Around the Helix: Cell and Gene Therapy Company Updates – July 6, 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. Gene Therapy for X-Linked Retinitis Pigmentosa Shows Safety, Efficacy in Phase 1/2 Study

MeiraGTx’s botaretigene sparoparvovec (AAV-RPGR), a gene therapy in development for the treatment of X-linked retinitis pigmentosa (XLRP) with disease-causing variants in the RPGR gene, has shown to be generally safe and well-tolerated and demonstrated improvements in several vision-related efficacy end points, according to data from the phase 1/2 clinical trial MGT009 (NCT03252847).

2. ADVM-022 granted Priority Medicines designation by the EMA for the treatment of wet AMD

ADVM-022, an investigational adeno-associated virus (AAV) vector gene therapy by Adverum Biotechnologies, improved anatomic outcomes in patients with wet age-related macular degeneration (AMD) treated in the phase 1 OPTIC trial (NCT03748784). It was granted Priority Medicines (PRIME) designation by the EMA for the treatment of wet AMD on June 24, 2022.

3. Gene Therapy for Canavan Disease Shows Early Promise in Safety and Pharmacodynamics

BridgeBio Pharma’s BBP-812, an investigational gene therapy for the treatment of Canavan disease, has a promising pharmacodynamic and safety profile, according to early data from the first 2 patients treated in the phase 1/2 clinical trial CANaspire (NCT04998396).

4. Vertex’s Type 1 Diabetes Islet Cell Replacement Therapy Resumes Dosing

The FDA has lifted a clinical hold on Vertex Pharmaceuticals’ phase 1/2 clinical trial (NCT04786262) of VX880, a pancreatic islet cell replacement therapy for the treatment of type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia.

5. Clinical Experts in Gene Therapy to be on Scientific Advisory Board announced by Coave Therapeutics

Coave Thereputics has announced the formation of a new scientific advisory board which will include international scientific and clinical experts on gene therapy and neurology. The scientific advisory board will provide guidance for Coave's pipeline of next-generation gene therapy candidates targeting Central Nervous System diseases.

6. Touchlight, Pfizer sign patent license agreement for the use of dbDNA in gene therapy manufacturing

The companies have signed a patent license agreement for Pfizer to use Touchlight's rapid, scalable, enzymatic doggybone DNA (dbDNA) technology in the clinical and commercial manufacture of mRNA-based vaccines, therapeutics, and gene therapies.

7. Mitsubishi Tanabe, EditForce sign deal to pursue discovery of gene therapies for a Central Nervous System disease

In the $146 million deal Mitsubishi Tanabe will use EditForce's proprietary pentatricopeptide repeat (PPR) protein platform technology to research and develop potential treatments for an undisclosed Central Nervous System disease.

8. AstraZeneca to Acquire TeneoTwo's Treatment for Relapsed and Refractory B-cell non-Hodgkin Lymphoma

The phase I clinical stage therapeutic antibody, TNB-486, is a CD19/CD3 T-cell engager and will be evaluated in multiple forms of lymphoma.

9. St. Jude Children’s Research Hospital Creates a Single-Cell Framework with Potential to Improve CAR-T Cell Therapy

The investigators found that a small subset of CAR T-cells out of the total amount prepared for therapy produced most of the enduring anti-cancer activity in children with acute lymphoblastic leukemia.

10. Cytiva, Bayer to Collaborate on Allogeneic Cell Therapy Manufacturing Platform

It will be the first modular end-to-end manufacturing platform for allogeneic cell therapies. The collaboration combines Cytiva’s experience in supplying end-to-end manufacturing solutions with Bayer’s experience with complex therapeutics.

11. Cell Technology Company Evotech Acquires Rigenerand Srl

The company will now operate as Evotech (Modena) Srl.

12. Turnstone Biologics, Moffitt Cancer Center Announce Strategic Alliance to Pursue Development of Tumor Infiltrating Lymphocyte Therapies

Turnstone will have priority access to Moffitt's research, manufacturing, and clinical capabilities for this purpose.