Around the Helix: Cell and Gene Therapy Company Updates – August 31, 2022


Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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. Sangamo’s Gene Therapy Promising for Fabry Disease

Isaralgagene civaparvovec (ST-920; Sangamo Therapeutics) has been well-tolerated and restored production of α-galactosidase A (α-Gal A) in people with Fabry disease, according to updated data from the phase 1/2 STAAR clinical trial (NCT04046224).

2. FDA Recognizes Ornithine Transcarbamylase Deficiency Gene Editing Therapy

The FDA has granted rare pediatric disease designation to iECURE’s gene editing therapy, GTP-506, being investigated for the potential treatment of Ornithine Transcarbamylase (OTC) deficiency.

3. Roctavian Approved for Hemophilia A in the EU

Roctavian (valoctocogene roxaparvovec), BioMarin’s gene therapy for treating severe hemophilia A, has been approved in the EU with conditional marketing authorization.

4. Allogeneic CAR-iNK Therapy to be Evaluated in B-Cell Malignancies

The FDA has granted investigational new drug (IND) clearance to Century Therapeutics’ chimeric antigen receptor (CAR) induced pluripotent stem cell (iPSC)-derived natural killer (NK) cell therapy, CNTY-101, being investigated for the treatment of B-cell malignancies.

5. Huntington Disease Gene Therapy Trial Cleared to Start in France

BrainVectis has received approval from France’s National Agency for Safety of Medicines and Health Products (ANSM) to begin recruiting patients for a phase 1/2 clinical trial of its investigational adeno-associated virus (AAV) gene therapy for the treatment of early-stage Huntington disease (HD), BV-101.

6. Iovance Starts BLA Submission of Lifileucel for Advanced Melanoma

Iovance Biotherapeutics has initiated a rolling biologics license application (BLA) submission for its tumor-infiltrating lymphocyte (TIL) therapy intended to treat unresectable or metastatic melanoma that progressed on or after prior anti-PD-1/L1 therapy, and if BRAF mutation positive, also prior BRAF or BRAF/MEK inhibitor therapy.

7. CARsgen Advances Autologous CAR-T Therapies in Multiple Myeloma, Advanced Gastric Cancer

CARsgen has completed patient enrollment in the phase 2 LUMMICAR STUDY 1 (NCT03975907) of CT053 (zevor-cel) and initiated a confirmatory phase 2 trial of CT041, marking the first of its kind trial of CAR T-cell therapies in solid tumors.

8. Allocetra Combination Therapy to Be Assessed in Solid Tumors

The Israeli Ministry of Health (MOH) has approved the initiation of a phase 1/2 clinical trial to investigate Enlivex’s Allocetra cell therapy, both alone and in combination with a PD-1 checkpoint inhibitor, for the treatment of solid tumors.

9. Forge Biologics Announces Positive Data from Gene Therapy Trial for Patients with Krabbe Disease

The first patient treated demonstrated improvements in motor function and FBX-101 was well-tolerated at 180 days.

10. REGENXBIO Announces Positive Interim Data from Clinical Trial of Gene Therapy for MPS II

RGX-121 was well-tolerated across 3 dose levels and demonstrated reductions in key disease biomarkers.

11. Gensaic, Ovid to Collaborate on Phage-Derived Gene Therapies for CNS Indications

Gensaic will utilize its phage-derived particle (PDP) platform to develop gene therapies for central nervous system (CNS) indications of interest to Ovid, who will have the rights to license and develop any of the resulting therapies.

12. ElevateBio, University of Pittsburgh to Create Process Development and Manufacturing Facility for Cell and Gene Therapies

The creation of the Pitt BioForge BioManufacturing Center is supported by a $100 million grant from Richard King Mellon Foundation, and the center will feature cell, vector, and protein engineering capabilities

13. Applied StemCell to Expand Manufacturing Facility

The expansion is set to begin within a month and will add 4 cGMP clean rooms, cryo-storage space, and process development space. Completion of the expansion is expected early next year.

14. Porton Advanced Solutions, Kun Tuo Medical Reasearch and Development Enter Strategic Partnership

The partnership will be focused on advancing clinical reasearch, development, and manufacturing for new cell and gene therapies.

15. BioCardia, BlueRock Therapeutics Enter Agreement to Deliver Cell Therapy to the Heart

BioCardia will provide its biotherapeutic delivery product candidates for BlueRock to use to deliver its cell therapy product candidates to the heart for the treatment of heart failiure.

16. Formation of New Cell and Gene Therapy Accelarator Mayflower BioVentures Announced

The accelerator is the result of the combined efforts of Mayo Clinic, Hibiscus BioVentures, and Innoforce, and will form new independent companies based around Mayo Clinic technologies to advance development of cell and gene therapies to adress unmet needs for patients with serious conditions.

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