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Krystal is the second company to have received the new FDA designation.

The RDEA program is meant to support use of novel end points for measuring efficacy in clinical studies for therapies aimed at treating rare diseases.

The FDA has set the PDUFA action date for the BLA at March 28, 2026.

Pending talks with the FDA, Regeneron intends to submit a regulatory application for DB-OTO before the end of 2025.

With regard to safety, MYR-101 was characterized as “well tolerated” with a “favorable safety profile.”

Review top news and interview highlights from the week ending October 10, 2025.

Verismo emphasized that the milestone strengthens its relationship with the Institute for Follicular Lymphoma Innovation.

Neurogene also relayed new preclinical findings on the use of intracerebroventricular and intrathecal lumbar delivery of NGN-401.

AVB-114 previously received fast track designation from the FDA in February of this year.

The PDUFA target action date for the BLA has been set at April 6, 2026.

The company cited strategic prioritization as the reason for its decision.

Taysha also announced that the FDA has aligned with the company on the pivotal trial protocol and statistical analysis plan.

Review top news and interview highlights from the week ending October 3, 2025.

UB-VV111 is currently being evaluated in a phase 1 clinical trial for adult patients with R/R CD19+ B-cell malignancies.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive® team.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

The documents cover topics including efficient trial design, expedited review programs, and postapproval data collection.

In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news related to cell therapy for such indications.

In observance of World Heart Day, held annually on September 29, we took a look back at news in cell/gene therapy for cardiovascular disease from the past few months.

MVX-220 is intended to provide a functional copy of UBE3A, the disease-targeted gene, to the neurons.

Review top news and interview highlights from the week ending September 26, 2025.

SAR446268 has previously received orphan drug designations from the FDA and European Medicines Agency.

The study also met a key secondary end point.

Jainu Jogani, the cofounder of Child’s Cure Genetic Research, discussed his daughter’s rare genetic disease and the need for new treatment options.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Part B of LIGHTHOUSE enrolled 9 patients and is expected to enroll 3 pediatric patients.

In honor of Duchenne Action Month and World Duchenne Day, held every year in September, CGTLive is taking a closer look at this ongoing study.