Around the Helix: Cell and Gene Therapy Company Updates – January 25, 2023

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. 10 Cell and Gene Therapies to Watch in 2023

With several Prescription Drug User Fee Act dates already on the calendar and a host of biologics license applications on the horizon, there are 10 therapies we’ll have our eyes on in 2023.

2. Editas Sells off iNK Preclinical Cell Therapy Program, Gene-Editing Technologies to Shoreline

Shoreline Biosciences’ is acquiring Editas Medicines’ cell therapy programs, including its induced pluripotent stem cell derived natural killer cell (iNK) preclinical programs, and licensing its SLEEK (SeLection by Essential-gene Exon Knock-in) and AsCas12a gene editing technologies for oncologic and other indications.

3. Late-Onset Pompe Disease Gene Therapy Cleared to Continue Trial

The FDA has lifted the clinical hold on Astellas Pharma’s phase 1/2 FORTIS trial (NCT04174105) of AT845 in patients with late-onset Pompe disease (LOPD). Astellas is working to resume dosing in the trial.

4. Rett Syndrome Gene Therapy Cleared for Clinical Trial

The FDA has cleared Neurogene’s investigational new drug application of NGN-401 and the company is planning to initiate a phase 1/2 clinical trial to evaluate intracerebroventricular (ICV) injection of the gene therapy in girls with Rett syndrome in 2023.

5. Catalent Announces New Case Management Service for Cell and Gene Therapy Supply Chains

The service will assign customers a case manager who serves as a point of contact for the entire process of supply, manufacture, and delivery.

6. Regen BioPharma Files Patent Application for Method of Stimulating Immune Response to Cancer

The use of survivin-engineered dendritic cells and exosomes to stimulate anti-cancer immunity is the focus of the provisional patent application.

7. Cellectis Makes Changes to Terms of $20 Million Convertible Note Issued by Cytovia Therapeutics

The note was originally issued in relation to the research collaboration and non-exclusive license agreement between the 2 companies.

8. University of Virginia Announces Plans to Launch Paul and Dian Manning Institute of Biotechnology

The institute, which will be launched with the assistance of a $100 million gift, will conduct research and provide care related to advanced medicines, including cell and gene therapies.

9. Medigene Gets $20 Million Milestone Payment from 2seventy bio

The payment relates to a MAGE-A4 cell therapy 2seventy bio is developing in a collaboration with JW Therapeutics that uses a T-cell receptor discovered with Medigene's technology platform.

10. bluebird bio to Put Proceeds from Public Stock Offering Towards ZYNTEGLO and SKYSONA

The net proceeds from the $120 million public offering of common stock will be used in part to fund commercialization and manufacturing activities related to the 2 approved gene therapies.

11. 4D Molecular Therapeutics Pauses Enrollment in Fabry Disease Clinical Trials

Cases of atypical hemolytic uremic syndrome occurred in some patients treated with 4D-310. The company announced intention to use a different immune inhibition regimen in any potential future studies of the gene therapy.