Around the Helix: Gene and Cell Therapy Company Updates - January 26, 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. GeneTherapyLive’s Around the Helix is your chance to catch up with the latest news in gene and cell therapies, including partnerships, pipeline updates, and more.

1. Kriya Licenses Next-Generation Gene Therapies for Geographic Atrophy

The Medical University of South Carolina (MUSC) Foundation for Research Development is developing next generation complement-targeted gene therapies. Kriya Therapeutics has entered into an exclusive agreement to license the therapies for geographic atrophy and other ocular diseases.

2. New Platform to Improve Accessibility of Cell and Gene Therapies

AmerisourceBergen and TrakCel have collaborated to launch a new, integrated platform, which combines TrackCel’s OCELLOS, an advanced therapy orchestration platform, and AmerisourceBergen’s Fusion, a customer relationship management and patient support platform, to improve therapy accessibility.

3. IND Cleared for Solid Tumors TCR Therapy

Lyell Immunopharma is developing LYL132, an NY-ESO-1-targeted T-cell receptor therapy in collaboration with GlaxoSmithKline. The therapy is developed using Lyell’s novel epigenetic reprogramming Epi-R technology. The companies are set to initiate a phase 1/2 trial following the investigational new drug application clearance.

4. T-Cell Therapy Gets Orphan Drug Designation for Pancreatic Cancer

The FDA has granted orphan drug designation to Marker Therapeutics’ multitumor-associated antigen-specific T-cell therapy MT-601, for the treatment of patients with pancreatic cancer. The therapy is designed to target 6 tumor-associated antigens highly expressed in pancreatic cancer: PRAME, NY-ESO-1, Survivin, MAGE-A4, SSX2, and WT1

5. CLN6 Batten Disease Gene Therapy Discontinued Due to Durability Issues

Amicus Therapeutics has made the decision to discontinue AT-GTX-501 for CLN6 Batten disease, after long-termstudy data (NCT04273243) revealed that initially stabilized disease continued to progress after 2 years of follow-up. The company is still developing AT-GTX-502 for CLN3 Batten disease

6. Stem Cell Gene Therapy Efficacious in Metachromatic Leukodystrophy

Orchard Therapeutics’ gene therapy atidarsageneautotemcel (arsa-cel;,Libmeldy) slows demyelination and brain atrophy and preserved cognitive function and motor development in patients with metachromatic leukodystrophy, according to data published in The Lancet.

7. Tevogen Bio to Study T-Cell Technology in EBV and MS

Following the recent study that revealed the strong connection between Epstein-Barr-Virus (EBV) and multiple sclerosis (MS), Tevogen Bio has announced their intention to develop EBV-specific CD8+ cytotoxic T lymphocyte, off-the-shelf cell therapies for the treatment of MS.

8. Adaptimmune to Evaluate TCR-T Therapy in Advanced Esophageal or EGJ Cancers

The company’s cell therapy ADP-A2M4CD8 will be evaluated in the open-label, single-arm, phase 2 SURPASS-2 trial (NCT04752358), which will run in parallel with the ongoing phase 1 SURPASS trial (NCT04044859) in patients with advanced esophageal or esophagogastric junction (EGJ) HLA-A*02- and MAGE-A4- positive cancers.

9. Lysogene Terminates Agreement With Sarepta for Mucopolysaccharidosis Type 3A Gene Therapy

Lysogene will regain global commercialization rights to LYS-SAF302 as of July 11, 2022. The therapy is currently being evaluated in the phase 2/3 AAVance clinical trial (NCT03612869) and initial data readout is expected in mid-2022.

10. Idecabtagene Vicleucel Approved in Japan for Relapsed/Refractory Multiple Myeloma

The approval of Abecma (Bristol Myers Squibb; bluebird bio) marks the first approved chimeric antigen receptor (CAR) T-cell therapy to be approved for multiple myeloma in Japan. Approval was based on efficacy and safety data from the phase 2 KarMMA study (NCT03361748) conducted in Japan, the US, EU, and Canada, and the phase 1 CRB-401 study (NCT03274219) in the US.