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Join us for this two-episode mini-series featuring lead study authors, Mario Castro and Njira Lugogo, as they discuss key findings from the VESTIGE trial and their implications on asthma care. Uncover: · The importance of patient phenotyping: How can biomarkers and imaging improve asthma management? · Mucus plugging and airflow obstruction: What does the latest research reveal? · Biologics and airway remodeling: What did the VESTIGE trial reveal about biologics and airway remodeling? · The role of imaging in clinical practice: How can CT scans provide new insights into asthma care? Speakers Mario Castro, University of Kansas School of Medicine, United States Njira Lugogo, University of Michigan, Ann Arbor, Michigan, United States Disclaimers: · This program is non-promotional and is sponsored by Sanofi and Regeneron Pharmaceuticals, Inc. · The speakers are being compensated and/or receiving an honorarium from Sanofi and Regeneron in connection with this program · The content contained in this program was jointly developed by AMJ, the speakers, and Sanofi and Regeneron, and is not eligible for continuing medical education (CME) credits · See full US Prescribing Information for dupilumab · MAT-US-2412937 v2.0 - Pro1 Expiration Date: 04/21/2026 Speaker disclosures: · MC reports research support from the American Lung Association, AstraZeneca, Gala Therapeutics, Genentech, GSK, NIH, Novartis, PCORI, Pulmatrix, sanofi-aventis, Shionogi, and Theravance Biopharma, consultancy fees from Allakos, Amgen, Arrowhead Pharmaceuticals, Blueprint Medicines, Connect BioPharma, Genentech, GSK, Merck, Novartis, OM Pharma, Pfizer, Pioneering Medicines, sanofi-aventis, Teva, Third Rock Ventures, and Verona Pharmaceuticals, speaker fees from Amgen, AstraZeneca, Regeneron Pharmaceuticals Inc., and Sanofi, and royalties from Aer Therapeutics. · NLL reports research support paid to institution from Amgen, AstraZeneca, Avillion, Genentech, Gossamer Bio, GSK, Regeneron Pharmaceuticals Inc., Sanofi, and Teva, consultancy fees from and participation on advisory boards with Amgen, AstraZeneca, Genentech, GSK, Novartis, Regeneron Pharmaceuticals Inc., Sanofi, and Teva, travel support from AstraZeneca, and honoraria for non-speaker bureau presentations from AstraZeneca and GSK. References: 1. Castro M et al. Effect of dupilumab on exhaled nitric oxide, mucus plugs, and functional respiratory imaging in patients with type 2 asthma (VESTIGE): a randomised, double-blind, placebo-controlled, phase 4 trial. Lancet Respir Med. 2025;13:208-20. doi: 10.1016/S2213-2600(24)00362-X.
Join us for this two-episode mini-series featuring lead study authors, Mario Castro and Njira Lugogo, as they discuss key findings from the VESTIGE trial and their implications on asthma care. Uncover: · The importance of patient phenotyping: How can biomarkers and imaging improve asthma management? · Mucus plugging and airflow obstruction: What does the latest research reveal? · Biologics and airway remodeling: What did the VESTIGE trial reveal about biologics and airway remodeling? · The role of imaging in clinical practice: How can CT scans provide new insights into asthma care? Speakers Mario Castro, University of Kansas School of Medicine, United States Njira Lugogo, University of Michigan, Ann Arbor, Michigan, United States Disclaimers: · This program is non-promotional and is sponsored by Sanofi and Regeneron Pharmaceuticals, Inc. · The speakers are being compensated and/or receiving an honorarium from Sanofi and Regeneron in connection with this program · The content contained in this program was jointly developed by AMJ, the speakers, and Sanofi and Regeneron, and is not eligible for continuing medical education (CME) credits · See full US Prescribing Information for dupilumab · MAT-US-2412937 v2.0 - Pro1 Expiration Date: 04/21/2026 Speaker disclosures: · MC reports research support from the American Lung Association, AstraZeneca, Gala Therapeutics, Genentech, GSK, NIH, Novartis, PCORI, Pulmatrix, sanofi-aventis, Shionogi, and Theravance Biopharma, consultancy fees from Allakos, Amgen, Arrowhead Pharmaceuticals, Blueprint Medicines, Connect BioPharma, Genentech, GSK, Merck, Novartis, OM Pharma, Pfizer, Pioneering Medicines, sanofi-aventis, Teva, Third Rock Ventures, and Verona Pharmaceuticals, speaker fees from Amgen, AstraZeneca, Regeneron Pharmaceuticals Inc., and Sanofi, and royalties from Aer Therapeutics. · NLL reports research support paid to institution from Amgen, AstraZeneca, Avillion, Genentech, Gossamer Bio, GSK, Regeneron Pharmaceuticals Inc., Sanofi, and Teva, consultancy fees from and participation on advisory boards with Amgen, AstraZeneca, Genentech, GSK, Novartis, Regeneron Pharmaceuticals Inc., Sanofi, and Teva, travel support from AstraZeneca, and honoraria for non-speaker bureau presentations from AstraZeneca and GSK. References: 1. Castro M et al. Effect of dupilumab on exhaled nitric oxide, mucus plugs, and functional respiratory imaging in patients with type 2 asthma (VESTIGE): a randomised, double-blind, placebo-controlled, phase 4 trial. Lancet Respir Med. 2025;13:208-20. doi: 10.1016/S2213-2600(24)00362-X.
