Around the Helix: Cell and Gene Therapy Company Updates – September 25, 2024

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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social and use #AroundTheHelix!

1. Poseida Therapeutics Garners FDA RMAT Designation for Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma

Poseida Therapeutics’ P-BCMA-ALLO1, an allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, has received regenerative medicine advanced therapy (RMAT) designation from the FDA for the treatment of adults with multiple myeloma (MM) whose disease is relapsed/refractory (r/r) following at least 3 previous lines of treatment that included a proteasome inhibitor, an immunomodulatory agent, and an antiCD38 antibody.

2. Rocket Pharmaceuticals Maxes Out Recruitment in Pivotal Trial for Danon Disease Gene Therapy RP-A501

Rocket Pharmaceuticals has finished enrolling all patients in a pivotal phase 2 clinical trial (NCT06092034) evaluating RP-A501, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of Danon disease.

3. Kiromic BioPharma Gets Greenlight to Move to Dose Expansion in Trial for NSCLC T-cell Therapy Deltacel

Kiromic BioPharma has received a unanimous recommendation to move ahead dose expansion from the safety monitoring committee (SMC) for the phase 1/2 Deltacel-01 clinical trial (NCT06069570) evaluating KB-GDT-01 (Deltacel), an investigational allogeneic gamma delta T-cell (GDT) therapy, for the treatment of non-small cell lung cancer (NSCLC).

4. Biomarker Data from Oncology Trial for Adicet Bio’s Gamma Delta T-cell Therapy ADI-001 Indicates Potential for Use in Autoimmune Disease

Biomarker data collected in the phase 1 GLEAN clinical trial (NCT04735471) evaluating Adicet Bio’s ADI-001, an investigational allogeneic CAR-engineered gamma delta T-cell therapy, for the treatment of B-cell malignancies, indicates the therapy’s potential to treat autoimmune disease, according to the company.

5. uniQure Snags Orphan Drug Designation for Fabry Disease Gene Therapy

AMT-191 is an AAV5 gene therapy product that delivers an α-galactosidase A (GLA) transgene targeted to the liver to produce the GLA protein deficient in patients with Fabry disease. “This designation supports our phase 1/2a clinical trial and we look forward to rapidly generating clinical proof-of-concept data and providing initial data in 2025," Walid Abi-Saab, MD, the chief medical officer of uniQure, said in a statement.

6. bluebird bio Downsizing

The company has announced a restructuring intended to reduce expenses, which will include letting go of around 25% of its employees. Patient use of bluebird bio’s most recently approved gene therapy product lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia) in the commercial setting has remained low. The company noted that across its 3 gene therapy products, which also include Zynteglo and Skysona, 41 patent starts have been recorded thus far this year.

7. Ori Biotech and Charles River Announce Results from Test of Next-Generation Manufacturing Platform

The 2 companies collaborated on head-to-head process runs comparing Ori's IRO, a next-generation cell and gene therapy manufacturing platform, to Charles River Laboratories (CRL)'s own optimized CAR-T manufacturing process. Notably, IRO produced 2.1 billion total CAR+ cells in comparison to 1.6 billion for CRL's process. "IRO automates better biology to solve critical manufacturing bottlenecks and improve product viability by dramatically increasing throughput, decreasing COGS, and reducing batch failures, with the ultimate goal of enabling widespread patient access to life-saving cell and gene therapies," Jason C. Foster, MBA, the CEO of Ori Biotech, said in a statement.

8. Belief BioMed and AskBio Team Up

The 2 companies have announced their intent to develop novel gene therapy products that will utilize a liver-targeted approach to treat indications with high unmet need. "Collaborating with innovative, like-minded partners with complementary gene therapy expertise enables us to find the best way forward for our pipeline assets and bolster our efforts to advance new therapies for conditions with significant unmet need," Gustavo Pesquin, MBA, the CEO of AskBio, said in a statement.