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

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.

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

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

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

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

FDA Activity Recap: September 2025 Features New Draft Guidance Documents, RMAT Designation, and More
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

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.

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.

Review top news and interview highlights from the week ending September 19, 2025.

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

SAR402663 comprises a onetime intravitreal treatment and is intended to inhibit VEGF by providing a gene for soluble FLT01.

Review top news and interview highlights from the week ending September 12, 2025.

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

Review top news and interview highlights from the week ending September 5, 2025.

Notably, the European Medicines Agency also granted MCO-010 orphan drug designation for 5 retinal dystrophy categories.

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.

Review top news and interview highlights from the week ending August 29, 2025.

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

Review top news and interview highlights from the week ending August 22, 2025.

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


























































