Gene Therapy

FDA CBER Patient Listening Meeting on Gene Therapy Safety: Patient and Care Partner Perspectives

This FDA CBER patient listening meeting captures patient and care partner perspectives on safety considerations for approved gene therapy treatments for rare diseases. It also explores expectations for long-term follow-up and how people want to receive risk information.

$2M Gene Therapy Cures Need a Financing Model

Gene therapies can carry six- and seven-figure prices, with some reaching about $2 million for a single intervention. This plan centers on Zolgensma, sickle cell gene therapy pricing, and the financing gap behind one-time cures.

Gene Therapy FDA Approval for Rare Disease: Updated List and Analysis

Only about 5% of rare diseases have an FDA-approved drug, but gene therapies are gaining ground. This analysis covers the latest FDA approved gene therapy list, including the recent approval of Kresladi for LAD-I, and what it means for pharma teams and investors.

Exclusive: FDA issues industry-friendly gene therapy guidance: What BD teams need to know

The FDA issued draft guidance to help developers bring gene therapies to market more efficiently. This article explains the changes, their impact on pharma strategy, and what to watch next.

FDA Issues June 2026 Guidance: Gene Therapy, Payor Comms, and CMC Flexibilities

The FDA released eight new guidance documents between April and June 2026, including draft guidances on gene therapy genome editing and payor communications, plus final guidances on pregnancy safety studies and CMC flexibilities for cell and gene therapies. These updates signal the agency's evolving regulatory approach and create both opportunities and compliance requirements for pharmaceutical companies.

FDA seeks to cut repeat work for gene therapy developers

Structured plan for FDA seeks to cut repeat work for gene therapy developers

Tenaya Therapeutics to Present TN-201 Gene Therapy Data for HCM on June 3, 2026

Tenaya Therapeutics is set to release interim data from its TN-201 gene therapy trial for hypertrophic cardiomyopathy (HCM) on June 3, 2026. This data from the MyPEAK-1 study will offer insights into the potential of TN-201, an investigational AAV9 gene therapy targeting the MYBPC3 gene.

FDA Guidance on Next-Gen Sequencing for Gene Therapy Safety

The FDA has released draft guidance on using next-generation sequencing to evaluate gene therapy safety, impacting regulatory approaches and investment strategies.