Richard Gaster, M.D., Ph.D., is a Managing Partner at venBio with experience as a physician, entrepreneur, and life science investor. Now, a cancer dad. He has helped to launch, invest, and serve on the board of or as board observer of a number of venBio companies. Prior to joining venBio, Dr. Gaster served as the head of translational medicine at Pliant Therapeutics. As a key member of the founding management team, Dr. Gaster was instrumental in the formation and launch of Pliant Therapeutics from Third Rock Ventures where he previously served as a Senior Associate.Dr. Gaster has published numerous articles in top-tier peer-reviewed journals including Nature Medicine and Nature Nanotechnology, holds more than a dozen patents, was awarded first prize in the IEEE Change the World Competition, and was named one of Forbes “30 Under 30” in Science and Healthcare.Dr. Gaster holds a B.S.E in Bioengineering from the University of Pennsylvania where he graduated summa cum laude and was a University Scholar. He received his M.D. and Ph.D. in Bioengineering from Stanford University in the Medical Scientist Training Program.On March 1, 2024, my daughter, Claire experienced a seizure. She was diagnosed with a type of brain cancer called AT/RT (atypical teratoid rhabdoid tumor), a highly aggressive grade IV glioma..... tune in for the rest of the story!You can also get involved with the Gold Ribbon Kids Cancer Foundation or the National Pediatric Cancer Foundation through fundraising, volunteering, promoting awareness, or contributing to pediatric cancer research. Visit goldribbon-kids.org or nationalpcf.org for more informationTo contact Tiffany, please email info@goldribbon-kids.orgTo contact Kelly, please email kgoddard@nationalpcf.orgSupport the show
It Happened To Me: A Rare Disease and Medical Challenges Podcast
A physician-scientist father shares his heartbreaking story of the death of his daughter who was diagnosed with Tay-Sachs disease and how it motivated him to become the CEO of JScreen to prevent this experience in other families. Matt Goldstein is a physician-scientist and entrepreneur. He has founded companies, built R&D teams, and led strategy and execution of both pre-clinical research and clinical development. Prior to joining JScreen and Emory University, Matt was a Partner at Related Sciences, a venture creation firm. As an entrepreneur at Third Rock Ventures he spent a decade building and operating Third Rock portfolio companies. He was responsible for building and leading the Immunology program at Tango Therapeutics, the centerpiece of Tango's strategic multi-billion dollar partnership with Gilead Sciences, Inc. He also served as the development head for Tango's lead program which entered the clinic in 1H 2022. Matt was a co-founder of Neon Therapeutics leading Translational Medicine and Early Development through completion of their first clinical study and initial public offering. He is a graduate of Swarthmore College and the MD/PhD program at Stanford University, where he pioneered novel cancer immunotherapies in the lab of Ron Levy, MD. He completed his clinical training in Internal Medicine at Harvard Medical School, Brigham & Women's Hospital. He lives in Boston with his wife, Myra, their second daughter Kaia and son Ezra. His oldest daughter Havi died on January 20th, 2021 of Tay-Sachs disease. A quick update that during the episode Matthew mentioned there are 4,000 genetic counselors in the USA, this number has now surpassed 5,000. During the episode, Matthew recommends the book Bearing the Unbearable: Love, Loss, and the Heartbreaking Path of Grief by Dr. Joanne Cacciatore. Check out his wife, Myra's organization, Emotion, which is for grieving individuals to find community and cope with loss. In our next episode we will chat with Myra about Emotion and her upcoming book, Fifty-Seven Fridays, which consists of memoirs from Matt and Myra, Havi's diagnosis, and how they celebrated her life. Stay tuned for the next new episode of It Happened To Me! In the meantime, you can listen to our previous episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “It Happened To Me”. “It Happened To Me” is created and hosted by Cathy Gildenhorn and Beth Glassman. DNA Today's Kira Dineen is our executive producer and marketing lead. Amanda Andreoli is our associate producer. Ashlyn Enokian is our graphic designer. See what else we are up to on Twitter, Instagram, Facebook, YouTube and our website, ItHappenedToMePod.com. Questions/inquiries can be sent to ItHappenedToMePod@gmail.com.
Reid Huber, partner at Third Rock Ventures, on investing in the future of medicine.
