Around the Helix: Cell and Gene Therapy Company Updates – March 13, 2024

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. ODAC Meeting to Discuss Ide-Cel for Earlier Lines of Triple-Class Exposed R/R Multiple Myeloma Treatment

The FDA’s Oncologic Drugs Advisory Committee (ODAC) is meeting this week to discuss the supplemental Biologics License Application for idecabtagene vicleucel (Abecma; Bristol Myers Squibb; 2seventybio for triple-class exposed relapsed or refractory multiple myeloma (r/r MM).

2. CBER’s Hosts Webinar on Key Points From FDA’s CAR-T Guidance Document

On March 7, 2024, the FDA's Center for Biologics Evaluation and Research (CBER) hosted a webinar highlighting key points from the final (January 2024) version of the CAR-T guidance document.

3. Neurogene Steps Up the Pace in Trial for Rett Syndrome Gene Therapy NGN-401

Neurogene has amended the protocol for its phase 1/2 pediatric clinical trial (NCT05898620) evaluating NGN-401, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Rett syndrome, to include additional patients in its first cohort, to enable parallel dosing in this cohort, and to add a second cohort.

4. Atara Biotherapeutics Joins Lupus CAR-T Race With Allogeneic Candidate ATA3219

Atara Biotherapeutics’ ATA3219, an investigational allogeneic CD19-directed CAR-T therapy consisting of Epstein-Barr virus-sensitized T-cells that have not been genetically modified, has received clearance on an investigational new drug application from the FDA for a clinical trial in systemic lupus erythematosus and lupus nephritis.

5. Taysha Cleared to Use Higher Dose in Adolescent and Adult Trial for Rett Syndrome Gene Therapy TSHA-102

Taysha Gene Therapies has received approval from an Independent Data Monitoring Committee for a request to escalate to the higher dose cohort in its phase 1/2 REVEAL adolescent and adult clinical trial (NCT05606614) for TSHA-102, an investigational AAV vector-based gene therapy, for the treatment of Rett syndrome.

6. ReciBioPharm and GeneVentiv Therapeutics Join Forces to Tackle Gene Therapy for Hemophilia With Inhibitors

The 2 companies are working to push forward development of GeneVentiv’s GENV-HEM, a universal AAV vector-based gene therapy that is intended to be able to treat all types of hemophilia, including in patients who have developed inhibitors to the missing clotting factor for their disease.

7. Agathos Biologics Makes First Sale of its Recombinant AAV

Agathos has launched its recombinant AAV (rAAV) production service, which uses proprietary cell line AE1-BHK, with the sale of rAAV product to Genovac.“To our knowledge, this is the first sale of rAAV produced using triple transfection in a cell line other than HEK293,” James Brown, PhD, the CEO and cofounder of Agathos, said in a statement. "For our custom services, we will assist clients in the design of a rAAV expression cassette that contains their gene of interest and produce the rAAV for research use both in vitro and in vivo.”

8. NanoMosaic Makes its Tessie Platform Available for Gene Therapy Quality Control

NanoMosaic has launched Tessie, a technology for gene therapy workflow quality control that allows users to gather quantitative data on both AAV capsids and transgenes simultaneously. "The objectives pursued by NanoMosaic are positive steps forward for a space desperate for a fit-for-purpose tool that can be standardized throughout the entire gene therapy development and manufacturing processes," Sylvain Cecchini, PhD, an associate professor of microbiology & physiological systems and director of the AAV Large Scale Manufacturing Facility Core at UMass Chan Medical School, said in a statement. "The ability to better characterize full and intact transgenes can result in more positive patient outcomes with fewer side effects and lower chances for immunogenicity."

9. bluebird bio and Michigan Medicaid Establish Outcomes-Based Agreement for Lovo-cel

bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia) is an FDA-approved ex-vivo lentiviral vector-based gene therapy that is delivered as a 1-time treatment for sickle cell disease. The company noted that it has previously set outcomes-based agreements with a number of commercial insurance companies and is working on additional agreements with more than 15 more Medicaid agencies.