Podcasts about luxturna

In this latest episode of the BroadEye podcast, hosts Shawn Maloney and Dr. Bruno Fernandes sit down with Dr. Arshad M. Khanani, MD, MA, FASRS, a pioneer in vitreoretinal gene-therapy trials and Managing Partner at Sierra Eye Associates. Dr. Khanani shares front-line insights into how genetic medicines are reshaping treatment for inherited and degenerative retinal diseases, highlighting both the promise and the practical challenges of bringing one-time therapies to patients.   Why Gene Therapy Matters Today Recent years have seen the first US approvals of in-vivo retinal gene therapies, beginning with voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophy in December 2017. These one-off treatments offer durable vision improvements where none existed before, but they also raise questions of long-term safety, vector delivery, and equitable access.   Dr. Khanani's Journey With over a decade leading surgical trials in sustained-delivery and viral-vector therapies, Dr. Khanani has overseen early human work on next-generation agents—including subretinal injections of AAV vectors and novel capsid designs. He describes lessons learned from Phase 1 safety studies through global registrational trials, emphasizing iterative “vector evolution” to boost efficacy and minimize inflammation.   Key Takeaways Vector Optimization: Directed-evolution platforms are accelerating discovery of capsids that target photoreceptors more precisely, reducing off-target toxicity. Durability vs. Dose: Striking the right balance between transgene expression and immune activation remains central—higher doses don't always equal better outcomes. Regulatory Pathways: While Luxturna set a precedent, streamlined FDA and EMA guidance on trial endpoints and real-world evidence will shape the next wave of approvals. Cost & Access: Innovative payment models (e.g., outcomes-based installment plans) are emerging to address the multi-hundred-thousand-dollar price tags of these therapies.   About the Guest Dr. Arshad M. Khanani, MD, MA, FASRS, is a vitreoretinal surgeon, researcher, and Managing Partner at Sierra Eye Associates in Reno, Nevada. He holds dual degrees in medicine and medical humanities from the University of Nevada, Reno, and is a fellow of the American Society of Retina Specialists. Dr. Khanani has authored over 100 peer-reviewed articles on retinal disease and served as principal investigator on more than 30 clinical trials of gene and cell therapies for inherited and age-related retinal disorders. He also lectures internationally on surgical innovation, vector design, and the economics of advanced ocular treatments.

March 7, 2025. Aaron Nagiel, MD, PhD, a retina surgeon at Children's Hospital Los Angeles, talks to host Ben Shaberman about how gene therapy is administered to the retina, what the surgery and recovery is like for patients, and the vision improvements for young patients receiving LUXTURNA, the first FDA-approved gene therapy for the eye or an inherited condition. Dr. Nagiel also discusses other retinal gene therapies in clinical development.

In this episode of Molecule to Market, you'll go inside the outsourcing space of the global drug development sector with Imran Kausar, Founder of DeepHealth Consulting. Your host, Raman Sehgal, discusses the pharmaceutical and biotechnology supply chain with Imran, covering: His unusual blend of experience led to a road into big pharma... Switching the security of Big Pharma to join an exciting, scrappy, start-up biotech... before an exit to Roche Being part of the gene therapies evolution with an innovative biotech that later became Novartis Gene Therapies His views on the thorny issue of CGT products' price (and value) Opting to start his own business aimed at supporting the future of healthcare Imran Kausar is a visionary healthcare and life sciences leader, blending medical expertise with strategic foresight. A medical doctor by training, Imran specialized in anesthesia and critical care before transitioning to the pharmaceutical industry. Having launched five rare disease medicines and spearheaded innovative work in gene therapies like Zolgensma and Luxturna across Europe, Imran has earned his reputation as a trailblazer in the industry. As the founder of DeepHealth Consulting, Imran leads the flagship program DeepHealth Futures, which equips life sciences teams to navigate the transformative forces of AI, longevity science, and precision medicine.With nearly two decades of experience in medicine, medical affairs, and general management, Imran's career spans the cutting edge of rare diseases, antisense oligonucleotides, and healthcare transformation.    Please subscribe, tell your industry colleagues and join us in celebrating and promoting the value and importance of the global life science outsourcing space. We'd also appreciate a positive rating! Molecule to Market is also sponsored and funded by ramarketing, an international marketing, design, digital and content agency helping companies differentiate, get noticed and grow in life sciences.