Synopsis: Bobak Azamian, MD, Ph.D., is the CEO and Chairman of Tarsus Pharmaceuticals, a biopharmaceutical company that applies proven science and new technology to revolutionize treatment for patients, starting with eye care. Bobby talks about his early years working in the venture capital space before founding his first company. He shares what it's like running a publicly traded biotech for the first time and the evolution of his role over the years at Tarsus. He shares his viewpoint on unmet needs and the importance of having a true focus on what's really happening to the patient. He talks about future indications they're pursuing at the company and how he leverages learnings from the first programs they ran to what's coming up next. Finally, he discusses the company's decision to commercialize in-house versus with a partner and the challenges of taking the company public during the pandemic in 2020. Biography: Bobby cofounded Tarsus Pharmaceuticals, Inc. with Michael Ackermann in November 2016 and has served as its President and CEO since September 2018. He has served as the company's Chairman since December 2022. Since its founding, Tarsus has established a leading team and culture and has completed successful fundraising efforts through multiple private and public financings and global partnerships. Tarsus recently secured FDA approval for its first product, XDEMVY™ (lotilaner ophthalmic solution) 0.25%, which is the first and only FDA-approved treatment in the United States for the eyelid disease Demodex blepharitis. Beyond XDEMVY, Tarsus continues to advance its pipeline into Phase 2 clinical studies evaluating therapies that have the potential to create new categories to treat other major diseases, including meibomian gland disease, rosacea, and Lyme disease prevention. Bobby has cofounded and led other novel therapeutics companies, including Vibrato Medical and Metavention, and currently serves on the board of Vibrato Medical and as Chairman of the board of Osanni Bio. Bobby began his biotechnology career at Versant Ventures and Third Rock Ventures, where he was an investor and board observer for a variety of life sciences companies. He also served on the board of the nonprofit organization Octane. Bobby trained as a physician scientist, completing his residency in internal medicine and serving as an attending hospitalist at Brigham and Women's Hospital. He received an MD from Harvard Medical School (Harvard-MIT Health Sciences and Technology), a PhD in chemistry from Oxford University as a Rhodes Scholar, and a BA in physics from Rice University. He is also an author of numerous patents and peer-reviewed publications. Bobby enjoys sports, travel, arts and culture, and spending time in Newport Beach with his newborn son, wife, and dog.
Today's guest is Aaron Arvey, Director of Machine Learning at Third Rock Ventures. Founded in 2007, Third Rock Ventures push the boundaries of science and technology to make a difference in the lives of patients and their families. By starting with big ideas and fostering collaboration among brilliant people with expertise in science, medicine, business and strategy, Third Rock set out to do more than fund startups, they aim to build sustainable, innovative companies that can transform the lives of patients. Aaron's role at Third Rock is to advance therapeutic and diagnostic R&D with focused hypothesis-driven machine learning. Aaron initially trained as a computer scientist and mathematician, working at the intersection of machine learning theory and algorithm implementation. However, it wasn't until he happened upon molecular biology and the cellular microcosm that he found his passion. Since then, he has been in perpetual awe of the never-ending stream of high-impact scientific opportunities in biotech. In the episode, Aaron discusses: The mission at Third Rock Ventures, His role and responsibilities as the Director of ML, Use cases of the value his team brings to their portfolio companies, Trends that excite him in Biotechnology, Career opportunities with Third Rock Ventures
View the Show Notes Page for This Episode Become a Member to Receive Exclusive Content Sign Up to Receive Peter's Weekly Newsletter Ethan Weiss is a preventative cardiologist at UCSF, an entrepreneur-in-residence at Third Rock Ventures, where he is working on a project related to cardiometabolic disease, and a previous guest on The Drive. In this episode, Ethan compares and contrasts the diagnostic imaging tools, CAC (coronary artery calcium score) and CTA (CT angiography), used to image plaque—including the latest in CTA software—and how these tools inform our understanding of ASCVD risk and guide clinical decision-making. Ethan discusses the types of plaque that cause events and the data that make a case for optimal medical therapy over stenting outside of particular cases. He explains why high blood pressure is problematic and walks through the data from clinical trials testing aggressive treatment. He talks about the best way to actually measure blood pressure, why we shouldn't simply accept that blood pressure rises with age, and how he uses different pharmaceutical agents to treat hypertension. Additionally, Ethan explains our current, but limited, understanding of the role of metabolic health in ASCVD. He discusses the impact of fat storage capacity and the location of fat storage and explains how and why there is still a residual risk, even in people who have seemingly normal lipids, don't smoke, and have normal blood pressure. We discuss: Ethan's entrepreneurial work in the cardiometabolic disease space [4:30]; Calcium scans (CAC scores) and CT angiography (CTA), and how it informs us about ASCVD risk [6:00]; Peter's historical CAC scores, CTA results, and how one can be misled [10:45]; How Peter's CTA results prompted him to lower his apoB [14:45]; Calcium scans vs. CT angiogram (CTA) [21:15]; How Ethan makes clinical decisions based on CTA results and plaque burden, and the importance of starting treatment early to prevent ASCVD [28:15]; Improved methods of CTA to