hold people back.     About the Guest:   Kristin Smedley is Co-Founder and CEO of the only patient organization in the world for people living with the blindness her two sons are affected by, CRB1 LCA/RP. The Curing Retinal Blindness Foundation has raised over 4 million dollars and achieved a National Rare Eye Disease Awareness Day.  That legislation, H.R. #625, was the first in US history to be submitted in Braille and it advocates for better resources for blind and visually impaired Americans. Kristin partnered with Spark Therapeutics to help achieve the first ever FDA approved gene therapy to treat an inherited retinal disease in the United States. She has done a TEDx Talk in New York City to change perceptions of blindness and she partnered with Comcast media to spread awareness of the inclusive X1 product. Kristin is author of the bestselling book Thriving Blind: Stories of Real People Succeeding Without Sight and a new children's book, What I Can Be Is Up To Me. Kristin co-founded ThrivingBlindAcademy.org to solve the employment, literacy, and financial crisis in the blind community.  She is Co-Creator of the short film, The Great Equalizer, that addresses the unemployment crisis of the blind.     Ways to connect with Kristin:   Linked In https://www.linkedin.com/in/kristinsmedley/ Twitter https://twitter.com/KristinSmedley Facebook https://www.facebook.com/thrivingblind Instagram https://www.instagram.com/kristinsmedley/   About the Host: Michael Hingson is a New York Times best-selling author, international lecturer, and Chief Vision Officer for accessiBe. Michael, blind since birth, survived the 9/11 attacks with the help of his guide dog Roselle. This story is the subject of his best-selling book, Thunder Dog.   Michael gives over 100 presentations around the world each year speaking to influential groups such as Exxon Mobile, AT&T, Federal Express, Scripps College, Rutgers University, Children's Hospital, and the American Red Cross just to name a few. He is Ambassador for the National Braille Literacy Campaign for the National Federation of the Blind and also serves as Ambassador for the American Humane Association's 2012 Hero Dog Awards.   https://michaelhingson.com https://www.facebook.com/michael.hingson.author.speaker/ https://twitter.com/mhingson https://www.youtube.com/user/mhingson https://www.linkedin.com/in/michaelhingson/   accessiBe Links https://accessibe.com/ https://www.youtube.com/c/accessiBe https://www.linkedin.com/company/accessibe/mycompany/ https://www.facebook.com/accessibe/       Thanks for listening! Thanks so much for listening to our podcast! If you enjoyed this episode and think that others could benefit from listening, please share it using the social media buttons on this page. Do you have some feedback or questions about this episode? Leave a comment in the section below!   Subscribe to the podcast If you would like to get automatic updates of new podcast episodes, you can subscribe to the podcast on Apple Podcasts or Stitcher. You can also subscribe in your favorite podcast app.   Leave us an Apple Podcasts review Ratings and reviews from our listeners are extremely valuable to us and greatly appreciated. They help our podcast rank higher on Apple Podcasts, which exposes our show to more awesome listeners like you. If you have a minute, please leave an honest review on Apple Podcasts.     Transcription Notes   Michael Hingson ** 00:00 Access Cast and accessiBe Initiative presents Unstoppable Mindset. The podcast where inclusion, diversity and the unexpected meet. Hi, I'm Michael Hingson, Chief Vision Officer for accessiBe and the author of the number one New York Times bestselling book, Thunder dog, the story of a blind man, his guide dog and the triumph of trust. Thanks for joining me on my podcast as we explore our own blinding fears of inclusion unacceptance and our resistance to change. We will discover the idea that no matter the situation, or the people we encounter, our own fears, and prejudices often are our strongest barriers to moving forward. The unstoppable mindset podcast is sponsored by accessiBe, that's a c c e s s i  capital B e. Visit www.accessibe.com to learn how you can make your website accessible for persons with disabilities. And to help make the internet fully inclusive by the year 2025. Glad you dropped by we're happy to meet you and to have you here with us.   Michael Hingson ** 01:21 Well, hi, welcome once again to unstoppable mindset. I'm really looking forward to our guest today Kristin Smedley because she has two sons who are blind, I'm not prejudiced or anything like that, of course, but nevertheless, yeah. Nevertheless, she's got some interesting stories to tell. And she has been involved in doing a variety of things, including influencing Washington dealing with forming organizations, and we're gonna get into all that. So I will not talk anymore. But Kristen, let's just start with you. Welcome to unstoppable mindset. Oh,   Kristin Smedley ** 01:55 thanks so much for having me. I'm I'm a big fan of yours. And I'm happy to be here and chat. Well,   Michael Hingson ** 02:03 thanks for for coming on. Well, tell us a little bit about you first, Gordon growing up the early Christian Christian as it were.   Kristin Smedley ** 02:14 The early Christian all those years ago, law I O in   Michael Hingson ** 02:18 a galaxy far far away.   Kristin Smedley ** 02:21 I am a born and raised Philly girl. I have my fillies hat on for those that are watching this on video. And I was one of those kids, Michael that I went for a lot of stuff I had success. In almost every area of my life. I was raised by parents in I'm learning as an adult that I was raised in unconditional love. And I believe I've said it a lot that I believe that's what sets us up with a foundation to thrive. So I had a good support system to get out there and try stuff and go after dreams and, and I was sports school. I mean, you name it. I had a great time with it. But I will above I will admit that above all things I was a I was a planner, you know, and a list maker and a check it off the list, kind of person. So I really liked making plans, achieving them celebrating and going on to the next thing. I've I've played soccer my whole life. I still play actually, I'm going to be 52 And just last year, I perfected my left foot kick. So I figure you know, I'm a quick study, right.   Michael Hingson ** 03:38 But But you weren't invited to New Zealand for the World Cup this year. Hmm.   Kristin Smedley ** 03:42 Weird, right. And I was just looking at the at the pay rate of the top 10 Women's players and and I wasn't on there and I'm nowhere near that pay rate. So what's that about? Yeah, really. I've been playing longer than them.   Michael Hingson ** 03:53 So they're I don't know how to count for something.   Kristin Smedley ** 03:56 But yeah, I was very I was competitive and and love sports. And you know, being a Philly person. I don't know many people in our town that aren't Philly sports people. But I had a good time. I have four brothers, it was a crazy house. Very big family, lots of cousins. And, you know, just a typical, typical kind of kid growing up with dreams to be a teacher achieved all of that. And nothing, nothing really nothing really derailed plans at all until it did. Well.   Michael Hingson ** 04:33 And then it wasn't so much derailed. But it also goes to show that sometimes plans need to change. So along the way you you got a husband or whatever and, and did all that sort of stuff. I assume   Kristin Smedley ** 04:46 I did all the things that that everybody did. Right. And I mean, back then it was I mean like I said I'm going to be 52 Back then there wasn't a whole lot of of options. that that girls like me grew up with as as careers. My family was like there was absolutely no way that there was a future in soccer or sports for women back then. But I was I knew I was going to be a teacher from the time I was five years old. I am one of those bizarre people that just knew it from when I was very young. I would I would set up my my four brothers. In my dad's workshop at the back of our basement. He had this chalkboard and I would bring home the extra handouts from teachers at school and I would I would have my know why my brothers sat and did that. With me. I'd hand stuff out and I have them writing on the board.   Michael Hingson ** 05:40 They tolerated you.   Kristin Smedley ** 05:42 They sure did tolerate that's a great word, because they're still doing that.   Michael Hingson ** 05:46 I'm just gonna ask you if they still do that. They still tolerate   Kristin Smedley ** 05:49 me. They don't sit and let me hit him. They don't sell them handouts anymore lectures   Michael Hingson ** 05:52 anymore. Yeah, well, what so what did you teach? When you when you grew up and started teaching?   Kristin Smedley ** 05:58 I was an elementary school teacher.   Michael Hingson ** 06:01 Yes, it was my wife. I   Kristin Smedley ** 06:03 loved it. I just my whole life. I wanted to do that. And then when I was in the classroom, oh, boy, did I have a good time with that?   Michael Hingson ** 06:15 What? What grade did you want to teach? Or what grade did you find? Was your favorite grade?   Kristin Smedley ** 06:21 Well, that's it Sure. I will say first. And third, I never would have thought when I was when I was planning to be a teacher. I thought second grade was where it was that like that was where I really wanted to land. And I remember student teaching first grade, and I the first week. I remember coming coming back to the house, I lived in with a bunch of my friends at college. And they were like, they thought I had caught like a massive flu or something that I was exhausted my exhausted five days with first graders. And I said, I remember saying to my roommates, you even have to include in your directions not to eat the paste. To be very specific with first graders, but I love the fact that that first graders, just they kind of believe what you tell them. You know, they haven't really formed their their own individual personality. Some of them have, but most of them are along for a really fun ride, you know, third grade, though, they start developing their own personalities and the things that they they know that they want to do. But you're still cool. Third graders still think that teachers are cool. Fourth grade, they start to go maybe not. So I wanted to stay. I wanted to stay in the cool zone. My   Michael Hingson ** 07:42 wife loves third grade, she thought that that was the best grade to teach. Definitely the earlier grades. But she loved third grade the best because as you said, kids started to develop a personality, but you could affect it. You could teach them they would listen. But when you got beyond that, especially when you got to sixth and seventh grade, much less high school, of course, that got to be a real challenge. Oh,   Kristin Smedley ** 08:06 yeah. Oh, yeah. I have one of my best friends. We actually met at college orientation. She's taught middle school science for her whole career. And I'm like, Man, are we different? I couldn't I could not. I wouldn't accomplish anything with middle schoolers, but first and third grade. I'm your girl. That was a good time. I   Michael Hingson ** 08:28 suppose the idea of middle school science, though, is if you do interesting experiments, and you do things that they don't expect that is because they haven't really learned about a lot of that stuff. You can sort of keep their interest.   Kristin Smedley ** 08:43 Yeah, you know what, that's a that's a really good point. Because Stacy has kept it, she think makes things incredibly interesting. And I'm like, Oh, my goodness, she I've there's been times we've been sitting in and hanging out drinking wine, and she'll start showing me this, this PowerPoint of like, scientific stuff. And I'm like, and she's so into it. I'm thinking, okay, now I get it. I know why. No, I think kids were into it for all those years. Yeah.   Michael Hingson ** 09:11 But she's got the knack of being able to make it interesting for the kids. And of course, that's the issue. Right. Right. Right. And you're still teaching third grade in as you said, the cool zone. So that helps a lot. But you know, I, I know what you're saying. I remember. Oh, gosh, now it's been about 18 years ago, I was doing a talk in San Francisco. And I went to the school it was an elementary school K through six and the whole the whole school was there was an assembly and the teacher said Now look, you can't talk more than 15 minutes they will not sit and listen to you. Now we're sitting there going, just wait So of course, I come out with my guide, dog Roselle. If that isn't going to keep kids interested, give me a break. So like about 40 minutes after we started talking all about dogs and I talked a little bit about the World Trade Center, of course. But it was mostly what the dog did and how guide dogs work. And they all sat there and rapt attention. Then I finally opened the door to questions. And as I tell people, there's no question that anyone can ask me today that's off limits, because this third grade kid gets up a guy, right, a boy. And his question was, how do blind people have six? Oh, my God. And so, you know, no questions off limits? Well, I'm not dumb. I just said the same way everybody else does. And if you want to know more, you go ask your parents. You know, I'm not an idiot. But but you know, there's no question off limits. I've remembered that story ever since. But then the teachers came up afterwards. And they said, We don't know how you did it. And I said, it's the dog. And it's talking about the dog. And even the sixth graders were all interested. And, of course, everybody wanted to come and talk to the dog. So after it was over, I said at the end that if anybody wants to come up and visit with the puppy dog, they are welcome to do that. I knew Roselle very well. Roselle was one of those dogs who had discovered the scientific principle of maximum petting area, she would lay down on the floor and stretch out every appendage as far as she could to get as many people petting her at one time as and she loved it sweet. And, and all of my guide dogs have been that way. They and I wouldn't want it any other way. You know, the harness was off, and they just all love it. And the teachers kept an eye on things, but still, everyone got to come up and spend some time with Rosella. And she thought it was the greatest thing since sliced bread. Sliced bread too, but you know. But yeah, third graders, my wife always loved third graders and, and we've talked about it a lot. My teaching was at the high school level, I got a secondary teaching credential ended up going into other jobs. But I have my secondary teaching credential and, and taught, and I've actually had kids from my classes come up to me like 10 years later, and say, Do you remember me? And the voices of well, of course, all change. And I don't know, well, like one guy. I'm Marty, I was an eighth grader in your algebra one class in high school. And I remember coming into class and talking with you and solving problems with you. And Marty was actually, one day asked me a question, and I didn't know the answer. I just didn't happen to remember it. And I said, I'll go find out the answer, but I don't know it. And then the next day, I came in with the answer, but Marty did as well. And I said, alright, you come up and write it on the board. My master teacher said, That was incredibly smart, you did the best thing you could do, because these kids will know if you're blowing smoke. The fact that you said that you didn't know, scored you so many points. And that's really true. And it's I think is true today, and anything that we do, rather than bluffing your way through. It's better to be honest. I   Kristin Smedley ** 13:23 totally agree. And kids can, they can definitely. They can definitely tell. So every time Oh, yeah. No   Michael Hingson ** 13:33 doubt about it every single time they can tell those things. Well, so you taught and how long did you teach? A   Kristin Smedley ** 13:41 few years. And then I at the time I was married, we moved to Chicago. And that was after an extremely challenging third grade year was a great group of kids. But one of the remember at the end of that year, saying if I could survive that year, I can survive anything. I never should have said that out loud, because then all kinds of things happened. But I ended up going which was pretty cool. I want to take a break from the classroom for a little bit and was working with the Department of Ed and this is how old I am now that was back when we would go in and teach teachers and principals how to use technology in the classroom.   Michael Hingson ** 14:26 You're probably a lot of them will still need that but I hear you   Kristin Smedley ** 14:28 oh yeah, we actually organized big educational conferences and and it's funny how my life has gone because I said I always I had planned to be a teacher always wanted to be a teacher stayed in the teaching profession. But then as I watch everything that unfolded like those, planning those conferences and working with teams that were were in house and remote like it's all the things I'm doing now. All of those experiences gave me gave me experience As in being able to do the stuff I do now. So I always say to people, you know, when when, you know, when when you seem to have a roadblock, or are taking a different path for a little while, or maybe making a right turn where you thought you're going straight pay attention, because because every experience gives you tools for stuff that's coming later. Yeah,   Michael Hingson ** 15:20 and the, I think most successful people are the ones who realize that, and who can actually trace back and remember when they learned those tools and what they learned, I know that I believe our lives are really comprised of all the choices that we make. And all too often we forget the choices we make. And I think it's important. And I worked very hard at remembering what led me to where I am. And it doesn't mean that it was bad. Even if it didn't turn out the way I expected to. There's still things you'll learn along the way. Yeah,   Kristin Smedley ** 15:56 you know, I'll even take that a step further and say, I'm realizing now like, like, literally, within the past seven, eight months, when, when a sidestep or something or setback happens, I now pay attention in the moments of new things that I need to learn new perspectives that I need to have my eyes open to, like, instead of waiting until later, like I always did. Now I'm actually on the one of my friends, Chip Baker says, grow through your go through. So when you're going through something, what is what are all the growth opportunities that you can have your eyes open to and I'm telling you, it makes it makes it not that it takes struggle away or stress away, but it makes it a heck of a lot easier.   Michael Hingson ** 16:43 It does. Because everything that you do is a learning experience, no matter whether you think you learned something or not. You did. And, and just we don't pay nearly as much attention to that, which is not not really the way it ought to be. I love that go through your growth. grow through your go through. Yeah, tongue twister, but still. Well, so you've referred to it a whole bunch of times. So things sort of started to make you deviate and you had sudden unexpected changes. Tell us about some of that, if you would. Yeah,   Kristin Smedley ** 17:21 you know, I was at a point in my life where the Christian in the year 2000, I can tell you that Christian of 2023, I'm not sure that they would be friends. Because Christian back then had. I mean, like I said, I had planned, I had planned to be a teacher, I plan to be successful, I plan to get married everything I accomplished everything I had, you know, gotten the degree landed the job, married the guy at the got the big house, bought the brand new SUV. And, and my final not necessarily final dream, but my biggest dream of all was finally coming to fruition to fruition. And that was becoming a mom. And I have to tell you, Michael, I had an incredible Mom, I have I have a wonderful role model for mom and my grandmother, her mom was wonderful. And I was surrounded by a lot of people that were really good moms. And I of course, being competitive, couldn't wait to be a mom and do even better, right? Like I was even going to be even better than all of them. And, you know, most people they find out they're pregnant, and they're like, I just want a healthy baby. Right? And then and that's what I did. And then by when you're me by like month eight, it's Oh, is he going to be a pitcher for the Phillies or quarterback for the Eagles? Right? And is he? You know, will college really go to and and you know, you're envisioning all of the things. And when he was born, he was Michael was born in January of 2000. And on our street. Now, if you remember back then it was y2k was happening. And this was January, like we survived the computers, right. But there was I didn't even realize it at the time. There were so many people trying to have a y2k baby. So, on my street in January, there was like, eight people had babies within eight days of each other. It was crazy. Crazy. So everyone was in the hoopla of new babies and and, you know, the hospital stays and we would all be we weren't necessarily outside in Chicago in January, but we'd be in each other's Kitchens talking about all the things and that book, What to Expect When You're Expecting we knew every line of every page and, and all that stuff and talked about everything. And then I started noticing something about Michael was different from the other kids and I had gone back to work and had him a friend of mine who's a nurse was babysitting him every day. And I said to her Is it weird that When, when you lay him down on his back, his eyes swirl around and disappear. And she said, yeah, it is weird. You need to have that checked out. And after a few weeks of of no answers, and lots and lots and lots of tests, we finally flew home to Philly, and got an appointment with a specialist at Children's Hospital Philadelphia. And that's where that's where I heard a sentence that I had not planned for. And that's when he said to me, Kristen, your son is blind. And, you know, the planner in me that was not in the plans like valedictorian, summa cum laude, you know, professional athlete, those were the plans. And in that moment, I gotta tell you this, this, I can say it now I was embarrassed about this for years, but since my kids are successful, and I, I turned out, okay, I can tell you my first question to that doctor was like, I was trying to consider how blind I didn't understand blindness, right. And I said, Willie, play baseball. Can you imagine that? That doctor probably tells that story at parties all the time. at conferences with other ophthalmologist right was the dumbest question you ever heard? Yeah, I was. I said, Well, we play baseball. I was trying to get it in my mind. I just had an absolutely zero knowledge of blindness. And the doctor, of course, said no, he's not going to play baseball. And it was like everything. Willie drive. Now, will you go to school? Probably Probably not a regular school, all the things. And I said, Oh, my gosh, well, what is he going to do? And the doctor said, I have no idea but good luck. Now, oh, that was nice. Right? You know, and I, I'm like, Well, that was 23 years ago when that happened. But it's still happening. Doctors are still saying, Yeah, I don't know what to tell you. I have one of my friends that has choroideremia. He says, doctors are saying, Go home go blind. We got nothing for you. So we can get into that later. But well, we can because   Michael Hingson ** 22:00 it's absolutely worth doing. Doctors still believe that if they can't save your eyesight, they're failures. ophthalmology, schools don't teach the eyesight really isn't the only game in town, which doesn't mean you don't try to save eyesight. But eyesight is not the only game in town. And we don't deal with that nearly enough.   Kristin Smedley ** 22:18 Yeah, yeah, that was I've, I've often talked with, with folks about the fact that you know that there's that first do no harm for doctors. And I think it is, it is more than harmful to not send a family on their way with some kind of resources or, or one resource. that's ultimately why I ended up writing my first book, I'm like, if no one's handing over, if there's no resource to hand them, we're going to make a resource to hand over. But yeah, that's where I started. And I was actually just talking with somebody yesterday about this concept in terms of parenting, I believe now, when I heard those words, and you know, heard good luck, I literally crashed to the floor and all of my dreams had crashed to the floor. And I had no education, knowledge experience with blindness. I will say, I think the greatest thing that happened to me in my life, was that all of my dreams for my kids crash to the floor. Because when I'm noticing even even myself, I do have a sighted daughter also. With kids, I'm I'm seeing our biggest struggles, their biggest struggles and stress come from, they're walking away around with carrying the weight of their own dreams and ambition. But they also have ours on top of them and I one of the greatest things that ever happened to me and my kids was that everything that I had planned for them was eliminated because I didn't think it was possible and I had to I had to literally just I said to the boys I'm gonna I'm gonna get you what you need and follow your lead because I have no idea where this is going. Thank God thank God it wasn't there wasn't anything that I had intended for them that they went after at because that would have been such a limited life when I looked back on their on their where they're at now. So   Michael Hingson ** 24:17 what caused Michaels blindness.   Kristin Smedley ** 24:21 So we will find out later that the umbrella disease it's a it's an early retinitis pigmentosa it's Leber's congenital amaurosis, and we didn't find I didn't find out until oh nine that it's the CRB one gene causing it. Okay. Yeah. So and with with each pregnancy now for all the math minds out there you'll love this part and everyone else just hang in there because I don't talk about math all that long. I do have lip gloss on so I don't do math and statistics when I wear lip gloss. It's a rule that I have, but To the with CRB one LCA, there's a 25% chance with each pregnancy that the child will be affected. Now, optimistic Kristin, which people have said that my my memoir could be titled delirious optimist, heard 25% chance and stop listening. So I was like, well, I already you know, one in four babies, I already had one. So we're good, you know. And then I remember one of the specialists was like no Christian with each pregnancy. So it took it took me a while to get my head around how I was going to raise Michael. But I have to say ultimately, and and I believe the statistics are still that most LCA families there's I think 30 genes now identified in LCA. But most LCA families, once they have that baby, the LCA child, they don't have any after that. Because most people don't want to hear a second diagnosis or don't want to experience a second diagnosis. I was quite optimistic. But when I was really weighing all of it, to be honest, I thought, I started interviewing people, I'm a little bit of a nerd like that, like, I want to have as much information as I possibly can. And I talk to everybody that I possibly can. And I went and talked to people that I knew that were only children, because I couldn't get my mind. I couldn't get my head around blindness, but I could not get my head around an only child. And heard pros and cons of it and everything. And I thought ultimately, I would have a harder time raising a child that was a single child, then raising a child that couldn't see I figured I could figure out blindness much faster and better than I could figure out how to have a an only child. It just I guess it was just, you know, I was what 28 At that time and, and my experience had just been a big family was all I knew. And gosh, we I mean, it was crazy. But boy, did we have a heck of a lot of fun with cousins and everything. So you know, ultimately, I just I was like, let's let's go for it. And I was like, come on, what are the chances really? Like, I'm always in that 75% camp. I'm always on the better end of statistics, right? Oh, my goodness. And then and then. A family member always says with a very cynical tone that we hit the lottery twice, because Mitchell was diagnosed with CRB one LCA also. But I will say that I do say we hit the lottery. Three times, all three of my kids are extraordinary human beings. And I can't even imagine if if it was if I had an only child, I mean, I love Michael. He's great. But the dynamics of what all three of them have brought to my world are just incredible. And they're all different. Oh, boy, are they all different? The one retina specialist in Boston said after a day of testing, he goes Chris to any experience the boys for the day. He said they are different down to their retinas. Even the retinas aren't the same all.   Michael Hingson ** 28:19 So now we measure a difference by our retinas. Okay, and works. You know, but going back to what we talked about earlier, the whole issue of how the medical profession deals with it. It is so frustrating. I mean, you, you read the underdog. I read that years ago. Yeah. So you, you read my story. And the doctors told my parents that they should just send me to a home because no child who is blind could ever amount to anything. And my parents said no. And we we went from there, I don't know, never really talked about their fears. But I think if I had asked my parents tell me about your fears, they would have said no, we just assumed you would grow up to do whatever you chose to do. I think the fears were, were there in one way or another. But they just felt that. So all right, you're blind. We'll deal with it. And they were risk takers by any standard. But I don't even think they would classify themselves as risk takers. They were very unusual in the way they approached it, but they did. And the fact is that I got to grow up and do the things I wanted to do. And I always wanted to teach, but I ever ended up actually doing teaching in the classroom past student teaching. But I learned along the way that when I was confronted with a situation where I would either lose a job or go from doing scientific human factor studies into sales and chose to, as I love to say lower my standards and go from side It's the sales that in reality, UI though, in reality sales is if you do it, right, more teaching than anything else in the world, it's all about teaching. And it's all about helping people understand. But it's also because of that, about listening. And it's, it's important to do all of that. But the fact is that blind kids have as much opportunity to grow, or should have as much opportunity to grow and be whatever they choose, as anyone else. And part of the burden that we face is the prejudice that everyone has about blindness.   