Jason Comander, Md, PhD, clinical researcher, surgeon, and director of the Inherited Retinal Disease Disorders Service at Mass Eye and Ear, talks to host Ben Shaberman about his administration of LUXTURNA gene therapy to RPE65 patients, his team's re-analysis of vitamin A therapy for retinitis pigmentosa, and passion for retinal research and patient care.

Viren können ihre eigene Erbinformation in unseren Zellkern einschleusen, damit unsere Zellen den Virus vermehren. Wenn man deshalb einen Schnupfen bekommt, ist das lästig. Aber was, wenn man sich diese Eigenschaft der Viren auch zu Nutze machen kann? Können Viren verwendet werden, um Gene in Zellen zu bringen, damit man so genetische Erkrankungen heilen kann? Genau das ist das Konzept der Gentherapie. Mehr dazu erfahren Sie in dieser Podcastfolge von unserem Gast Dr. Immanuel Seitz.

Una semana más, Eva Belmonte nos lleva en su nave de los misterios para mostrarnos la verdad oculta tras el precio de los medicamentos. Hablamos del Luxturna o el Zolgensma, que puede llegar a costar más de un millón de euros por dosis.

In the first hour of NOW with Dave Brown: Canadian Tire has established the Women's Sport Initiative to improve gender equity in sport and Ashley Curran is here to explain more (15:19). Adam and Jenna Brown will share their experience as two of the first patients in the province to receive Luxturna gene therapy (24:01), and Community reporter Amy Amantea spent the last weekend traveling to the US. She has some thoughts on the accessibility of the trip (38:08).

In the first hour of NOW with Dave Brown: Dr. Larissa Moniz discusses Ontario's funding for ‘Luxturna' gene therapy (17:04). Meagan Gillmore returns with another accessibility report on the Canada Disability Benefit (27:08). And Dorothy Macnaughton explains how you can go about advocating for accessible pedestrian signals in your community (46:20).

In this podcast, Mark Penessi is joined by Isabelle Audo and Bart Leroy to discuss the impact of Luxturna and how gene therapy products will affect the treatment of inherited retinal diseases (IRD) in the future.  

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.

President and CEO of Fighting Blindness Canada, Doug Earl, shares important updates on the access to Luxturna Gene Therapy in Canada.

Ur innehållet: Första svenska ögonpatienten har fått genterapin Luxturna. Malmös färdtjänstbokning låg nere i fredagsrusningen. Många glaukompatienter missnöjda med ögonsjukvården. Hjälpmedelskunnig musiker från Lund är Månadens ansikte.

Thank you to all those who completed our recent Sight Loss Survey – almost 700 of you took the time to share your experiences. We carried out our first Sight Loss Survey in 2019 and used what we learned to improve our support. This included the launch of our Unlock Genetics and Discover Wellbeing resources. We also shared information with the decision-makers who agreed to fund Luxturna, the first treatment for inherited sight loss, on the NHS. This helped them to understand the day-to-day impacts of inherited sight loss. This spring we repeated the Survey, to learn what has changed since 2019 and shape our future plans. Here are some of the key findings.

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).

In this roundtable moderated by Allen C. Ho, MD, IRD experts discuss their experiences treating patients with voretigene neparvovec (Luxturna, Spark), tips and tricks for subretinal delivery, and the nuances of genetic testing. Participants include Mark E. Pennesi, MD, PhD; Jacque L. Duncan, MD; Andreas Lauer, MD; Aaron Nagiel, MD, PhD; and Artur V. Cideciyan, PhD. To read the article, click here. 

Vi fikk bli med da Torger Sætre var på kontroll etter å ha fått den banebrytende genterapibehandlingen Luxturna. Hjemmekontor med May Nina og Aage. Dagens aviser.

Ur innehållet: “En fantastisk nyhet och ett genombrott.“ Luxturna blir den första genterapin inom svensk ögonsjukvård. Kul att tävla igen – och medaljer till längdhopparna Viktoria Karlsson och Tobias Jonsson i EM i parafriidrott. Inte så kul att tävla igen – Sveriges herrlandslag i goalboll förlorade alla matcher i B-EM och åker ner en division till. Han träffar ju bollen varenda gång! Dövblinde Torbjörn Svensson överraskar sina blinda golfkamrater på Kullaberg.

