CGTLive’s Weekly Rewind – October 21, 2022

Welcome to CGTLive’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. CAR T Therapy to be Evaluated in Lupus Nephritis

KYV-101 was developed using the construct Kyverna licensed from the NIH in January 2022.

2. Improving Access to Rare Disease Care

Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access.

3. T-Cell Therapy Recognized for Treating Pediatric Hepatoblastoma

The therapy is currently being evaluated in the phase 1/2 ARYA-1, ARYA-2, and ARYA-3 studies in adults and children.

4. Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health Care

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

5. Otoferlin-Related Hearing Loss Gene Therapy Gets IND Clearance

In a rodent model, durable auditory brainstem responses to sound were observed after DB-OTO administration.