Around the Helix: Cell and Gene Therapy Company Updates – July 27, 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. Allogeneic CAR T-Cell Therapy Granted FDA Designations in T-Cell Malignancies

Wugen’s WU-CART-007 has been granted fast track, rare pediatric disease, and orphan drug designations.

2. Upstaza Approved by European Commission for AADC Deficiency

The European Commission has granted marketing authorization to PTC Therapeutics’ gene therapy Upstaza (eladocagene exuparvovec, PTC-AADC) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

3. Phase 3 Cardiosphere-Derived Cell Therapy Trial Doses First Patient With DMD

Capricor Therapeutics has dosed the first patient with Duchenne muscular dystrophy (DMD) in its phase 3 HOPE-3 study (NCT05126758) of CAP-1002, an allogeneic cardiosphere-derived cell therapy.

4. FDA Clears IND Application for TIL Therapy OBX-115 for R/R Metastatic Melanoma

The FDA has cleared the investigational new drug application for a phase 1 clinical study of OBX-115, an investigational tumor infiltrating lymphocyte (TIL) therapy developed by Obsidian Therapeutics and the University of Texas MD Anderson Cancer Center for the treatment of relapsed/refractory metastatic melanoma.

5. First Patient Dosed in RAG1-SCID Gene Therapy Trial

Mustang Bio has dosed the first participant with recombinase-activating gene-1 (RAG1) severe combined immunodeficiency (RAG1-SCID) with MB-110 in a phase 1/2 clinical trial (NCT04797260) in Europe.

6. CAR-Treg Cell Therapy TX200 Receives Orphan Medicinal Product Designation From European Commission

Sangamo Therapeutics' TX200 is intended to reduce the risk of rejection in solid organ transplant. The STEADFAST phase 1/2 clinical trial dosed its first patient in March 2022.

7. Big DNA Focused Company Replay Launches With $55 Million Seed Financing

The new company Replay will advance platform technologies that enable the writing and delivery of big DNA. These technologies could help enable polygenic therapy.

8. First Patient Treated With NK Cell Therapy CYNK-101 for HER2+ G/GEJ Cancers

Cellularity's allogeneic off-the-shelf human placental CD34+ stem cell derived NK cell therapeutic candidate is genetically modified to express high-affinity and cleavage-resistant CD16 variant to enhance antibody-dependent cell-mediated cytotoxicity.

9. CHMP Issues Positive Opinion for CAR T-cell Therapy for R/R Acute Lymphoblastic Leukemia

Kite's Tecartus, if approved, will be the only CAR T-cell therapy for patients 26 years of age and older with r/r B-cell precursor acute lymphoblastic leukemia.

10. Berkeley Lights Beacon Platform Integrated Into Otsuka Pharmaceuticals' Cell and Gene Therapy Research

The Beacon Platform, which Otsuka Pharmaceuticals acquired in October 2021, is also being used for cell line development research and antibody discovery.

11. License and Supply Agreement for LentiVector Platform Signed between Oxford Biomedica and Undisclosed US Partner for CAR-T Therapy

The undisclosed private biotechnology company will gain the right to use the LentiVector platform in their lead CAR-T program, and in return Oxford Biomedica will receive an undisclosed upfront payment and various other payments and royalties related to development, manufacturing for clinical trials, and/or sales.

12. A-Gene, a Case Study Aiming to Incorporate Quality by Design Principles Into Gene Therapy Manufacturing, has Been Released

A-Gene, a collaboration between The Alliance for Regenerative Medicine and the National Institute for Innovation in Manufacturing Biopharmaceuticals, will attempt to address the issues that come from a lack of standardized methodologies and training for Chemistry, Manufacturing, and Controls in the field of gene therapy.