Latest Retina UK Podcast features Luxturna treatment Retina UK Annual Conference The Big Give Christmas Challenge Charles Bonnet Syndrome Patient Information Event Local groups Christmas Raffle Tech tip Sight Village news 10 for 10 Virtual Challenge

Retina UK Research Development Manager Kate Arkell speaks to three of the first people to receive the Luxturna (voretigene neparvovec) treatment in the UK. Jake, Lee and Matt all received the Luxturna gene therapy at the start of 2020. They all have two faulty copies of the RPE65 gene and are living with Leber congenital amaurosis type 2 (LCA2) and severe early-onset RP.

Lederen af Sverigesdemokraterne Jimmie Åkesson besøger Danmark i dag. I Sverige diskuteres problemer med bandekriminalitet i mange byer, og i den forbindelse har det vakt opsigt i Sverige, at statsminister Stefan Löfven fra Socialdemokratiet i Riksdagen har anerkendt, at indvandringen til Sverige har negative konsekvenser i form af mere kriminalitet. VI lytter med på Åkessons besøg, og får en analyse af situationen i Sverige fra DR Nyheders Europakorrespondent, Anna Gaarslev. Et andet besøg, vi følger i dag, er Hviderusland præsident Lukajenkos besøg hos Putin i Rusland. I går var flere end 100.000 demonstranter på gaden i den hviderussiske hovedstad Minsk i protest mod Lukajenko. Vi spørger om Rusland bliver ved med at støtte ham? Gen-terapien, Luxturna, der kan kurere den sjælden øjensygdom Lebers syndrom, er kun begyndelsen på en stor bølge af gen-terapibehandlinger, der kan kurere sygdomme, vi tidligere ikke har kunne gøre meget ved. Men det bliver dyrt og har vi råd? Det taler vi med Anne-Marie Gerdes om. Hun er klinikchef på Rigshospitalets Klinisk Genetiske Klinik og professor ved Københavns Universitet, og så er hun også formand for etisk råd. Karin Axelsson og Louise Windfeld-Høeberg er dagens værter. Birgitte gadegaard er redaktør. www.dr.dk/orientering

Jimmy and Roya discuss what's in your genes, and the importance of DNA.Sources:On Purpose with Jay Shetty: https://jayshetty.me/podcast/Eye Tracking Remote: https://www.wired.com/story/comcast-eye-tracking-tv-remote/?itm_campaign=TechinTwoRPE65 gene therapy LUXTURNA: https://luxturnahcp.com/id-appropriate-patients/rpe65-gene/

We dip into the gene therapy pool one more time, talking with Jay Duker, MD about Luxturna’s impact on ophthalmology. We also discuss how the acquisitions of Spark and Nightstar are benefiting earlier-stage gene therapy start-ups, including his own Hemera Biosciences.

Together, Roche and Biogen paid over $5.5 billion for gene therapy companies Spark and Nightstar. What does this mean for the emerging technology? Spark, of course, received FDA approval for Luxturna as a treatment for inherited retinal disease. But the drug isn’t Roche’s primary focus. Meanwhile, Nightstar’s clinical-stage products could give Biogen a second shot at a gene therapy portfolio after the company severed ties with AGTC in December. Could Biogen make a move to bulk up its ophthalmology offerings?

This episodes focuses on a recent publication (https://www.nature.com/articles/s41591-018-0295-0) in Nature Medicine concerning the successful use of an intravitreal oligonucleotide for a form of Leber Congential Amaurosis (LCA). First, Dr. Allen Ho, one of the authors of the paper, rejoins the program to discuss the project and its implications. Then Dr. Matthew Weed returns to offer his perspective as a ophthalmic genetics specialist on this trial compared to other modalities such as Luxturna and what it means for the future. Dr Weed and Dr. Sridhar have no relevant financial disclosures. Dr. Ho has receieved grant funding from Pro QR.

Terapia genética chega a Oftalmologia porém com um preço ainda pouco acessível.

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

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.

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.