Around the Helix: Cell and Gene Therapy Company Updates – November 30, 2022

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.

1. Hemgenix Approval May Pave Way for Val-Rox in Hemophilia A

BioMarin’s biologics license application (BLA) review for its adeno-associated virus gene therapy valoctocogene roxaparvovec (val-rox; Roctavian) may not be greatly delayed after all, as the company announced that the FDA will no longer be holding an advisory committee meeting to discuss the therapy’s benefit in hemophilia A. The FDA will still complete the scheduled pre-licensure inspection of BioMarin’s gene therapy manufacturing facility in Canada.

2. Sarepta’s DMD Gene Therapy Accepted for Priority Review

The FDA has accepted and granted priority review to Sarepta Therapeutics’ biologics license application (BLA) for SRP-9001, an investigative gene therapy for treating Duchenne muscular dystrophy (DMD), with a PDUFA date set for May 29, 2023.

3. FDA Recognizes Allogeneic CAR-T Therapy for Lymphomas

The FDA has granted fast track designation to Caribou Biosciences’ chimeric antigen receptor (CAR) T-cell therapy CB-010 for treating relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) and regenerative medicine advanced therapy designation for treating R/R large B cell lymphoma (LBCL).

4. ARV Cardiomyopathy Gene Therapy Gets Orphan Drug Designation

Tenaya Therapeutics’ TN-401, an investigational adeno-associated virus (AAV)-based gene therapy intended to treat arrhythmogenic right ventricular cardiomyopathy (ARVC), has been granted orphan drug designation by the FDA.

5. Turnstone Biologics, National Cancer Institute to Collaborate on TIL and Viral Immunotherapy Research

The research will focus on evaluating Turnstone's oncolytic viruses' ability to generate Selected tumor infiltrating lymphocytes (TILs).

6. Eikonoklastes Therapeutics, Forge Biologics Partner to Bring ALS Gene Therapy to the Clinic

Forge Biologics will provide manufacturing services for Eikonoklaste's investigational adeno-associated virus (AAV) vector-based gene therapy, ET-101, with the goal of helping to bring it to clinical trials for the treatment of amyotrophic lateral sclerosis (ALS).

7. AVROBIO Integrating Mission Bio's Method for Determining Cell Transduction Percentage

AVROBIO presented data regarding the collaboratively developed one single-cell transduction assay's ability to evaluate percent transduction of genetically modified cell product at the 4th Annual Gene Therapy Analytical Development (GTAD) Summit on November 29, 2022.

8. iECURE Finishes Round of Series A-1 Financing for Investigational Gene-Editing Therapies for Rare Pediatric Liver Disease

iECURE intends to use the $65 million raised to advance its gene-editing products intended for the treatment of ornithine transcarbamylase deficiency and citrullinemia type 1 towards the clinic.

9. Eli Lilly and Company's Acquisition of Akouos to be Consummated

The tender offer expired on November 29 at 1 minute past 11:59 PM, and the consummation is expected to occur on December 1, 2022.

10. Researchers at MIT Develop New DNA Insertion Method

The programmable addition via site-specific targeting elements (PASTE) method, expounded in a study published in Nature Biotechnology, could allow for large gene insertion without necessitating the use of DNA repair pathways.

11. Invitrx Therapeutics Receives Warning Letter from FDA

The November 9 letter is related to a March 2022 inspection that identified potential concerns regarding the company's cellular and exosome products.