Around the Helix: Cell and Gene Therapy Company Updates – November 2, 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. GSK, Lyell Immunopharma Terminate Agreement for Targeted Cell Therapies

Lyell Immunopharma announced in an October 24, 2022, SEC filing that GlaxoSmithKline (GSK) had informed the company of the decision to terminate an agreement regarding its development of several investigational cell therapy products which target NY-ESO-1 and utilize Lyell’s genetic and epigenetic reprogramming technologies.

2. Hepatocellular Carcinoma RNA-Editing Therapy Gets FDA IND Clearance

Rznomics’s RZ-001, an investigational ribozyme-based RNA reprogramming therapy, has received clearance of its investigational new drug (IND) application from the FDA, which will allow for initiation of a phase 1/2a clinical trial for patients with hepatocellular carcinoma (HCC).

3. Gaucher Disease Gene Therapy Granted FDA Rare Pediatric Disease Designation

AVROBIO’s AVR-RD-02, an investigational gene therapy intended to treat Gaucher disease type 1, has received rare pediatric disease designation from the FDA. It also received ILAP designation in the UK earlier in October.

4. CAR Monocyte Therapy Fast Tracked for Peripheral T-Cell Lymphoma

Myeloid Therapeutics’ MT-101, an investigational autologous mRNA engineered chimeric antigen receptor (CAR) monocyte therapy, has received fast track designation from the FDA for the treatment of patients with relapsed or refractory (r/r) CD5+ peripheral T-cell lymphoma (PTCL).

5. Polyplus, ENCell Partner to Optimize Viral Vector Manufacturing

As part of the memorandum of understanding signed between the 2 companies, Polyplus will provide support to the optimization projects taking place at 2 of ENCell's South Korean facilities.

6. ABL, RD-Biotech Partner to Provide End-to-end Gene Therapy Manufacturing Services

The strategic partnership between the 2 CDMOs will enable them to offer customers manufacturing services for both plasmid DNA and viral vectors in a streamlined process.

7. Replay Launches New Herpes Simplex Virus Vector-based Gene Therapy-Focused Company Eudora

Eudora will utilize Replay's synHSV technology and pursue gene therapies for indications including retinitis pigmentosa, Stargardt disease, and Usher syndrome type 1B.

8. Second Line Axicabtagene Ciloleucel Injection NDA Accepted for Review by China's NMPA

Axicabtagene Ciloleucel Injection (YIKAIDA) is listed for priority review for the treatment of patients with large B-cell lymphoma who did not respond to first-line immunochemo therapy or relapsed in 12 months or less post-treatment.

9. Eterna Therapeutics and Michael Andreeff, MD, PhD, to Collaborate on iPS Gene-edited Cell Therapy Research

Michael Andreeff, MD, PhD, professor of leukemia, The University of Texas MD Anderson Cancer Center, will investigate the ability of Eterna's investigational induced pluripotent stem (iPS) cell therapies, which are intended to treat various indications such as acute myeloid leukemia and solid tumors, to initiate enhanced immune responses and promote multilineage differentiation and hematopoietic regeneration.

10. Obsidian Therapeutics, Bristol Myers Squibb to Extend Cell Therapy Collaboration

The collaboration is focused on research and development for cell therapies that use Obsidian’s cytoDRiVE technology for controlled expression of CD40L.

11. Atossa Therapeutics Invests in Dynamic Cell Therapies

Atossa expects that the investment in Dynamic Cell Therapies, which is in the process of developing CAR-T therapies for difficult-to-treat cancers, will put the company in a strategic position for future CAR-T therapy-related opportunities.