grade plaque lesions and how it's shaped medical decisions such as stenting [33:45]; Why Ethan favors optimal medical therapy over stenting outside of particular situations [41:45]; The need for FFR CTA, and the potential for medical therapy to eliminate ASCVD [54:00]; The fat attenuation index (FAI) and other ways to measure inflammation in a plaque [57:30]; Statins and exercise may increase the risk of calcification, but what does this mean for risk? [59:45]; The root cause of statin hesitation despite evidence that statins are a profoundly important intervention [1:05:30]; Importance of keeping blood pressure in check, defining what's normal, and whether we should just accept higher blood pressure with age [1:10:45]; Blood pressure variability, how to best measure it, and data suggesting the enormous impact of keeping blood pressure down [1:21:00]; Drugs for treating high blood pressure recommended by the ALLHAT trial [1:35:15]; What the SPRINT trial says about the aggressive treatment of hypertension, and the risks of such treatment [1:38:15]; Confirmatory results in the STEP trial for blood pressure, and how Ethan uses the various pharmacological agents to lower blood pressure in patients [1:43:15]; The role metabolic health in ASCVD: what we do and don't know [1:51:00]; The impact of fat storage capacity and the location of fat storage on metabolic health and coronary artery disease [1:56:15]; and More. Connect With Peter on Twitter, Instagram, Facebook and YouTube
Recorded on 12/16/22 Cowen analyst Yaron Werber speaks with Abbie Celniker, partner at Third Rock Ventures, and Nina Kjellson, partner at Canaan, about their outlook for company formation and financing in FY23, how companies are adjusting to the new market realities, and their experiences as women leaders in biotech venture capital. While there has been great progress towards gender equality in the industry, a lot of work remains to be done. For Disclosures, click here bit.ly/3cPHkNW
Top startups news to follow this week: 1. Startups keep laying off swaths of employees as the downturn continues - TechCrunch 2. With biotech in retreat, Third Rock Ventures raises $1B for life sciences investing 3. Raleigh diagnostics startup Gemelli Biotech closes $19M Series A – investors include Carolina Angel Network 4. Zomato acquires Blinkit for $568 million in instant-grocery delivery push 5. AI-Powered Hong Kong Biotech Startup Raises $60 Million From Top VCs Including B Capital, Qiming - Forbes 6. Security-as-code startup JIT comes out of stealth with $38.5M in seed funding 7. Amsterdam cyber startup Hadrian closes €10.5M Seed for a platform that simulates hacker attacks, TechCrunch reports 8. Cybersecurity startup RevealSecurity raises $23M for global expansion 9. EuraTechnologies raises €24 million to back deep tech startups and open 10 incubators in Eastern Europe 10. London-based Stotles secures €6.1 million to streamline how businesses and governments work together Read more https://hyetechminds.com/ Sign up for Startup Monday Newsletter subscribepage.com/n5x5l8 --- Send in a voice message: https://podcasters.spotify.com/pod/show/hyetechminds/message
David Kaufman is a Partner @ Third Rock Ventures.Before joining TRV, he was Chief Medical Officer and head of translational development at the Bill and Melinda Gates Medical Research Institute (MRI). He helped establish the Gates MRI as a "nonprofit biotech," developing novel drugs and vaccines for tuberculosis, malaria, enteric diseases, neonatal health, and other global health challenges. Prior to that, Kaufman led translational oncology at Merck Research Laboratories, where he oversaw immuno-oncology translational medicine, novel biomarker development and global translational research partnering. He joined Merck through the Merck Drug Development and Leadership Program, and worked across vaccines, infectious diseases and clinical oncology. Before joining Merck, he was a physician in the Division of Infectious Diseases and the Center for Virology and Vaccine Research at Beth Israel Deaconess Medical Center and an instructor in medicine at Harvard Medical School. Kaufman received his M.D. from Cornell University School of Medicine and his Ph.D. in immunology and molecular virology from The Rockefeller University. He trained in internal medicine at the University of California, San Francisco, and in infectious diseases at Massachusetts General Hospital and Brigham and Women's Hospital. He is a member of the board of directors of Hookipa Pharma and a director ex officio of the Society for the Immunotherapy of Cancer (SITC).Thank you for listening!BIOS (@BIOS_Community) unites a community of Life Science innovators dedicated to driving patient impact. Alix Ventures (@AlixVentures) is a San Francisco based venture capital firm supporting early stage Life Science startups engineering biology to create radical advances in human health.Music: Danger Storm by Kevin MacLeod (link & license)
Laurence joined Decibel in January 2020. He previously served as an entrepreneur in residence at Third Rock Ventures, where he focused on novel drug discovery and development opportunities. Before that, Laurence was CEO of Warp Drive Bio, a small molecule company focused on novel oncology and antibiotic drug discovery based on natural products, until its merger with Revolution Medicines in 2018. Prior to that, he was the senior vice president and chief business officer of Alnylam Pharmaceuticals, where he was responsible for the company's business development, finance and legal functions. Before coming to Alnylam, Laurence was the chief business officer at Ensemble Discovery, where he led the company's strategic planning and corporate development efforts. Laurence previously spent ten years at Millennium Pharmaceuticals in a range of general management and business development positions. In addition to his professional interests, Laurence is a board member of the Possible Project and board advisor to Life Science Cares. He earned his Ph.D. from King's College London and his B.A. from Cambridge University.