Kristin Smedley ** 30:39 You know what that that's, that's, oh, my gosh, I'm taking a deep breath, because it is so frustrating to me that in this day and age, that that bias is still there with with all of the you know, you feel like you do all this advocacy and your stories out there. And my social media platforms are huge. And there's all these other stories out there, and people still have no idea I just did an event here in in my town where my boys have grown up and done all kinds of things. And we are I mean, you're, you're kinda it's hard to not be famous in a small town when you got two blind kids. I mean, everybody knows who we are, everyone has seen all the stuff that they do. And I did an event with a short film that I just co created about the bias against blindness, and hiring people that are blind. And after the film, people that have watched, I mean, elected officials that I know very well, incredibly smart, successful. People were coming up to me saying, Oh, my gosh, I had no idea that blank, people could do all the things and I recite, but you you had two examples in front of you for two decades. How is that possible? I guess they figured my kids were some anomaly or I was constantly opening doors for them. I don't know. But they were blown away. And I was, it was a weird, I don't know what the word is for it. I have to have to go into chat GPT to give me some words for this. But it's it was like angry and happy at the same time. They're not angry, astonished. Yeah. And happy at the same time that the 20 Minute. Video got through to them. But I thought how could you not know. But that's that is how it is?   Michael Hingson ** 32:22 Well, you know, and I joined the National Federation of the Blind in 1972. It's a consumer organization, I'm sure you've heard of it. And it does a lot of things. But even with a lot of blind consumers, who have adopted a philosophy that blindness isn't the problem, we are not having a lot of success, at changing people's attitudes, not nearly the success that we have to have, in order to truly make it possible for us to have the same opportunities as everyone else. And the consumer organizations can help they do help. The National Federation of the Blind, and its legal efforts, changed the insurance industry so that blind people could buy insurance, you know, back in the 1980s, no blind or other person with a disability, physical disability could buy insurance because the insurance industry said you have a higher mortality rate, you're a higher risk. And wow happened was that somebody came along and said, You do everything based on scientific data and evidence, Where's the proof? And they said, Well, we have it, but they could never produce it because it didn't really exist. It was all based on prejudice. So by around 1985, legislation had been passed in every state saying you can't discriminate unless you got the proof. But the fact is, it was still there. There's still the attitudes and even that didn't deal with it. And I think part of the if I were to say one thing that doesn't happen that needs to really make a difference is we've got to become more part of the conversation, the whole human dialogue. And we're just not even some of the so called Disability experts. Don't push enough. We need to be in the conversation a lot more. Oh,   Kristin Smedley ** 34:14 I 100% agree and and we also need to be in every facet of life that sighted people are in right I think that's why I'm so passionate with, with stories with with, especially the children's book that I just put out and film and Hollywood, I tell you this, I put a post on Twitter, or x, whatever it's called these days. Yeah, I'm just gonna go with Twitter. Another story. Oh, my. Anyway, I put a post about my son Mitch, who's home for the summer from college. In our home, we are addicted to the show suits. I don't know if you follow that show.   Michael Hingson ** 34:56 I don't I've heard of it. I gotta watch it.   Kristin Smedley ** 34:58 I gotta Oh my gosh, one Now it's on. It's on Netflix. And we're we're rewatching the whole series. We watched it through COVID. And now we're rewatching it and it is I mean, it's attorneys and it's it's egos and it's just great. We love it. And we all have our favorite characters will Mitchell who just turned 20 years old, literally bounced, like jumped out of his stool. He sits on this little this funny little stool, the cracks, we have this big, tall 20 year old with an attitude right sits on this little stool in front of this giant TV and is glued to the show, jumps off the stool and bounces into the kitchen yelling mom. Netflix put audio description on suits. Yeah, he you would have thought that he just got like his the the bike he always wanted for Christmas, you know, like he was so excited. So I put a post out on Twitter that said, Oh my gosh, that feeling when your son bounces in the room and I put a thing about how him announcing that Netflix, put audio description on suits. And I said, Thank you so much Netflix, for being inclusive, whatever. I did a hashtag that the suits, I didn't realize how passionate the suits community is about that hashtag. It is now I think it's at 7000 people it's reached, people went crazy. We didn't know that was a thing. Oh my gosh, tell us more. What is audio description. And then um, that was like this teachable moment. But people have absolutely no idea that something like that is out there. But it also, you know, it went back to my point of when people that are blind are involved in all facets of life. That's when the education really starts to spread. And that's when perspectives are shifted. And that's when I see the bias disappear. I mean, my when my boys oh my gosh, I will never actually I'm writing the screenplay now for the moment. And I just wrote out the moment, the scene that we experienced when Michael told me, he wanted to play Little League baseball in our town when he was nine. I mean, he was playing blind baseball in the city. And but he was going to public school. And he wanted in on those lunchtime conversations where all the kids are screaming at each other of whose team cheated in which arm sock and all that stuff, right? And he's like, I want to play baseball, I said you do play baseball. And he's like, No, I want to play baseball here in this league where all my friends play. And when I walked up on registration day to baseball registration. When I talk about this, I should actually have like a button I hit with music that's like, you know, it's more than Disney World and all these happy cheering people that are there for registration. And Michael walked in with his white cane and said he wanted to play baseball. And as grouchy and grumpy as the Commissioner is of a person, I will give them the credit that he did give it some thought and long story short, Michael ended up on a baseball team. And in his second year, they won the championship like they were the worst team in the league and came back and won the championship and, and he was an all star and led the team in RBIs. And there was a dad that I knew was not happy. Michael was on that team at the first practice that came up to me after that championship when and he said, he said Kristen, you know, when all of our kids started this season, and came together, they were all just a bunch of spoiled kids to get everything they want. And he said one by one, your son changed all of them. And that changed all of us and watching him has been phenomenal. And I thought that's what it is. It's it's when it's when we're out in the world in all facets of life, doing life, that we change those perceptions and those biases. So so I want people that are blind and visually impaired and their parents and everybody around them, get out there in the world. And like you said, be in the conversations be in the experiences. And if we can, if we can multiply that then I think that we can really get rid of this bias a lot faster.   Michael Hingson ** 39:11 So how did Michael play baseball? Well, interesting.   Kristin Smedley ** 39:14 Now I'm in so many conversations, you know, in the ENI stuff and workplaces and we keep saying reasonable accommodation. I'm like what I didn't know it was what we were I was asking at the time, but it was reasonable accommodations I we weren't changing rules. We didn't change much. But he was able to hit off a tee now this is 910 11 year olds, they were they were kid pitching hit off a tee. And he played in the outfield with another with another guy. That guy would feel the ball and Michael had to throw it in to where the play was okay. Then I I've actually spoken at some sports stuff. And you know on the topic of parents and and sports I say I always say listen for coaches. If you have problem with parents and vocal parents and how, you know, parents have become a nightmare at youth sports, get a blind kid on your team. Because when Michael we get the ball when that guy would hand it to him in the outfield, he had to listen to one voice to know where to throw the ball so that the kids learned quick and they shut the parents down even quicker. No, but as soon as he got that ball, it would be silent. And one person if the kid if the play was at second, that kid would stand there and call Michael's name, and he could throw that ball to him on a dime. It was really cool. Now, for people that are listening or thinking, Okay, at this point, you know, Michael's nine and a half 10. And I'm saying to him, you have to hit off a tee he did not he did not initially, he wasn't on board with that. He was like, no one's hitting off a tee. That's, that's, that's stupid. No one, there is no tee. In this in this league, I want to swing at the pitchers like everybody else. And it was an interesting conversation that night that I said, you know, you can do that if you're against the tee. And you think that that's what you should be doing. But let's think about this. Those I've seen in this age group pitch, there is no consistency. It's not like it's gonna they're even going to try to help you out and direct that pitch, you know? And I said, and you still don't, we're not changing the rule, you still only get three strikes and you're out water. And Michael's a very scientific, math minded kind of kid. I said, what's the probability that you're going to hit that ball with that kid pitching it. So then he went into a whole thing about velocity, and oh, my god, he like nerded out on science about the ball not moving and an object not moving. And I was like, guess what I've turned his light off. I'm like, good night. We'll talk about this tomorrow. And the next morning, we sat there eating breakfast. And he said, he said, I'm not happy about having to hit off a tee. But I don't want to, I don't want to let the team down. And I don't want to be that guy that they can count on will be an out every time I get to the plate. So he did, he had to set up the tee on his own, put the ball on there. And and he got, you know, if there was if he missed it three times, he was out. He never did miss three times he got on base almost every single time he actually led the team in RBIs. That very first season and I said you know you didn't contribute by hitting a home run. But you sure did set everybody else up to cross home plate.   Michael Hingson ** 42:33 I presume he had to practice a lot though, to be able to hit it and make good contact.   Kristin Smedley ** 42:38 Oh, oh my gosh, the practice. And I will say this for parents that are listening. We did I want to make sure I re emphasize he did start in blind baseball. Like he had people that were trained in how to teach a blind child baseball. So he knew the mechanics of swinging the bat connecting with the ball, throwing the ball, like he knew all that. And then we just did I mean, I played softball, my whole my whole childhood. So I have some skill there. And we just practiced and practiced and practice and we would get to the games early and run the bases run the bases just so we had that memory of where the those bases were, when he ran his his coach the first year this guy Rich, who was absolutely tremendous. He didn't he just he was on board with everything. But he did not want him out there running on his own and having a sound box or something at the bases. That was where he drew the line. He was like he was too nervous. So I said okay, you know, he was on board with everything else. Let's let's not have them have a stroke here. Let's Okay, so rich would run with them. But as Michael got more and more confident and really knew where those bases were, he was getting faster and faster. And then there was fewer so play in the one game, and it was a tight game. And the kid the kid just clocked this ball and everyone on base Michael was at first the bases were loaded. Now they're running and they're rounding the bases. And Michael enricher running and they turn third and Michael just he just guns it for home and he outran rich so and then all the parents instead of cheering for Michael they were cheering for rich to run faster.   Michael Hingson ** 44:20 Well, you know Rich needs all the help he   Kristin Smedley ** 44:22 can get to was so funny. It was so quiet. And then he looked like we were like, oh god, somebody better get rich some oxygen and I'm like, You think maybe it's time that my   Michael Hingson ** 44:34 zone? Yeah. And what happened?   Kristin Smedley ** 44:39 He did what he ended up doing he would go to like second and just call his name once but he was he hadn't he had a valid concern. He was nervous that if Michael You know, yeah, would do it himself and was on second waiting for a hit. He would never be able to duck. If the ball was coming at him and we didn't I didn't want to rely on on a nine year old to be standing there and tell him So we just had to coach out of the base and it worked out just fine.   Michael Hingson ** 45:02 And, you know, we get back to the whole discussion that you sort of alluded to a reasonable accommodation there. The reality is that there's no reason not to allow for accommodations to permit people who are different than we to be able to perform the same thing. And, again, we we really, collectively, I think, misjudge it a lot. But the fact is that Michael obviously proved he could do it. Now what admits do, did he play baseball or any of that?   Kristin Smedley ** 45:39 Oh, he sure did. He a few years later was on the exact same team. So that was Michael for his first season. The second season is when they won the championship. And I remember watching the whole thing unfold. And then when the whole town was on our sideline, watching and everything that happened, I thought, oh my gosh, this this is like, this is like the feel good movie of the year. And I would talk about I'm like, I gotta make this into a movie. Mitch played a few years later, this same orange Mets team, we are Phillies people, the fact that I have had two kids on teams called the Mets was brutal. But anyway, he was on that team. And, and he won the championship. Also, Mitchell was a completely different. He's a completely different kind of kid made a completely different impact, equally huge impact. But he was they had to figure out real quick about him running the bases because he wanted to steal second, he didn't want to just run the second one, there was a hit. Yeah, he wanted to steal bases, he figured out he was actually the fastest base runner on the Team Mitchell is quick. And he has an even if it's even possible, and even better spatial memory. Or maybe because he has this little see had this little sliver of vision in the in the right corner of his right eye. And if he tilts his head, just so and he was so much smaller and closer to the ground, maybe he was able to navigate the bass line a little better. But he did the same thing. He hit off a tee. And he played the outfield. And I have this I have this incredible picture of him and his best friend Nick, on that team. And Nick's dad was the coach Mitchell, you know, Michael and Mitchell and Shay achieve everything they want in a day. Right? Michael will do it all by himself. I mean, if he was he was moving in Florida the other day, and I swear he was going to try to figure out a way to get a U haul on his own because he did not want to wait for somebody, right? He does, as much as he can all by himself accomplishes everything. And he's exhausted at the end of the day. Mitch uses every ounce of charm, good looks everything to get people to do things for him to accomplish once and he's so he's so crazy with it that that when they would him and his buddy would come in from the outfield. I have a picture of it. Mitchell would hop on Nick's back like Oh, Nick, my legs are tired. We've been out here the whole day. Give me a ride. And he could run with Michel   Michael Hingson ** 48:10 blindness issue? Nope.   Kristin Smedley ** 48:14 It's a laziness issue.   Michael Hingson ** 48:17 Now Oh, my goodness Michael doing today. You said Mitch was in college still. Yeah. Michael   Kristin Smedley ** 48:23 Michael graduated Penn State last year. And you know, I had said that one of my things I thought about was summa cum laude. And sure enough, he was summa cum laude from Penn State. And he had two majors, two minors and a business certificate. There were a couple of semesters that he took 28 credits, they now have a law and Penn State you can't do that. I said, if I get a second tuition bill, that they think there's two of you, you're gonna have to stop doing this. But he's, he was a communications and, and audio engineering, double major. And now he's at Disney. In, in a situation where it's only Michael, I always say I'm coming back in my next life is my son, Michael, because things work out for him in ways that are just unbelievable. But he My mom always says Michael wakes up every day expecting it to be the greatest day and everything to work out. And sure enough, that's what happens for him. But he started with Disney in the live entertainment, doing sound design and things like that. And then he had an opportunity to slide over to working in contracts, and he eventually wants to go to law school and be in copyright law and stuff like that. So he went, he's like, Oh, I could try that out for a little bit. So they're holding only Michael. They're holding his position, the first position while he tries the other one for six months and then decides what he wants to do. In this day and age where 70% of this community is unemployed. People aren't even going to work companies can't get people to work. And then they say to Michael Michaels, like I want to try this and you want to hang on to that. position in case they don't like it. And they said, sure they're loving them down there.   Michael Hingson ** 50:05 You know, you're speaking of Disney and you're talking about descriptions, descriptive audio descriptions. We got the Disney Channel, my wife and I signed up for Disney in 2019, because we wanted to watch Hamilton. And I assumed that it would be audio described and it was, but before I watched Hamilton, I decided, I want to go see one of my favorite Disney movies, if they haven't the sign of Zorro, which goes back to I think 1959 with Guy Williams. And it was audio described, Disney has done a wonderful job of putting in audio descriptions on everything. I haven't watched Davy Crockett yet with this, Parker, but I know it's going to be audio described. Oh, man, it's really amazing that they have done such a tremendous job of putting audio descriptions on the things that they do, which is wonderful.   Kristin Smedley ** 51:00 Oh, yeah. Well, he Michael said they are they are so majorly focused on accessibility and all that they're doing now, especially at the parks, and he's on committees and, and all kinds of things working on his ideas for it's actually how he got the job. He in his interview, you know, there's the whole thing in the blind community, whether you disclose or don't disclose your blindness in the interview. And I said to him, I go, of course, that we were coming out of the, you know, we were in in zoom times coming out of COVID, when he had that interview, and I said, Of course you it's your luck that you get to do a zoom interview, and they will never know Michael is very good at at setting up the camera and the lighting and looking straight on. So there was nothing to tell anybody physically, visually, that he's blind. And I said, Are you going to disclose you're not and he was like, I have no idea. And he was five minutes before the interview, he still wasn't sure what he was going to do. And it just, uh, conversely, he called me afterwards, he said, Well, I made the person cry. And I said, Oh, my God. He said that they he went, they went through all the technical questions. And then there was something to the effect of, of how can you make Disney an even better company? Or what can you really bring to the table, something along those lines? Well, I told a story about growing up. We used to go to the Disney Parks every year. And he said, one of the biggest reasons he loved going to the Disney Parks is that they thought about kids like him, they thought about people that access the world differently. So he could have a phenomenal experience just like his sighted friends, and they could talk a lot about everything that they got out of being at the parks, he didn't feel like he missed out on anything. And he said, he said but also, knowing what I know now and and, and the things that I know professionally, we can make it 10 times better for all abilities, disabilities, all different ways that people access the world. I mean, he said it much more eloquently than that. And it was absolutely magnificent. And, and he ne harped on the fact that it was because of his blindness, that he'd be the biggest asset because he really knew that the couple of tweaks that they needed to do. And then this woman ended up in tears because she said she had never heard somebody so passionate, and so confident that they could make the changes that would enhance the company. And she was in full belief that that would happen. So after he tells me this whole thing I said I'm so what you're saying is you disclosed.   Michael Hingson ** 53:34 It has always been a debate. And I realized, well as back in 1989. I had owned my own company for four years selling CAD computer aided design systems to architects. I didn't need to work the system. All I needed to know was how to work it. And I decided though eventually I was going to go back into the workforce. So my wife and I were looking at jobs, and we found this great one that sounded perfect. And we talked about do you say you're blind or not. And finally, I went off and I wrote a cover letter. And I decided I'm a sales guy. Sure I should be able to talk about and so I wrote, in the cover letter, I said, the most important thing that you need to know about me is that I'm blind. And the reason that's important is because I have as a blind person, have had to sell all of my life to convince people to let me buy a house, take a guide dog into places because we didn't have the ADA back then rent an apartment, go into grocery stores or do anything else that I wanted to do. So do you want to hire somebody who comes in for eight or 10 hours a day? And then they go home because the job is over? Or do you want to hire somebody who truly understands sales for the science and art that it is and sells 24 hours a day as a way of life? And I got the job because of that   Kristin Smedley ** 54:46 that's that's what I love. It's it's it's so many and I was I was the same way for so long until recently looking at it as Oh, I got it. I gotta convince these people that this It's okay. And my kids are okay. And it's going to be just I gotta convince them to give them the chance now I'm like, chance. Are you kidding me? Hiring someone like, like, my Michael gives you the competitive edge? I'm like, exactly. They've got skills they've been practicing oh my gosh, when everyone was talking about being resilient after COVID Michaels like if I hear resilient, one more stinking time, he's like, we have been resilient 57 times a day since the day we were born, like, oh my gosh, it was so funny. He's like, Oh, this 82 People are being resilient. Now.   Michael Hingson ** 55:32 It's a beautiful thing. You know, and we, we keep hearing, and I heard it so often after September 11, we got to get back to normal. And it took me a while to realize that's ridiculous. We can't get back to normal or it'll happen again, normal will never be the same. And I hear it after COVID and everything else. And we, we really need to, to look at things differently than we do. And we need to give everyone the opportunity to use their gifts, to be able to to thrive as much as they can. We talked about conversation, one of the things that I think we knew need to do collectively is to change words we use. I've never I've learned not to be a fan of the whole concept of blind and visually impaired. And I and I realized that my problem with visually impaired after thinking it through was twofold. One, just because I'm blind I'm not visually different visually has nothing to do with that's what the experts did, to screw it up and impaired compared to what why do I need to be compared with eyesight? So I believe that blind low vision is a much more accurate terminology. Deaf people realize that some time ago they will bristle or maybe eliminate you from the world. If you say hearing impaired, for the very reason, you know, visually impaired is is a horrible thing. But that continues to promote the attitude that we really need to change.   Kristin Smedley ** 56:56 Yeah, I 100% agree. And actually, when I'm when I was writing my children's book last summer, I wrote it and then it just came out a few months ago, I have a friend that was my educational consultant on it in terms of words and language. And it's geared specifically for first graders for six year olds. It's best not well, actually, it was funny because it was at school, it was this is how I love looking back on my journey and seeing where everything just aligned beautifully. And this is why I had said earlier, I really pay attention now when things happen to take it all in so that I don't have to wait 10 years to see the gain, as opposed to the loss. So yeah, when I was getting so frustrated in my work with with my first book, thriving blind was wonderful. I mean, it was, you know, 13 people that were role models for me and my boys, I'm sharing with the world. And so that opened a lot of doors to a lot of stages and a lot of conversations. And then you know, with the this unemployment statistics, and I do the short film to convince companies and adults, I felt like I've just I've worked 24 hours a day. And I'm still kind of banging my head against the wall trying to change the biases of adults. And I said to myself, What if the bias never happens in the first place? What if What if kids come into the world with a whole different story about blindness, just like the kids that grew up with my kids, those kids that grew up with my kids, they're out in the world, they'll meet a blind person, and I'm certain that they're like, what football team? Were you on? What position? Did you play in baseball? You know, how many college degrees you have, they have a whole different view of blindness than the rest of the world. So I said, How about if we did, if I do a children's book, that we tell them from the very beginning that differences so it's not that they don't matter? It's that it's what makes you unique, and it's what's inside of you and what you believe about yourself, is what matters because the world is going to tell you a whole bunch of different things. And so to the point of my educational consultant she was looking at it as making it educationally sound for to be in schools and align with curriculum and all that kind of stuff. But we went through every single word to make sure that every single word was empowering and not you know, there's no you don't the word disability isn't even in the book its abilities we say we all my the words are it's in first person about the child telling themself all this and it's about my abilities make me who make me me. It's there's nothing about this in there.   Michael Hingson ** 59:43 Well, what I've also realized is that there's nothing wrong with the term disability. It's a characteristic and the reality is disability doesn't need to mean lack of ability and sighted people Have as much a disability as blind people, except that since Thomas Edison invented the electric light bulb in 1877, we've covered up your disability by making sure that you have light everywhere you go. But it doesn't change the fact that it's there. And we really don't deal with with the whole issue at all. But you know, I was in a hotel in March, and then the power went out. And so when it did, of course, everybody started to scream because they couldn't see and they were grabbing for their phones and flat or looking for flashlights, and all that proves my point. The fact is that disability doesn't mean a lack of ability. And we all have that characteristic, in one way or another. And it's high time that we start to move away from thinking and just because some people's characteristics are more visible than others, that they're less than we are, that's just not true. Yeah.   Kristin Smedley ** 1:00:54 Yeah. And honestly, when that message was delivered to my Michael, by way of Eric weimarer, the mountain climber when he was Michael was six, when he met Eric Eric had just come off Everest, back then. And was was being honored in the city of Philadelphia with this big award. And I took Michael down there to meet them. And I'm looking at my little Michael, right, and his little suit, he was he was short for his age. And I watched his, I watched the moment of him talking with Eric and realizing in his own little mind, oh, my gosh, this blind guy is the coolest, he's just like me, he just did the coolest thing. And I've never heard I've never, he never met a sighted person that climbed Everest, you know, we've never met anybody that cool. And this guy happened to be just like him. So in that moment, at six, Michael believes that anything was possible for him. And he listened Eric talks in the speech that he gave, and in the conversation with Michael, it was all about the tools that he had to, had to figure out like he was in full responsibility for, he took full responsibility to achieve that goal. And it was all on him to achieve it, and he believed he could do it, and he found people to help them. That was the message that Michael got that day. And it never wavered. It has never wavered in his mind that he, he believes that things are possible for him, he just has to go get the tools and build a team and do all the things. So I thought, Gosh, I need all six year olds, whether they're blind, sighted, deaf, whatever, to understand that, or to at least get the correct story. That what they can do in this world is up to them, not what other people think about them. And let's let's change that and put the correct story out with the little kids because I'm tired of changing adults minds. Much harder work, it's much easier. And because I taught first grade and was like, I was like a Broadway show with some of the books that I really love. But this book is just like going on a bear hunt, you know, and you're all these actions, and you're meeting these cool people. And then you don't even know that they're blind until the end of the story. Like it's just, you're riding a skateboard and, and you're climbing a mountain, you're painting a picture, like it's just really fun stuff. Because I also I feel like so much of the information that's there for people to get educated about blindness is boring, or it's like heavy, right? Like, it's, it's a lot for people to take in where I'm like, why isn't it just part of the regular story? You know, it's just a regular story happening. And oh, by the way, here's the tools that they use to be able to do that. Because they don't, they're, they don't use their eyes to see like, no big deal. No, but let's talk about that. You know, like, it's just simple and fun. And let's change the story from like I've been saying from the youngest sets of eyes. Is there an accessible version of the book? It's only printed in an accessible version. It's in print and Braille. It's in print in Braille. Yeah, that's the only format we're doing it in right now. Which is incredibly

