Around the Helix: Cell and Gene Therapy Company Updates – August 14, 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.

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1. Ocugen’s Retinitis Pigmentosa Gene Therapy Available in EAP Ahead of BLA Submission

The FDA has approved an Expanded Access Program (EAP) for Ocugen’s OCU400 gene therapy program for adults with retinitis pigmentosa.

2. BioMarin Limits Geographic Scope of its Plans for Hemophilia A Gene Therapy

BioMarin has announced that it will be scaling back its efforts with regard to valoctocogene roxaparvovec (val-rox, marketed as Roctavian), its gene therapy product for the treatment of adults with severe hemophilia A. Going forward, the company is mainly focused on distributing the gene therapy in the United States, Germany, and Italy, the 3 countries in which it has been approved for use by relevant regulatory authorities and is reimbursed.

3. FDA Lifts Clinical Hold on Trial for 4D Molecular Therapeutics’ Fabry Disease Gene Therapy 4D-310

The FDA has lifted a clinical hold on the phase 1/2 INGLAXA (NCT04519749) clinical trial for 4D Molecular Therapeutics (4DMT)’s 4D-310, an investigational adeno-associated virus (AAV) vector-based gene therapy being evaluated for the treatment of Fabry disease.

4. TG Therapeutics Receives Clearance from FDA for Phase 1 Trial in Multiple Sclerosis for Allogeneic CAR-T Azer-Cel

The FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). Azer-cel was originally developed by Precision BioSciences, but licensed to TG Therapeutics for further development for the potential treatment of autoimmune diseases and other noncancer indications.

5. Genprex Garners Patent for Reqorsa Combination Treatment in Singapore

Genprex has received a patent from the Singapore Patent Office relating to the use of quaratusugene ozeplasmid (Reqorsa), an investigational immunogene therapy intended to treat various forms of lung cancer, in combination with anti–PD-1 antibodies. The patent, which provides coverage through 2037, joins patents the company has received for the gene therapy's use in combination with an immune checkpoint inhibitor in several other countries across the globe.

6. Sangamo and Genentech Team Up to Tackle Neurodegenerative Disease

Sangamo Therapeutics and Genentech have entered a new license agreement aimed at the development of genomic medicines for neurodegenerative disease associated with the tau gene, such as Alzheimer disease, and another undisclosed neurology target. Via the agreement, Genentech has secured exclusive license to use Sangamo's STAC-BBB AAV capsid, and will be responsible for clinical development through commercialization of any therapeutic candidates that come out of the agreement.

7. SK pharmteco and Rznomics Join Forces

Under a new memorandum of understanding, contract development, manufacturing, and analytical testing organization SK pharmteco and biopharmaceutical company Rznomics will work together to bring the latter's RNA-based gene therapy products through clinical development and potential commercialization. “This partnership will mark late-stage development and manufacturing of our gene therapy pipeline to prepare the path towards phase 2 and later stage clinical trials and potential commercialization," Seong-Wook Lee, PhD, the president and CEO of Rznomics, said in a statement.

8. Eli Lilly Launches New Lilly Seaport Innovation Center for DNA and RNA-Based Therapeutics

Eli Lilly and Co. has launched a new research and development facility, referred to as the Lilly Seaport Innovation Center (LSC), which will conduct activities related to the company's programs in DNA and RNA-based therapy. Located in Boston Seaport, LSC comprises as 346,000 square foot facility spread through a 12-story building and will constitute the workplace of approximately 700 people, including both Lily employees and employees of companies that make up Lilly Gateway Labs.