Dr. Agnieszka Czechowicz is driven by her passion for making an impact and changing the way that care is provided. Throughout her career she has taken science and translated it into medicines, many of which are having helping patients. Agnieszka has worked with a wide range of companies, from those that do gene editing to those that focus on hearing and balance disorders. However, her main area of research is in the field of hematopoietic stem cells. In today's episode, Agnieszka explains the importance of these cells, and the benefits of the hematopoietic stem cells transplants. Agnieszka's interest in transplant medicine began at a young age, and her passion for the field continues to grow year after year. Tune in today to hear about how Agnieska is contributing to the transformation of the medical field! “We showed that your own stem cells compete for space with the transplanted stem cells in a hematopoietic stem cell transplantation process.” — @aneeshka Key Points from this Episode An overview of Dr. Agnieszka Czechowicz's incredible career. Where Agnieszka's interest in transplant medicine originated. What keeps Agnieszka motivated, despite the challenges of being a physician scientist. The power of hematopoietic stem cells. An explanation of the two bone marrow failure syndromes that Agnieszka specializes in. Discoveries that Agnieszka made many years ago when conducting research into hematopoietic stem cell transplantation. Problems with the genotoxic conditioning that is used prior to bone marrow and hematopoietic stem cell transplants. The mission of Magenta Therapeutics, of which Agnieszka is a co-founder. Feedback on progress that Magenta Therapeutics is making. The potentially transformative power of agent CD117 ADC. How agents developed by Magenta Therapeutics can be used in gene editing and gene therapy. Agnieszka's involvement with Jasper Therapeutics, and the therapy that they are currently working on. Interventions that Agnieszka believes will cause a profound reduction in graft versus host diseases. Work that Agnieszka has done for Third Rock Ventures. Some of bluebird bio's achievements and setbacks. Agnieszka's involvement with Editas, Decibel Therapeutics, and Beam Therapeutics (and where the name Beam came from). Ex vivo versus in vivo manipulation techniques. What drives Agnieskza to do the work that she does. Agnieszka's passion for women's health.
Is it important for MDs to consider taking a more prominent role in the process of developing and getting drugs to market, either on the operating side or the investing side? How can we change and fix financial incentives in drug development? In this episode, we answer all these questions and more with our guest, David Kaufman, M.D.David is a Partner at Third Rock Ventures, a leading healthcare venture firm that specializes in company creation rather than the traditional venture investing model.David has an MD from Cornell University, and a PHD in immunology and molecular virology from Rockefeller University.
Did you know that hearing loss is one of the largest areas of unmet need in medicine? It affects approximately 466 million people worldwide, including 48 million people in the United States. Today's guest Laurence Reid, CEO of Decibel Therapeutics dives deeper into meeting that unmet need. Decibel has architected a whole platform to restore hearing and balance loss. With several treatments in their pipeline now, one in phase 1 clinical trials, they're making great progress. Learn more about how Decibel started, current pipeline, and the future of biotech industry. About Laurence Reid, Ph.D.Laurence joined Decibel in January 2020. He previously served as an entrepreneur in residence at Third Rock Ventures, where he focused on novel drug discovery and development opportunities. Before that, Laurence was CEO of Warp Drive Bio, a small molecule company focused on novel oncology and antibiotic drug discovery based on natural products, until its merger with Revolution Medicines in 2018. Prior to that, he was the senior vice president and chief business officer of Alnylam Pharmaceuticals, where he was responsible for the company's business development, finance and legal functions. Before coming to Alnylam, Laurence was the chief business officer at Ensemble Discovery, where he led the company's strategic planning and corporate development efforts. Laurence previously spent ten years at Millennium Pharmaceuticals in a range of general management and business development positions. In addition to his professional interests, Laurence is a board member of the Possible Project, a board member of Garuda Therapeutics and board advisor to Life Science Cares. He earned his Ph.D. from King's College London and his B.A. from Cambridge University.About DecibelDespite the overwhelming burden of hearing loss and balance disorders, the search for therapeutics to treat these conditions remains one of the largest areas of unmet needs in medicine. At Decibel, we are exclusively focused on discovering and developing transformative treatments to restore and improve hearing and balance. We have assembled a highly experienced scientific team and built a unique platform to achieve this goal.Show notesDecibel website: https://decibeltx.com/Pictures of the inner ear: https://www.decibeltx.com/our-approach/QualioPrevious episodes: https://www.qualio.com/from-lab-to-launch-podcastApply to be on the show: https://forms.gle/uUH2YtCFxJHrVGeL8Music by keldez
Dr. Greg Verdine is an eminent serial entrepreneur and investor in the life sciences industry. He has founded a number of companies and currently serves as President, CEO, and CSO of both Fog Pharma and LifeMine Therapeutics. Greg also worked in the venture capital industry as a Venture Partner with Apple Tree Partners, Third Rock Ventures, WuXi Healthcare Ventures, and Texas Pacific Group. Prior to his career in the biotech industry, Greg had an academic career that spanned 3 decades and he was named the Erving Professor of Chemistry at Harvard University. He has made significant contributions to the study of epigenetics and DNA damage repair and has more recently pioneered a new therapeutic modality termed “stapled peptides” that are poised to expand the space of druggable targets. He received his Ph.D. in Chemistry from Columbia University.In this episode, we discuss the development of new drug modalities, the transition from academic science to entrepreneurship and investing, and the changing biotechnology landscape.