Our teacher today is Jeff Marrazzo, Co-Founder and former CEO of Spark Therapeutics. Under Jeff's leadership, this biotech company was the very first in history to receive FDA approval for a gene therapy. Spark has helped to ignite and expand the art of what's possible and how we will seek to treat and potentially cure hundreds of genetic conditions over the coming decades. Jeff provides us with an insider's overview of the biotechnology industry and shares the full story of Spark, from its humble origins in the labs of Children's Hospital of Philadelphia all the way through to its nearly $5 billion acquisition by Roche. I hope you enjoy our class with the inspiring Jeff Marrazzo.  For the full show notes, transcript, and links to mentioned content, check out the episode page here. ----- This episode is brought to you by Summus. Summus is a revolutionary health benefits solution, driving superior employee engagement while dramatically lowering your company's enterprise-level healthcare spend. They're completely transforming the world of health benefits by providing employers and their employees in any location, fast access to over 5,100 of the top medical specialists from America's very best medical centers for support across all health questions. If you're looking for a benefit that drives your employee engagement, truly takes care of your people in their most scary and vulnerable times, all while improving your healthcare ROI, visit GoSummus.info/AOI. This episode is brought to you by Hunt Club. Hunt Club unlocks relationships and helps companies grow. Whether it's recruiting your next hire, landing your next big partner, or financing your business, a trusted introduction always works more effectively. Hunt Club's tech-enabled search model leverages the largest community of its kind to refer amazing talent on your behalf. Additionally, its software program, Atlas, helps organize the entire network of a company or fund and assists in streamlining the right introductions. If you're looking for the ideal solution for all of your talent needs, visit HuntClub.com/AOI. ----- Art of Investing is a property of Pine Grove Studios in collaboration with Colossus, LLC. For more episodes of Art of Investing, visit staygrovey.com.  Stay up to date on all our podcasts by signing up to Colossus Weekly, our quick dive every Sunday highlighting the top business and investing concepts from our podcasts and the best of what we read that week. Sign up here. Follow us on Twitter: @ArtofInvest | @Buhrman_Rick | @PaulBuser | @JoinColossus Show Notes (00:06:20) - [First question] - Witnessing the impact of aiding others (00:10:45) - The genesis of the vision for Spark Theraputics (00:15:11) - Securing FDA approval (00:20:22) - Navigating the venture capital landscape (00:27:26) - Confronting periods of doubt (00:33:21) - The significance of gene therapy in the US (00:38:40) - Drawing inspiration for interdisciplinary collaboration (00:43:58) - Guidance for youth seeking to capitalize on opportunities (00:50:20) - Anticipating advancements in life sciences and healthcare (00:54:34) - The role of AI in advancing gene therapy (00:58:38) - Ways to engage with biotechnology (01:04:55) - Simplifying the complexities of biotechnology investment

