Mike Hyungkyu Park’s Post

This should be interesting.

Gene therapy is currently far from perfect, and one of the main concerns remains the safety of using these treatments. Patients who have undergone gene therapy often face autoimmune reactions, toxicity, and other adverse effects. I think the open FDA webinar, which you can register for, will provide a lot of new information and allow for discussion of key safety issues in gene therapy. I'm going!

#GeneTherapy research gives hope to many patients living with serious diseases and conditions. But #clinicaltrials in this field are often held back by high costs and study complexity. Learn how Veristat reduced their trial database costs by 30%, thanks to efficient gene therapy patient data management support from Zelta. https://lnkd.in/gYUAw4ky

ICYMI: CGTLive took a look for World Hemophilia Day at how the #hemophilia field has adapted to the introduction of gene therapy to the treatment landscape. https://lnkd.in/e3pfrkS2

Using cell and gene therapy for a specific condition involves a complex assessment of the treatment's benefit-risk ratio. In this Cell & Gene article, Tome’s Chief Scientific Officer, Jonathan (John) Finn, discusses how real-world examples, evolving science, and long-term risks all influence the decision to use cell and gene therapy. Read more here: https://bit.ly/44QldTe #cellandgenetherapy

Expanded access plays an important role in the success of cell and gene therapy. Read our white paper which explores go-to-market models for success. https://lnkd.in/esNjw3if #ExpandedAccess #Gotomarket #MarketStrategy #PharmaMarketStrategy

Expanded access plays an important role in the success of cell and gene therapy. Read our white paper which explores go-to-market models for success. https://lnkd.in/esNjw3if #ExpandedAccess #Gotomarket #MarketStrategy #PharmaMarketStrategy

Registration closing in an hour - FDA's public patient listening session to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases. Listening session is tomorrow, September 20th: https://lnkd.in/e8F4Driq. #FDA #GeneTherapy #SafetyConsiderations #RareDiseases #PatientPerspectives

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Lexeo Therapeutics' gene therapy shows potential for Friedreich's ataxia cardiomyopathy with early data: Lexeo Therapeutics thinks its gene therapy has the potential for accelerated approval following positive interim data from two early-stage studies, the company announced Monday. Lexeo is developing the therapy, dubbed LX2006, for Friedreich’s ataxia cardiomyopathy, a fatal disorder that currently has no treatment. The disorder is caused by a loss of function mutations in the frataxin gene, and cardiac dysfunction is the cause of death in up to 80% of patients with the disease. Across two trials — a Phase 1/2 trial dubbed SUNRISE-FA and a Phase 1a investigated-initiated study conducted at Weill Cornell Medicine — patients saw improvements in cardiac biomarkers that increased over time. That includes hypertrophy, a hallmark of FA cardiomyopathy, according to Lexeo development chief Sandi See Tai. Sandi See Tai “Together with the increases in frataxin protein expression observed in SUNRISE-FA cardiac biopsies to date, these results further highlight the potential of LX2006 to positively impact outcomes for people with FA cardiomyopathy,” Tai said. The interim data come from just eight patients who had at least six months of follow-up. Of the four patients with an elevated left ventricular mass index (LVMI) at baseline, 75% saw a 10% reduction at one year. Out of all patients evaluated at 12 months, 50% achieved a 10% reduction in LVMI. Elevated LVMI can be a predictor of sudden cardiac death. The company reported improvements in other biomarkers at 12 months: Six patients saw their left ventricle wall thickening reduced by 13.6% on average, while a biomarker of myocardial injury called troponin I was reduced by 53.3% on average among five patients. Lexeo CMO Eric Adler said in a statement that the company is looking into “expedited” clinical development of the gene therapy as well as the potential for accelerated approval. While Biogen, through its acquisition of Reata Pharmaceuticals, markets a treatment for Friedreich’s ataxia, it treats the neurological manifestations rather than cardiological ones. Other companies are also working on a gene therapy in FA cardiomyopathy: Astellas is hoping to dose the first patient in the second half of this year for its gene therapy AT808, which it’s developing in two different programs to target two different aspects of FA cardiomyopathy. #lucidquest #genetherapy #celltherapy

New on the Blog - As a follow-up to our previous Gene Therapy blog article, we explore the challenges and regulatory developments of this medical advancement. BLS invites you to read "Gene Therapy; Challenges and Regulatory Developments." #genetherapy https://ow.ly/59Bt50RC4jr