Episode 49. Richard Ransohoff is a Venture Partner at Third Rock Ventures and interim Chief Medical Officer at Abata Therapeutics. Richard is a physician-scientist. He completed his undergraduate degree at Bard College and his MD at Case Western Reserve University.
Dr. Cristoph Lengauer Ph.D MBA is a Partner at Third Rock Ventures, an innovative venture capital firm focused on company creation, and supporting entrepreneurs from idea to discovery to the clinic. In this episode, Dr. Lengauer discusses Third Rock's unique approach to venture funding, common sources of company ideation, and his path from the Carey School of Business to the boardroom. Hosted by Farai Sikipa.
This episode will discuss what it takes to build companies that develop novel therapies, vaccines, and diagnostics in the pharma industry.Since the pandemic, everybody got familiar with the key elements of pharma product development.But let’s take a closer look into the value chain of the industry.What does it really take to pick up a technology and bring it to patients?Questions we will discuss:Some questions we will focus on:What strategic approaches do exist for life science companies?How important is the team in developing a company to an IPO?Where can scientists find help when they want to start a company?About Christoph Lengauer (from Third Rock Ventures)“The most powerful thing about being a part of this industry is having the opportunity to improve the lives of patients and their families.”Christoph loves the ideation of new company concepts and focuses on the formation, development, and strategy of our portfolio companies. He is an accomplished scientist and drug hunter with the ability to translate that knowledge, insight, and expertise into compelling business strategies. Christoph has contributed to the discovery and development of eight FDA-approved medicines.Christoph is dedicated to making a dramatic difference in patients’ lives. He is enthusiastic about Third Rock’s approach to discover, launch and build great companies by focusing on bold ideas that meet at the intersection of science, strategy, business, and medicine. He is especially excited by initiatives at the firm focusing on the early detection of diseases in order to not only treat but cure patients.By leveraging his experiences and networks from academia, pharma, and biotech, he hopes to continue building great teams that help young companies succeed with this important mission.Over his career, Christoph has received numerous awards, including the Novartis Oncology’s President’s Award for Top Innovator, and has been elected to membership in the Johns Hopkins Society of Scholars.About Third Rock VenturesWe discover, launch, and build great companies based on bold ideas that meet at the intersection of science, strategy, business, and medicine – where transformational science meets operational reality – providing the best opportunity to make a dramatic difference in patients’ lives.A FORMULA FOR INNOVATIONWe only invest in the best of those ideas. We are actively involved in the early stages of our companies’ development, typically serving as members of the founding management teams, to build a strong foundation, put in place the right people and culture, and set the companies on a path to scientific and operational excellence. We believe in the companies we launch and make big initial investments, often complemented by strategic alliances, to enable our companies to advance life-changing therapies, devices, and diagnostics. By creating value for patients, we create value for our investors.Link to the Video of the Recording:YouTubeSpeaker:Christoph LengauerChristian SoschnerOrganizations:CS Life Science InvestThird Rock VenturesSupport the show (https://lsg2g.substack.com/subscribe?)
Our guest for today's podcast is Lorence Kim, Venture Partner, Third Rock Ventures and Former CFO of Moderna. If there was ever someone you wanted to picture as having a perfect life, that's Lorence. Lorence graduated magna cum laude from Harvard, then went on to earn a dual degree (Doctor of Medicine and MBA) from Penn. Upon graduation, he joined Goldman Sachs and rose through the ranks becoming a Managing Director and co-head of the Biotechnology Investment Banking division. In 2014, Lorence pivoted and joined a little known biotechnology firm called Moderna. The rest is history!!! What I admire about Lorence, in addition to his awesome professional career and accomplishments, is that deep down inside, he is a humble guy and a loving father and husband. I look forward to all the amazing things he will continue to do in the industry, Without further ado, here is our conversation with Lorence Kim.