Today we're speaking with Arun Kejariwal, and we will discuss pharmaceutical research and development commercialization in the pharma space. Arun has a lengthy career with all the major leading pharma companies, whether it be Merck, Pfizer, GSK, or Spark Therapeutics, where he is right now as the asset general manager for Huntington's disease and other CNS programs.

Kathy High, MD, shares her personal and scientific journey, from hemophilia to retinal disease, during her one-on-one conversation with American Society of Gene & Cell Therapy President Hans-Peter Kiem, MD, PhD, in the second episode of ASGCT's Giants of Gene Therapy. Dr. High is the president of therapeutics at AskBio. Before that, she was the co-founder, president, and head of research and development at Spark Therapeutics, where she oversaw the development, approval, and launch of Luxturna, the first AAV gene therapy approved for use in the United States. Dr. High has published extensively in the space of gene therapy and received many highly prestigious awards for her work. She is also a past president of the American Society of Gene & Cell Therapy. Music by: Steven O'Brienhttps://www.steven-obrien.net/ "Making Progress" (Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Dr. Katherine High, MD, is President, Therapeutics, at Asklepios BioPharmaceutical (AskBio - https://www.askbio.com/), where she is also member of the AskBio Board of Directors, and has responsibility for driving the strategic direction and execution of pre-clinical and clinical programs of the company. AskBio is a wholly owned and independently operated subsidiary of Bayer AG, set up as a fully integrated gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. Most recently, Dr. High was a Visiting Professor at Rockefeller University and previous to that, she served as President, Head of Research and Development, and a member of the Board of Directors at Spark Therapeutics (a subsidiary of Hoffmann-La Roche), where she directed the development and regulatory approval of Luxturna® (a gene therapy medication for the treatment of the ophthalmic condition Leber congenital amaurosis), and represents the first gene therapy for genetic disease to obtain regulatory approval in both the United States and Europe. Dr. High was a longtime member of the faculty at the University of Pennsylvania and medical staff at The Children's Hospital of Philadelphia, where she was also an Investigator of the Howard Hughes Medical Institute. She served a five-year term on the U.S. Food and Drug Administration Advisory Committee on Cell, Tissue and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy. Dr. High received her bachelor's degree in chemistry from Harvard University, an MD from the University of North Carolina School of Medicine, a hematology fellowship at Yale University, a business certification from the University of North Carolina Business School's Management Institute for Hospital Administrators and a master's degree from the University of Pennsylvania. She is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, and the faculty of Pharmaceutical Medicine of the Royal College of Physicians (London).

