Around the Helix: Cell and Gene Therapy Company Updates – December 21, 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. FDA Approves Ferring's Gene Therapy for Bladder Cancer

The FDA has approved the first gene therapy for the treatment of bladder cancer, according to an announcement from the agency and the drug's manufacturer, Ferring.

2. Partial Hold Lifted on bluebird bio's Sickle Cell Disease Gene Therapy

The FDA’s partial clinical hold suspending enrollment, cell collection, conditioning, and infusion of patients under the age of 18 in bluebird bio's phase 3 HGB-210 clinical trial (NCT04293185) of lovotibeglogene autotemcel (lovo-cel; bb1111), an investigational gene therapy intended to treat sickle cell disease (SCD), has been lifted.

3. Ocugen Announces Positive FDA Interactions Regarding Optical Gene Therapy and Orthopedic Cell Therapy

The agency granted OCU400 orphan drug designation for additional indications and agreed to a phase 3 trial design for NeoCart. Ocugen also announced that a new current Good Manufacturing Practice cell therapy manufacturing facility is under construction that will support clinical and commercial production of NeoCart.

4. Solid Tumor CAR T Collaboration Cleared for Trials

The FDA has cleared the investigational new drug application (IND) of Invectys and CTMC’s human leukocyte antigen A (HLA-G) targeting chimeric antigen receptor (CAR) T-cell therapy IVS-3001 for treating solid tumors.

5. Kite Announces Upcoming Acquisition of Tmunity Therapeutics

In addition to Tmunity's rapid manufacturing processes, ‘armored’ CAR-T technology platform, and other programs in various stages of development, the acquisition will also provide Kite with access to a strategic research and licensing agreement with the University of Pennsylvania.

6. Mustang Bio Announces Updates Regarding CAR-T Programs

It was noted that the MB-106 clinical trial is continuing to enroll patients, while the Safety Review Team for MB-102's clinical trial recommended a dose escalation to 300x10⁶ CAR-T cells, and positive preclinical data regarding the company's MB-109 combination therapy was presented at the American Association for Cancer Research Annual Meeting 2022. An exclusive license agreement with the Mayo Clinic for an in vivo CAR-T platform technology was also highlighted.

7. Taysha Gene Therapies Announces Appointment of Sean P. Nolan as New CEO

In addition, the company announced that is has appointed Sukumar Nagendran, MD, as the new president and head of research and development.

8. Celyad Oncology Announces Updates to Business Strategy

The company intends to focus on intellectual property partnering and discovery research in several areas in the wake of its discontinuation of the CYAD-211 CAR-T clinical program for relapsed/refractory multiple myeloma.

9. Sio Gene Therapies to Dissolve

After considering various strategic options, the Board of Directors determined that complete liquidation and dissolution is in the best interest of the company's shareholders.

10. CHMP Recommends Hemophilia B Gene Therapy Etranacogene Dezaparvovec for Approval

The European Medicines Agency’s Committee for Medicinal Products for Human Use made the recommendation for the conditional marketing authorization based on results from HOPE-B (NCT03569891), a phase 3 clinical trial.

11. Oxford Biomedica Solutions Partners With 3 Undisclosed US-based Biotechs

Oxford Biomedica Solutions will support the development of the biotechnology companies' gene therapies by providing access to its platform, which is intended to accelerate development and produce more doses per batch.

12. Genprex Enters License Agreement Related to Preclinical Type 1 Diabetes Gene Therapy Technology With the University of Pittsburgh

The technology is intended to enable macrophages to reduce autoimmune activity.

13. Catalent Announces New Biologics Analytical Services Facility

The Durham, North Carolina-based facility will be 80,000 square feet and will provide analytical development and testing services for biologics, including cell therapies and gene therapies.

14. Liso-Cel Approved for Second-Line LBCL in Japan

The approval of Breyanzi is based on the results of clinical trials in patients with relapsed or refractory aggressive B-cell non-Hodgkin lymphoma after first-line therapy, including global phase 3 clinical trials (JCAR017-BCM-003), phase 2 clinical trials (017006) in the United States, and cohort 2 of phase 2 clinical trials (JCAR017-BCM-001) in Europe and Japan.