A “very basic” type of gene therapy could potentially cure hemophilia, but a major hurdle has been the lack of an effective mode of delivery. Recent strides in using adeno-associated virus (AAV) vectors are changing that, and Glenn Pierce, MD, World Federation of Hemophilia Vice President, Medical, predicts approvals in the next 12-18 months. Dr. Pierce shared his personal experience with hemophilia and discussed his and others’ ongoing research on the use of AAV-mediated gene therapy with host David Henry, MD, in this episode. Hemophilia and AAV gene therapy key points: Hemophilia is caused by a monogenic defect and could, theoretically, be cured by gene replacement or augmentation, says Dr. Pierce, who notes that “it sounds disarmingly simple, but behind that simplicity is a very complex procedure.” The approach uses “gene addition,” which is a basic gene therapy involving the addition of a normal gene to the variant in an individual. This ultimately corrects the clotting factor deficiency. The complexity is in getting the normal gene into the body where it can have the intended therapeutic effect. After more than 20 years of working to overcome that barrier, Dr. Pierce and others are finding success with using AAVs. The approach has some similarities to that used in developing the mRNA COVID-19 vaccines but requires the use of DNA established within the virus (rather than mRNA) to provide a more stable effect. Questions about how long it will last are currently being investigated. Multiple phase 3 trials are underway or completed. Data from two of those have been released in recent months, and the results are very encouraging: “It’s a remarkable achievement – many patients are doing well and, for all intents and purposes, could be considered free of [hemophilia],” Dr. Pierce says, adding that he would “potentially … use the ‘C word’ – cured – for at least a period of time.” The therapy is generally well tolerated. Efforts are ongoing to identify the best ways to proactively and reactively use prednisone to manage side effects such as mild increases in transaminase levels. To date, the risk-benefit profile appears reasonable for patients with clotting factor IX deficiency, and it is likely that the therapy in that setting “will march toward the regulatory process to determine if it’s safe and effective for approval,” he said. Responses in those with clotting factor VIII deficiency have been more variable, with some patients requiring long-term prednisone use, which is problematic. More information is needed about this, but investigation is ongoing, he said. Registries are available and many companies are involved in clinical trials. Clinicians and patients can look for information at clinicaltrials.gov, wfh.org (which publishes trial results and conducts workshops and meetings), and at the US National Hemophilia Foundation (Hemophilia.org) and the Society of Thrombosis and Hemostasis (ISTH.org). Show notes written by Sharon Worcester, MA, a reporter for MDedge and Medscape. Disclosures Dr. Henry has no relevant disclosures. Dr. Pierce disclosed relationships with Ambys Medicines, BioMarin, BridgeBio, CRISPR Therapeutics, Decibel Therapeutics, Frontera, Geneception, Generation Bio, Novo Nordisk, Pfizer, Regeneron, Third Rock Ventures, Voyager Therapeutics, Global Blood Therapeutics, VarmX SAB, the National Hemophilia Foundation Medical and Scientific Advisory Council, and the World Federation of Hemophilia. * * * 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
Thank you for tuning into my podcast, “Snack Sized Sales,” where I focus on sharing three actionable tips on one topic that you can use to transform your sales. Today, my guest is Jeff Behrens, a serial biotech entrepreneur with experience in general management, business development, finance, and operations. Jeff will highlight the one thing that all successful people have in common and tell us why listening more than talking can help us thrive in sales. He is currently the CEO of GelMEDIX, an ophthalmology startup based on work from Mass Eye and Ear and UCLA. He is also the Founder and CEO of LabShares Newton, a biotech incubator for biotech startups run by biotech entrepreneurs. Formerly, Jeff was President and CEO of Siamab Therapeutics, a biotech company focused on developing antibodies targeting glycan targets in cancer that he exited in 2019. Previously, Jeff served as Senior Director, Business Development and Operations at Edimer Pharmaceuticals (funded by Third Rock Ventures) and also worked at Alnylam and Biogen Idec, where co-founded Biogen's Innovation Incubator. In 2003, Jeff sold his healthcare IT company, The Telluride Group, to mindSHIFT Technologies, a Fidelity-funded rollup. Jeff has a Ph.D. from EPFL (Lausanne, Switzerland), an MS from the Harvard/MIT Division of Health Sciences and Technology (HST), an MBA from MIT Sloan, and graduated from Harvard College. He teaches HST590, a Ph.D. level course at MIT, and lives in Newton and Wellfleet, MA. This is one episode you won't wanna miss so stay tuned. On Today's Episode of Snack Sized Sales: Being in business and healthcare simultaneously (02:24) Success by being in a sales mode most of the time (03:32) A necessary mind shift needed to succeed as a salesperson (05:22) The multi-step sales process of raising funding for a biotech business (06:40) Listening and asking relevant questions versus lecturing (07:00) Building a sales team around getting a product out to market (10:20) One thing that's missing in a lot of sales organizations today (12:47) Connect with Jeff Behrens: https://www.linkedin.com/in/jbehrens/ (Jeff on LinkedIn) Email Jeff at Jeff@LabShares.com Rate, Review, Learn, and Share Thanks for tuning into the Snack Sized Sales podcast! If you enjoyed this episode and want to learn even more about what it takes to simplify and transform your company's back-office and online customer journey. Don't forget to tune into our other shows and share your favorite episodes on social media! Join the community of Snack Sized Sales fans onhttps://www.facebook.com/WesleyneGreerCEO ( Facebook), https://twitter.com/WesleyneGreer (Twitter),https://www.linkedin.com/in/wesleynegreer/ ( LinkedIn) and https://www.transformedsales.com/ (visit my website )for even more content, information, and resources about leveraging the media.