Jacob(Jake)Rubens, Co-Founder, Chief Innovation Officer Jake is Co-Founder and Chief Innovation Officer of Tessera Therapeutics, as well as a Principal at Flagship Pioneering. Jake joined Flagship Pioneering in 2015 and works as part of a venture-creation team, founding and growing companies based on new biotechnology. At Flagship, Jake launched Kaleido Biosciences and co-founded Sana Biotechnology. Jake was the Head of Innovation at Cobalt Biomedicine, where he invented and developed the company's Fusosome platform prior to its merger with Sana Biotechnology. Before joining Flagship, Jake received his Ph.D. in microbiology from MIT, working in the Synthetic Biology Center with Professor Tim Lu with the support of a National Science Foundation Graduate Research Fellowship. At MIT Jacob invented gene circuits that allow engineered cells to do novel analog, digital, and hybrid computations, enabling the emerging field of “intelligent” cell therapies. Jake's work has resulted in multiple pending patents and publications, including articles in Nature and Nature Communications. Jake was honored in 2017 in Forbes' 30 under 30 list in science. Hari Pujar, Chief Operating Officer Hari is Chief Operating Officer of Tessera, as well as Operating Partner at Flagship Pioneering. At Tessera, his responsibilities span across research, manufacturing, program strategy and management and IP. Hari is a global Biopharmaceutical executive with 20+ years of value creation in the biologics and vaccine industry. Before Flagship, Hari served as Chief Technology Officer of Spark Therapeutics, leading the technical operations, process and technology development and quality assurance organizations. At Spark, Hari was responsible for growing and scaling the company's cutting-edge technology capabilities for an expanding development pipeline. Prior to Spark, Hari was head of Technical Development & Manufacturing at Moderna Therapeutics. At Moderna, he built and led the technology and early manufacturing organizations that delivered on supply for more than a dozen clinical programs in a brand-new technology area. Previously, Hari held a variety of scientific and cross-functional leadership positions during 18+ years at Merck & Co in the company's commercial, R&D and manufacturing divisions, culminating in the franchise leadership of pediatric and adult vaccines representing over $2B in revenue. Hari has a Ph.D. in Chemical Engineering from the University of Delaware and an MBA from the Wharton School at the University of Pennsylvania. He is a Fellow of the American Chemical Society and the American Institute of Medical and Biological Engineers.

