Lexeo Therapeutics’ Post

The recent failure of Pfizer's late-stage trial for Duchenne muscular dystrophy (DMD) gene therapy marks a significant setback for patients but opens the door for competitors like Sarepta Therapeutics. Pfizer’s gene therapy did not improve motor function in young boys and follows a similar setback for Sarepta’s Elevidys. Despite this, Sarepta remains optimistic, citing positive secondary outcomes and ongoing FDA support. As the biotech landscape evolves, Symphony Benefits Solution is positioned to be a steadfast resource in the gene and cell therapy space, offering the latest insights and support for advancements in these critical therapies. Stay informed with Symphony Benefits Solution for all your gene therapy updates and needs. https://lnkd.in/gr-fpZM4

Retinal gene therapy development news from Market Scope: SalioGen Therapeutics announced April 17 that it had chosen a development candidate, SGT-1001, for the treatment of #Stargardt disease. SGT-1001 is being developed as a one-time, non-viral gene therapy to slow or stop the progressive loss of central vision in people with Stargardt, regardless of the type of ABCA4 gene mutation. Jason Cole, chief executive officer of SalioGen, said the company plans to complete preclinical studies for SGT-1001 in the second half of 2024 and file an investigational new drug (IND) application in the first half of 2025. #retina #inherited #genetherapy #drugdevelopment

Did you know that out of the current 3,905 gene therapies in development in 2023, only 60 made it to clinical trials by Q3? To achieve the FDA's ambitious goal of 10-20 new cell and gene therapy approvals a year by 2025, developers and manufacturers must overcome a wide range of challenges to bring their life-changing treatments to patients.  In our latest piece with Pharma Focus America, our experts Kai Lipinski, Xiaojun Liu and Jing Zhu, explore these challenges further and discuss the solutions to help overcome them. 

Potency Assurance for Cellular and Gene Therapy Products FDA Draft Guidance. On December 28, 2023, FDA announced the availability of the draft guidance, Potency Assurance for Cellular and Gene Therapy Products. This draft guidance provides recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product. A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect. For a high-level overview of this guidance document, please view the recorded webinar at: https://lnkd.in/gDwZcrPq This webinar features Dr. Matthew Klinker, Cell Therapy Branch 2 Chief, Office of Cellular Therapy and Human Tissues, Office of Therapeutic Products, CBER. #celltherapy #cellandgenetherapy #celltherapymanufacturing #genetherapy #gmp #fda #fdacompliance #fdaregulations

Retina gene therapy clinical trial news from Market Scope: 4D Molecular Therapeutics reported Feb. 3 that it had seen positive topline results from the Phase II PRISM trial evaluating 4D-150, an intravitreal gene therapy candidate, in wet age-related macular degeneration (AMD) patients with severe disease activity and a high treatment burden. At 24 weeks, in the high-dose group, 84 percent of patients received none or one supplemental #aflibercept injection—representing an 89 percent reduction in the annualized anti-VEGF injection rate. Visual acuity and central subfield thickness were both stable vs. aflibercept. #retina #clinicaltrial #genetherapy

It’s difficult to navigate the balance phase-appropriate needs with commercial readiness during cell and gene therapy development. Partnering with a trusted and experienced viral vector #CDMO can help unlock unique insights and provide guidance to achieve the right balance for your therapeutic. Here are 4 recommendations to keep in mind during preclinical and clinical development:

It’s difficult to navigate the balance phase-appropriate needs with commercial readiness during cell and gene therapy development. Partnering with a trusted and experienced viral vector #CDMO can help unlock unique insights and provide guidance to achieve the right balance for your therapeutic. Here are 4 recommendations to keep in mind during preclinical and clinical development:

Finally, CBER/OTP has released its draft guidance on Potency assurance for gene and cell therapy products. I find this guidance uniquely helpful for the industry as it contextualizes potency assurance beyond just an assay. Per the FDA, this guidance seeks to provide recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product. This is a much welcome news! https://lnkd.in/e-cvVEiC

With the FDA expecting to approve 10–20 Cell and Gene Therapies (CGTs) annually by 2025, it is evident that the field will continue to hold immense commercial and medical opportunities. To harness this potential, stakeholders across the CGT ecosystems must collaborate to overcome barriers to delivering life-changing treatment to patients.   Delivered through an interactive virtual platform, this event features over three hours of insightful sessions led by 6 speakers. The discussions will provide industry perspectives on essential success factors for developing CGTs, covering topics such as: 1. Large scale processing of mesenchymal stem cells (MSC) and T cell immunotherapies 2. Integrated, cutting-edge solutions and process advancements that supports end-to-end workflow 3. Regulatory considerations in raw material selection from research through commercialization 4. Analytical platforms to evaluate the quality, safety, and efficacy of cell-based products #freewebinar #cellgenetherapy https://2ly.link/1wu5O

It’s difficult to navigate the balance phase-appropriate needs with commercial readiness during cell and gene therapy development. Partnering with a trusted and experienced viral vector #CDMO can help unlock unique insights and provide guidance to achieve the right balance for your therapeutic. Here are 4 recommendations to keep in mind during preclinical and clinical development in our recent article