Today I discuss with Vyas Ramanan, of Third Rock Ventures and Maze Therapeutics, the evolution of the field's understanding of genetic modifiers and the role they play in disease, how drug discovery works when attempting to recapitulate protective effects of certain mutations, and how the convergence of certain advances in statistics and genomic sequencing and perturbation tools enable pressure testing of hypotheses at an unprecedented scale. Check out the glossary of terms, definitions, and resources (and get a sneak peak of the future conversations lined up!) here: bit.ly/datapulse-glossary --- Support this podcast: https://anchor.fm/the-data-pulse/support
Alexis Borisy is CEO and Chairman of EQRx, and a long-time biotech builder with Third Rock Ventures. His conversation with Nature Biotechnology covers growing up as a “faculty brat” in Wisconsin, dropping out of his PhD program at Harvard and why, even as an investor, biotech cannot be all about the money. See acast.com/privacy for privacy and opt-out information.
The small, Chicago-based biotech venture Errant Gene Therapeutics, which was engaged in a court battle against a well-funded biotech called Bluebird Bio and a venture capital firm known as Third Rock Ventures. With allegations of breach of contract, tortious business interference, and fraud, TrialSite News initiated a project to review publicly available information, including court records, to derive a more thorough understanding of the case at hand. First podcast is located here
John Keilty is the Chief Data Sciences Officer at Decibel Therapeutics and a Strategic advisor at Third Rock Ventures. He is a biologist and bioinformatics guy by training and has been active in the biotech space for over two decades. In his career, John has been responsible for building out genomix, and genetics capabilities for early stage biotech companies. In this episode of Biotech 2050, John goes through his journey into the biotech industry and the work he’s doing at Decibel Therapeutics and Third Rock Ventures.
The small, Chicago-based biotech venture Errant Gene Therapeutics, LLC was engaged in a court battle against a well-funded biotech called Bluebird Bio (which has raised over $400 million) and a venture capital firm known as Third Rock Ventures. With allegations of breach of contract, tortious business interference, and fraud, TrialSite News initiated a project to review publicly available information, including court records, to derive a more thorough understanding of the case at hand. In this podcast we ask Mr. Girondi about how this all started.
Patient-led collaborations are transforming drug discovery and development in rare disease. How they are doing this will be the topic of a panel discussion at the 2019 Biotech Showcase in San Francisco January 8 at 4:30 p.m. Ahead of that panel, we spoke to Walt Kowtoniuk, principal at Third Rock Ventures and moderator of the panel to discuss the challenges of rare disease drug discovery and development, how patient-led collaborations are addressing these challenges, and how patient involvement can change the risk profile of drug development to attract drug company partners and investors. For information on the panel or to register, go to www.globalgenes.org/rareinthesquare
Christoph Lengauer is a venture partner at Third Rock Ventures, the president of Celsius Therapeutics, and executive vice president at Blueprint Medicines. His conversation with Nature Biotechnology covers growing up in Austria, why he nearly gave up research for a career helping settle refugees, and his role in the Henrietta Lacks story. See acast.com/privacy for privacy and opt-out information.
Abbie Celniker of Third Rock Ventures on her career, and chipping away at gender inequities in biotech.
Fulcrum Therapeutics is working to develop small molecule drugs to modulate gene activity as a way to treat certain rare diseases. The company, established by the venture capital firm Third Rock Ventures, is initially focusing on Fragile X and a form of muscular dystrophy known as FSHD. We spoke to Walt Kowtoniuk, director of strategy and operations for Fulcrum, about its approach, what he learned working in a venture capital firm, and what advice he would offer rare disease patients turned investors.
Ophthalmology CEO turned VC Abbie Celniker, now a partner at Third Rock Ventures, discusses her role at the firm and reveals how it will invest its new $616 million fund.
Greg Verdine has evolved from merely being a Harvard prof, to consulting for big pharma, to starting biotech companies, to being a Venture Partner, and even to co-founding a non-profit institute – not just sequentially, but largely in parallel. He has teamed up with Third Rock Ventures, TPG Biotech, AppleTree Partners, and he unveils his brand new gig with WuXi Ventures. Greg co-founded the biotechs Enanta (ENTA), Tokai (TKAI), Gloucester (now Celgene), Aileron, Warp Drive, WaVe (WVE), and Fog. His day job today, if forced to list just one, is CSO of Warp Drive Bio. He reveals a couple of secrets about Warp Drive strategy and lifts the fog off of Fog Pharma, his latest – and still stealth – newco. As different as these companies are, a common theme runs through them: to cure the incurable by drugging the undruggable. With apologies to Star Trek, that is Greg’s personal mission: to boldly go where no drug has gone before. In fact Greg wants to blow up the conventional meaning for the word "drug". He sees beyond the pharma industry’s small molecules plus the biotech industry’s biologics, because they seem positively “puny” in the context of human biology. And he has clever tricks and other modalities at his disposal – not to mention the blueprints: 135,000 bacterial genomes, where the answers cannot hide.
In “Orphan: The Quest to Save Children with Rare Genetic Disorders,” Philip Reilly, a clinical geneticist turned venture capitalist, recounts the history of developing therapies to treat rare diseases. We spoke to Reilly, a venture partner with Third Rock Ventures, about his own experiences in the clinic treating patients with rare diseases, how his perspective has changed as a venture investor, and what the future holds in the battle against rare diseases.