Many patients living in rural America encounter unique boundaries to receiving the critical healthcare they need, particularly when faced with a rare disorder or disease. March is Bleeding Disorders Awareness Month, and Dr. Len Valentino is working to educate and advocate for upwards of 10,000 rural Americans living with a rare blood or bleeding disorder. “Understanding rural health is critical to serving the population of the US.” ~Dr. Len Valentino As CEO of the National Hemophilia Foundation, Dr. Valentino brings more than 35 years of clinical and research experience related to inheritable blood disorders to the organization. Prior to his most recent work with Spark Therapeutics, a biotech startup, he founded and led the Hemophilia and Thrombophilia Center at Rush University Medical Center in Chicago, where he successfully balanced a $10 million budget to support research grants, research, and clinical teams–keeping the patient and their families as his core focus. He earned his undergraduate and medical degrees from Creighton University and Creighton University School of Medicine.  He then completed the University of Illinois at Chicago's Pediatric Medicine Residency before completing a fellowship in pediatric hematology-oncology at the David Geffen School of Medicine at UCLA. Dr. Valentino remains an active member of multiple professional organizations, including The American Society of Hematology; International Society of Thrombosis and Hemostasis; Medical Affairs Professional Society; and The Hemophilia and Thrombosis Research Society To learn more, check out www.hemophelia.org

Drs. Ozlem Goker-Alpan and Swati Sathe discuss how our growing awareness of the central symptoms and comorbidities associated with many lysosomal storage diseases is changing how we manage these rare diseases. This CME/CE activity is possible through an educational grant from Takeda, Cheisi, Ultragenyx Pharmaceuticals, and Spark Therapeutics.To obtain credit for this activity, please visit https://checkrare.com/learning-center/courses/

Drs. Ozlem Goker-Alpan and Uma Ramaswami discuss how the success of therapies to treat lysosomal storage disorders like Pompe disease, Gaucher disease, and various MPSs, has created phenotypes that previously did not exist. This CME/CE activity is possible through an educational grant from Takeda, Cheisi, Ultragenyx Pharmaceuticals, and Spark Therapeutics.To obtain credit for this activity, please visit https://checkrare.com/learning-center/courses/

Jay Newman, Head of U.S. Commercial at Spark Therapeutics provides a detailed overview of gene therapy for inherited retinal disease. Jay discusses the challenges associated with gaining regulatory approval and how this approval has impacted the company and the low vision community at large. 

Jeff Marrazzo co-founded Spark Therapeutics in 2013 and has led the growth of the company from a research center within the Children's Hospital of Philadelphia to a fully integrated, commercial gene therapy company. Under Jeff's leadership, Spark received regulatory approval for the first gene therapy for a genetic disease in both the U.S. and EU, launched the first gene therapy for a genetic disease in the U.S., and established human proof-of-concept of Spark's gene therapy platform in both the retina and liver. In more than eight years, Jeff has built an organization of more than 700 employees while securing over $1 billion in capital and successfully orchestrating a merger with Roche. Outside of his role with Spark, Jeff currently serves on the boards of the Life Science Cares Philadelphia and The Rendell Center for Civics and Civic Engagement. Jeff received a B.A. in economics and B.S.E. in systems science and engineering from the University of Pennsylvania. He also holds a dual M.B.A. / M.P.A. from The Wharton School of the University of Pennsylvania and Harvard Kennedy School, a program which he founded.

Spark Therapeutics' Clinical Development Lead, Hematology, Dr. Tiffany Chang explains the company's data from its ongoing Phase 1/2 clinical trial of investigational SPK-8011 for hemophilia A, the largest gene therapy trial in this disease to date as well as the misconceptions and unknowns about the disease.

Drs Ozlem Goker-Alpan and Gregory Grabowski discuss how new research into the pathophysiology of lysosomal storage diseases is changing how we manage these rare diseases. . This CME/CE activity is possible through an educational grant from Takeda, Cheisi, Ultragenyx Pharmaceuticals, and Spark Therapeutics.To obtain credit for this activity, please visit https://checkrare.com/learning-center/courses/

At least 17 cases of thrombosis and thrombocytopenia have been reported in patients who received the Johnson & Johnson COVID-19 vaccine in the United States. Such events have been reported in patients who received the AstraZeneca vaccine as well. In this episode, Adam C. Cuker, MD, of the University of Pennsylvania, Philadelphia, tells host David H. Henry, MD, how to identify and manage patients with these vaccine-induced events. What’s in a name? The phenomenon of vaccine-induced thrombosis and thrombocytopenia has been given different names, including: Vaccine-induced immune thrombotic thrombocytopenia (VITT) Vaccine-induced prothrombotic immune thrombocytopenia (VIPIT) Thrombosis and thrombocytopenia syndrome (TTS). Dr. Cuker’s preferred acronym is VITT. VITT is an immune-mediated reaction to the Johnson & Johnson and AstraZeneca vaccines that “results in thrombocytopenia and a strong propensity for thrombosis,” Dr. Cuker explained. Dr. Henry noted that VITT is reminiscent of heparin-induced thrombocytopenia (HIT). Incidence unclear VITT appears to be “very rare,” but “we still don't have a great sense of how common it is” because additional cases may not have been recognized or have yet to present, Dr. Cuker said. VITT occurs about 5-30 days after vaccination. VITT appears to be mediated by IgG antibodies, which take time to build up. The exact mechanism is unknown, but VITT could be related to the adenovirus vector used in the Johnson & Johnson and AstraZeneca vaccines, Dr. Cuker said. The first 15 cases of VITT associated with the Johnson & Johnson vaccine occurred in women, and most patients were aged under 50 years. In Canada, where the AstraZeneca vaccine is available, cases of VITT have been reported in patients in their 80s and 90s. Diagnosing VITT Symptoms of VITT can include severe, unrelenting headache; severe abdominal pain; nausea and vomiting; as well as typical signs and symptoms of deep vein thrombosis or pulmonary embolism. To determine if a patient has VITT, Dr. Cuker recommends ordering a disseminated intravascular coagulation panel – prothrombin time, partial thromboplastin time, fibrinogen, and D-dimer – as well as a standard HIT enzyme-linked immunosorbent assay (ELISA). Rapid immunoassays for HIT are not reliable for VITT, so HIT ELISA must be used, Dr. Cuker emphasized. Most patients with VITT have a “strongly positive” ELISA with optical density values “well in excess of 100 or 1.0,” depending on the scale, Dr. Cuker said. Manage VITT like HIT Patients should receive an anticoagulant, but not heparin, Dr. Cuker said. It isn’t clear if heparin will be harmful in patients with VITT, but current guidelines recommend avoiding heparin. He also advised against using warfarin or vitamin K antagonists in patients with VITT “at least until their platelet count recovers.” High-dose intravenous immunoglobulin (e.g., 1 g/kg for 2 consecutive days) is recommended, as it is believed to interfere with platelet activation. Show notes written by M. Alexander Otto, a reporter for MDedge and Medscape. Disclosures Dr. Henry has no relevant disclosures. Dr. Cuker has served as a consultant for Synergy Pharmaceuticals; has received authorship royalties from UpToDate; and his institution has received research support on his behalf from Alexion, Bayer, Novartis, Novo Nordisk, Pfizer, Sanofi, Spark Therapeutics, and Takeda. *  *  * For more MDedge Podcasts, go to mdedge.com/podcasts Email the show: podcasts@mdedge.com Interact with us on Twitter: @MDedgehemonc David Henry on Twitter: @davidhenrymd

World-renowned hematologist and scientist Dr. Katherine High has spent much of her career focused on achieving a goal few believed was possible: developing gene therapies and getting them to patients. Dr. High joins Madeline to talk about her journey from idea to breakthrough – and what it was like to create Spark Therapeutics, a company based on her innovations.

Join us this week as we chat with Kristin Smedley. A single mom of 3 kids, which 2 of them developed blindness. In 2011, Kristin launched a nonprofit to fund research and resources for children living with the rare eye disease her sons have, CRB1 LCA/RP. In just nine years, the Curing Retinal Blindness Foundation has raised over 1.4 million dollars and achieved a National Rare Eye Disease Awareness Day. That legislation, H.R. #625, was the first in US history to be submitted in Braille and it advocates for better resources for blind and visually impaired Americans. Kristin partnered with Spark Therapeutics to help achieve the first ever FDA approved gene therapy to treat an inherited retinal disease in the United States. She has done a TEDx Talk in New York City to change perceptions of blindness and she partnered with Comcast media to spread awareness of the inclusive X1 product. In 2019, Kristin published her first book called Thriving Blind: Stories of Real People Succeeding Without Sight. It achieved #1 New Release on Amazon for paperback and kindle. Kristin partnered with TMobile and the National Braille Press to make the book available in Braille. Kristin recently received one of the highest honors in the rare disease community: The Champion of Hope Award from Global Genes. During the COVID19 pandemic Kristin and a friend launched an online show/podcast called Brilliantly Resilient where they help their “tribe” Reset, Rise and Reveal their Brilliance after life's sucker punches. https://brilliantlyresilient.net/ Podcast Promos The Derek Duvall Show https://www.listennotes.com/podcasts/the-derek-duvall-show-derek-duvall-NPO48BHytWQ/ Podbreed https://www.podbreed.com/ www.krisandkristineshow.com Don't forget to SUBSCRIBE, rate and review, we love those five star reviews! Thanks for tuning in and until next time…keep moving forward! Twitter:https://twitter.com/k2showsandiego Instagram: https://www.instagram.com/thek2showsandiego/?hl=en Facebook: https://www.facebook.com/k2showsandiego Support us on Patreon at: www.patreon.com/thekrisandkristineshow Email us at: krisandkristinepodcast@gmail.com The Kris and Kristine Show Podcast is recorded in Audacity https://www.audacityteam.org/ Post-production editing is completed using Levelator which can be found at: http://www.conversationsnetwork.org/levelator Remote podcasts and interviews are completed using Cleanfeed which can be found at: https://cleanfeed.net/

Episode 005 | Kristy Lee (Genetic Counselor) joins the podcast to discuss genetic testing, counseling, gene therapy, and the results of Lance's own genetic tests. | Kristy's featured organization was Spark Therapeutics - a fully integrated, commercial company committed to discovering, developing and delivering gene therapies. Spark Therapeutics challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. To learn more, visit: sparktx.com | Need help finding a genetic counselor? Visit: www.nsgc.org | For more podcast content, visit: seethroughpod.com

To the surprise of many, on August 19th the US Food and Drug Administration denied the first application for a one-time gene therapy for hemophilia A. Patrick and Amy process this major news then share soundbites from their conversations with doctors Valentino, Quon, Young, and Wang during #NHF2020's Science Fair about gene therapy, emicizumab, and novel therapies. Presenting Sponsor: Takeda Response to FDA's Actions: HFA Statement The Science Fair is made possible by Spark Therapeutics 

One of the goals of the Human Genome Project was to improve therapeutic options for those born with serious inherited diseases. Today gene therapy is beginning to realize this promise, with the ever-increasing level of clinical investigation in the space, and the first products coming to licensing in the last few years. In this podcast, first recorded at the 2019 PODD: Partnership Opportunities in Drug Delivery conference, Dr Katherine High, Spark Therapeutics, reviews the current state of gene therapy, including the development of therapies for diseases that were previously untreatable, and, for diseases where therapeutics already existed, the development of therapies that greatly lessen the burden of treatment for the patient and the family. Hurdles in clinical development of gene therapy will be discussed. Examples will be drawn from already approved products and from investigational agents in late stage development. To learn more about the 10th annual PODD: Partnership Opportunities in Drug Delivery conference please visit www.theconferenceforum.org

Lewis talks with brothers Bradford and Bryan Manning co founders of Two Blind Brothers, who are on a mission to cure blindness. The funds from two blind brothers are driving life-changing treatments such as the “Voretigene Neparvovec” treatment, a gene therapy developed by Spark Therapeutics. 70% of their workforce are blind or visually-impaired in the factory in Dallas, Texas. At a young age, the brothers were both diagnosed with an eye disease that destroys central vision over time. In this episode they talk about how they are fighting back, leaving their former careers in finance to start a clothing company, Two Blind Brothers, which is one of the fastest growing charitable clothing brands in the country with endorsements from Ellen DeGeneres, Ashton Kutcher, Richard Branson, NBC Nightly News, and many others. *This episode was recorded on google hangouts. Show notes: Two Blind Brothers: https://twoblindbrothers.com/pages/about-us -------------------------------------------------------------------------------­----------------- This episode is supported by Audible: Start your 30 day free trail today! using the following link: bit.ly/2YVdZJU - Auto-renews at £7.99/month after 30 days - 1 book monthly membership - Cancel anytime Take your pick from the world’s largest selection of audiobooks including best sellers, latest releases, sci-fi, fantasy and more. Disclaimer: If you sign up using our link, we make a small percentage and you help to support our podcast for which we are truly grateful. This doesn't affect our opinions.

Dr. Daniel Chung is the global medical strategy lead for ophthalmology at Spark Therapeutics. Spark Therapeutics concentrates on discovering, developing, and delivering gene therapy for rare diseases. Dan works in the area of ophthalmology, and he and his colleagues brought the first FDA-approved gene therapy for a genetic disease to market. This therapy was created to treat an inherited retinal disease that results in blindness and is caused by variants or mutations in the RPE65 gene. When he isn’t working or traveling, Dan enjoys spending time with his family. He is also an avid photographer who loves capturing photos of nature, landscapes, and wildlife. In particular, he has really enjoyed photographing the panoramic landscapes of Monument Valley in Arizona, brown bears in Alaska, and polar bears in Northern Canada. Dan earned both his bachelor’s degree in biology and master’s degree in family counseling from Eastern Nazarene College in Massachusetts. He also holds a doctorate degree in Osteopathic Medicine (D.O.) from the New York College of Osteopathic Medicine. Afterward, Dan became a research training award fellow at the National Eye Institute of the National Institutes of Health, studying retinal gene therapy, and he went on to complete his residency in ophthalmology within the Summa Health System in Ohio. Dan joined the Cleveland Clinic as a pediatric ophthalmology clinical/ocular genetics research fellow and subsequently worked as a senior investigator at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania for eleven years before joining the team at Spark Therapeutics in 2014. In this interview, Dan shares more about his personal and professional passions, as well as his research.

At a young age, brothers Bradford and Bryan Manning were diagnosed with an eye disease that causes blindness over time. They’ve used their circumstances to help work toward a cure for blindness through the sales of their ultra-soft designer clothing line. Their vigilance for details carries over into the quality of their products, and you’ll love hearing the coincidental story of how the idea for their brand came about. Join me as I learn more about the inspiring Bradford and Bryan Manning. 3:08 - The Brad and Bryan 101 Brad and Bryan were diagnosed with Stargardt’s disease when they were each seven years old. Stargardt’s is a genetic disease that typically destroys center vision.  The idea for the Two Blind Brothers business started to form after Brad and Bryan shopped in the same store now knowing until after they’d left that they’d purchased the same shirt based on how it felt to the touch. Touch is a sense that’s critical to those with vision impairments. Brad and Bryan started talking about focusing on the sense of touch to develop their  clothing brand. One hundred percent of the proceeds from Two Blind Brothers is donated to organizations that fight diseases like Stargardt’s and other forms of vision impairment. 7:55 - It’s A Community  The goal was never a successful business, but a pure passion to help the community and raise funds for research. The brand was driven by people who wanted to support the cause, rather than just shop for an item of clothing. After learning that man with a retinal eye disease who’d never met another person with an eye disease found an online community through Two Blind Brothers, a huge paradigm shift occurred in the business. It became about fostering hope for the community that the brothers deeply care about. 15:42 - Shop Blind Holiday Brad and Bryan created a video on social media about how those who are vision-impaired rely on trust to get around in the world. During the holiday season, Two Blind Brothers then ran a campaign called “Shop Blind”, and pulled all the images from their website and products and added the question “Will You Shop Blind?” The Shop Blind experiment gave customers an option to pick from different price points, but they were not able to see what was being selected for them. Not only did the Shop Blind experiment raise money for retinal research, it also raised awareness to the experience of those who face obstacles due to vision impairment. Building trust builds your ability to connect and be social. When you can’t see, you automatically put yourself out there to become closer to others much faster. 38:41 - Getting To Know Our Guests You’ll think Brad and Bryan are even more impressive as you hear what they’d pick as their pump-up songs, the things no one would ever guess about them, and more. Of course, stay tuned to hear what it means to Brad and Bryan to run a business with purpose.    Memorable Moments “We started with the full intention that this would empower folks who have a vision impairment or relate to facing challenges and making a social impact.” “Everyone in the world has their issue. It doesn’t make you better.” “There really is no growth without friction. You’re never going to really unlock your potential, creativity, assertiveness, resourcefulness unless you’re put in a position to challenge those things.” Meet Your Guests, Co-Founders, Two Blind Brothers Bradford and Bryan Manning are curing blindness! At a young age, both brothers were affected with Stargardt disease, a form of macular degeneration that destroys central vision over time.  To fight back, they left their former careers in finance to start Two Blind Brothers. Within 1 year, they propelled the small charitable clothing company into one of the fastest growing brands in the country with endorsements from Ellen DeGeneres, Ashton Kutcher, Richard Branson, NBC Nightly News, and many others. Their luxury clothing project is focused on quality, comfort, and "sense of touch". The clothing is produced by a team of blind and visually-impaired workers in Dallas, Texas. Two Blind Brothers donates 100% of its profits to researchers and the Foundation Fighting Blindness to find cures for blindness. The funds from Two Blind Brothers are driving life-changing treatments that are already being used in patients such as “Voretigene Neparvovec”, a gene therapy developed by Spark Therapeutics. The brothers are evangelists for charitable and social enterprises. They were recently nominated to the Facebook Small Business Council for their use of social media to empower others and consult on branding, story-telling, corporate responsibility, and sustainable fashion. The brothers have also participated in various public speaking engagements, most notably two TEDx talks since founding the company. Originally from Charlottesville, Virginia, both brothers graduated from the University of Virginia. Bradford received a degree in finance and Bryan a degree in statistics. Prior to launching Two Blind Brothers in 2016, Bradford worked in investment management and Bryan worked in data sales. They currently live together in New York City.

In this podcast, Valerie Bowling, Director of PODD, walks you through what to expect at PODD 2019 in under 4 minutes. The 9th annual PODD conference features the following: Building a Company around a Transformative Drug Delivery Invention with MIT's Dr Robert Langer, Dr Sangeeta Bhati, Dr Giovanni Traverso and Harvard Medical School's Dr Jeffrey Karp.Modality Fluid Development: Tales of Evolving Pipelines and Drug Delivery Solutions with Merck's, VP, Pharmaceutical Sciences, Dr Allen TempletonGene Therapy Keynote with Spark Therapeutics, President and Head of R&D, Dr Katherine HighZeitgeist Presentation on Immune Targeting Nanomedicine by AstraZeneca’s, VP, External Innovation and Novel Product Technologies, Dr Anand SubramonyConference Chair opening remarks provided by AstraZeneca's, Dr Shawn Davis Additional Featured Speakers: Roche’s, Senior Portfolio Strategy Director, Dr Beate Bittner will moderate the Formulation & Device Lifecycle Management for Biotherapeutics – Clinical and Industry Perspectives on Subcutaneous Delivery panel.Drug Delivery Pipeline and Technologies, A Year in Review, with Pharmacircle’s, VP, Kurt SedoDelivering Flexible Dosage Forms with Pfizer's Dr Advait Badkar The 9th annual PODD conference will take place on October 7-8, 2019 in Boston, MA. Learn more at theconferenceforum.org

Dr. Katherine High is Emeritus Professor of Pediatrics at the Perelman School of Medicine of the University of Pennsylvania and president and head of research and development at Spark Therapeutics. Stephen Morrissey, the interviewer, is the Executive Managing Editor of the Journal. K.A. High and M.G. Roncarolo. Gene Therapy. N Engl J Med 2019;381:455-464.

Dr. Seenu Hariprasad from the University of Chicago joins the program to discuss corticosteroids for the treatment of retinal vascular disease including diabetic macular edema, among other topics. Dr. Sridhar has served as a consultant for Alcon Laboratories and Thrombogenics. Dr. Hariprasad is a consultant or serves on the speaker’s bureau for Clearside Biomedical, EyePoint Pharmaceuticals, Allergan, Alimera Sciences, Alcon Laboratories, OD-OS, Novartis, Spark Therapeutics, and Regeneron.

This week, the P4A team discuss the spate of mergers and acquisitions involving Big Pharma companies in the gene therapy space. Particularly, we looked at the deals behind Roche's acquisition of Spark Therapeutics and Biogen's buyout of Nightstar. Also, Vertex's continued struggle to get its cystic fibrosis drug Orkambi reimbursed in the UK. Presenter: Joanna Fernandes Contributor and Producer: Aparna Krishnan

Here's why Roche's acquisition of gene therapy pioneer Spark Therapeutics and Biogen's acquisition of gene therapy upstart Nightstar Therapeutics could put Regenxbio in play. RHHBY, ONCE, BIIB, NITE, RGNX Check out more of our content here: TMF's podcast portal YouTube Twitter Join Our Motley Fool Podcast Facebook Group LinkedIn StockUp, The Motley Fool's weekly email newsletter

Spark Therapeutics, Rite Aid, and Alnylam shares have tumbled following news, but that doesn’t necessarily make all three stocks worth buying. Stocks: ONCE, RAD, ALNY

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Christian Guardino is a 17-year-old singer who competed last year on America’s Got Talent and made it to the semifinals. Christian was diagnosed with Leber congenital amaurosis (LCA) as an infant, and his vision continually declined as he got older. At 13, he entered the RPE65 gene therapy clinical trial being conducted by the Children’s Hospital of Philadelphia and Spark Therapeutics. Since then, Christian has enjoyed a 75–80 percent improvement in his vision. Currently, Christian is serving as a Champion for Children’s Miracle Network Hospitals.   Listen to this episode and learn: What it was like growing up with limited eyesight How facebook helped Christian’s mother find the answer to a cure for his disease Christian’s advice for others going through a difficult time How Christian fulfilled his dream of performing in front of Simon Cowell. What is was like work with Grammy award winning songwriter Sacha Skarbvek and American Idol winner Jordin Sparks

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Novartis, Antares Vision, Syneos Health, Celgene, Impact Biomedicines, Pfizer, Roche, Techdow Pharma, Spark Therapeutics, Chiltern.Persone: Elisabeth Barrett (Novartis), Robert Kowalski (Novartis), Robert Landewé (Amsterdam Rheumatology Center).Nuove terapie: secukinumab, adalimumab, antidemetilasi & antideacetilasi, palbociclib, ocrelizumab, Inhixa, Luxturna.Patologie: Alzheimer, Parkinson, spondilite anchilosante, artrite psoriasica, cancro, tumore al seno, sclerosi multipla, trombosi, retinopatia ereditaria.Lavoro: CRA, Regulatory Affairs.Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/Hai un dispositivo Apple? Seguici e abbonati al podcast tramite la app iPod http://nelfarmaceutico.link/pharma-apple

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Novartis, Antares Vision, Syneos Health, Celgene, Impact Biomedicines, Pfizer, Roche, Techdow Pharma, Spark Therapeutics, Chiltern.Persone: Elisabeth Barrett (Novartis), Robert Kowalski (Novartis), Robert Landewé (Amsterdam Rheumatology Center).Nuove terapie: secukinumab, adalimumab, antidemetilasi & antideacetilasi, palbociclib, ocrelizumab, Inhixa, Luxturna.Patologie: Alzheimer, Parkinson, spondilite anchilosante, artrite psoriasica, cancro, tumore al seno, sclerosi multipla, trombosi, retinopatia ereditaria.Lavoro: CRA, Regulatory Affairs.Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/Hai un dispositivo Apple? Seguici e abbonati al podcast tramite la app iPod http://nelfarmaceutico.link/pharma-apple

Roger Longman, CEO of Real Endpoints, a firm assisting pharma companies with their pricing strategies, discusses the state of the value-based pricing market while lending some insights on what specialty drug companies like Spark can do to get payers on board with higher prices.

We conclude our biotech review-preview series with our final installment this week as we take a look at the year ahead as the industry readies for the JPMorgan Healthcare Conference. We spoke to Jon Gardner, U.S. News Editor for EP Vantage, about the EP Vantage 2018 Preview(http://bit.ly/2CT2RDP), the outlook for big-value drug launches, and whether dealmaking or clinical success will drive stock prices in 2018. As a note, this podcast was recorded prior to Spark Therapeutics announcement of its pricing for its gene therapy Luxturna.

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Celgene and bluebird bio report a stunning success in multiple myeloma, Juno Therapeutics takes it on the chin despite positive data, and Spark Therapeutics suffers in its showdown with BioMarin in hemophilia.

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Last month, Spark Therapeutics submitted its gene therapy Luxturna, an experimental treatment for a rare inherited disease that causes blindness, to regulators in the United States and Europe. The company also won U.S. Food and Drug Administration designation for the experimental therapy as a treatment for a rare pediatric disease. Luxturna could be the first treatment for inherited retinal disease and the first gene therapy for a genetic disease approved in the United States. We spoke to Paulo Falabella, medical affairs ophthalmic lead for Spark Therapeutics, about Luxturna, what clinical trials to date tell us, and why this represents a significant development for not just Spark, but the entire field of gene therapy.

In this episode, we take a look back at a historic summer that saw the National Hemophilia Foundation's (NHF) Annual Meeting and the World Federation of America's (WFH) World Congress convene in Orlando for the largest gathering ever of people with bleeding disorders. In addition to featuring some conference related content, our Like Share Comment segment highlight a story on Elijah Warren, a high school swimming star with hemophilia as well as a recent article from John Hopkins University on the power of humor in healthcare. Finally, we close this month's episode with a remembrance of Matt Stinger, a community member and advocate who passed unexpectedly this month at 33 years old. Headlines Novo Nordisk’s B-HERO-S study has found, to the surprise of some, that many patients with mild or moderate hemophilia B in the United States experience their condition negatively impacting their career, education, and recreational activities- something that can be overlooked in those affected by mild or moderate hemophilia as opposed to severe. Link. High schooler Federico Parres is one of five team members on the VEX Robotics World Championship Team F, responsible for creating Caerus-7, a ball-flinging robot. Federico lives with severe hemophilia A and plans on becoming an engineer one day. Link. Bayers’ Hemophilia Awards Program recently announced 16 physicians and researchers from 11 countries who will receives grant money totaling about two million dollars, including 6 individuals from the United States. This is the fourteenth year Bayer has run this program. Link A study published in the European journal Haematologica examines a new potential model for how to treatment patients with hemophilia A who undergo surgery. Something I personally find interesting given our ongoing need to learn more about aging with hemophilia. Link The FDA has approved a new needleless reconstitution device called Baxject III for Shire’s long-lasting factor FVIII therapy Adynovate. For those familiar, it is a very similar device to that which is found in Advate. Link Two medical advisory updates have been announced by the National Hemophilia Foundation regarding existing products: NHF Medical Advisory 419 (four-one-nine) states that Bayer has announced a voluntary recall of two lots of Kogenate FS for a loss of potency. The lot numbers are 270TN1C and 270R978. For more information or to arrange for an exchange please call the Recall Processing Center at 855-838-5782. Link And NHF Medical Advisory 420 (four-two-zero) states that CSL Behring has announced a voluntary recall of two lots of Helixate FS for a loss of potency. Those lot numbers are 270TN1G and 270R979. For more information or to arrange an exchange for these products, call CSL Behring’s Customer Service at 1800-683-1288. Link Product Related Headlines (not mentioned in the show) Genentech/Roche has released encouraging Phase 1/2 extension results for their investigational subcutaneous medicine, emicizumab-previously referred to as ACE910. Link The Netherlands-based uniQure, a company self-described as a leader in human gene therapy, announced encouraging clinical data from their ongoing Phase 1/2 trail of AMT-060, an investigational gene therapy for patients with hemophilia B. Link​ BioMarin has presented positive interim data from a Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for people severe hemophilia A. The company has announced intent to begin another study with an eye toward regulatory approval in both the US and Europe. Link CSL Behring’s hemophilia B therapy Idelvion shows promise after encouraging Phase 3 trial results. Link The company also posted positive results from a Phase III study from its FDA approved, hemophilia A treatment AFSTYLA, unique for being the first and only single-chain recombinant product available in the US. Spark Therapeutics and Pfizer received Breakthrough Therapy Status from the FDA for expedited development of a hemophilia B gene therapy treatment SPK-9001. The treatment is currently also undergoing a Phase 1/2 clinical trial. Link. Alnylam Pharmaceuticals announced positive results of a Phase 1 trial for their subcutaneous therapy fitusiran, designed to treat patients with both hemophilia A and B, with or without inhibitors. Link. Like Share Comment Segment NHF Daily, a digital recap of Annual Meeting. Link WFH The Congress Daily. Link High school swimming star Elijah Warren. Link John Hopkins University article on therapeutic humor. Link BloodStream on Social BloodStream Facebook Page BloodStream Twitter Account BloodStream Host Patrick James Lynch on Twitter and Instagram.

Jeffrey D. Marrazzo, Co-Founder and CEO of Spark Therapeutics, discusses the successful Phase III trial of its lead candidate, SPK-RPE65, was able to improve functional vision in patients with a rare form of a genetic disorder known as RPE65-mediated inherited retinal dystrophies. SPK-RPE65 is intended to treat rare blinding conditions caused by mutations in the RPE65 gene, which is linked to subtypes of Leber congenital amaurosis (LCA type 2) and retinitis pigmentosa (RP type 20). Wall St. approved as well--Spark shares rose almost 60% to $70 in trading before the market opened the day of the announcement. The study results represent the first successful randomized, controlled Phase 3 trial ever completed in gene therapy for a genetic disease, Spark executives said.

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