Around the Helix: Cell and Gene Therapy Company Updates – September 20, 2023

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. New Expert Insights at ANA 2023

Catch up on CGTLive’s interview coverage of the 148th Annual Meeting of the American Neurological Association (ANA), held September 9-12, 2023, in Philadelphia, Pennsylvania.

2. Orchard Therapeutics’ BLA for Metachromatic Leukodystrophy Cell Therapy Arsa-Cel Accepted With Priority Review

Orchard Therapeutics’ biologics license application (BLA) for atidarsagene autotemcel (arsa-cel, also referred to as OTL-200 and approved as Libmeldy in the European Union, UK, Iceland, Liechtenstein, and Norway), a gene-edited cell therapy being evaluated for the treatment of metachromatic leukodystrophy, has been accepted by the FDA with priority review.

3. Hemogenyx Clears a Path for AML CAR-T Therapy

Hemogenyx Pharmaceuticals has reached an accord with the FDA on its plan to resolve the agency’s current clinical hold on its HEMO-CAR-T investigational new drug application for treating acute myeloid leukemia (AML).

4. Rocket Pharmaceuticals Confirms Phase 2 Trial Plans for Danon Disease Gene Therapy With FDA

Rocket Pharmaceuticals has come into alignment with the FDA on plans for its pivotal phase 2 clinical trial evaluating RP-A501, an investigational adeno-associated virus vector-based gene therapy intended to treat Danon disease, according to an announcement from the company.

5. New Company AlveoGene Will Direct Efforts Towards Inhalation-based Gene Therapy

AlveoGene, a new company launched with funding from investors including Oxford Science Enterprises and Harrington Discovery Institute, will initially pursue the development of a lentiviral gene therapy for alpha-1 antitrypsin deficiency disease that will utilize a nebulizer for an inhalation delivery method to the epithelial cells of the lungs.

6. AI-focused Company Generate:Biomedicines Closes Out Round of Series C Financing

The $273 million round was supported by Amgen, NVIDIA’s venture capital arm, and other investors. Generate:Biomedicines’ pipeline, which includes therapeutics based on its machine-learning-powered generative biology platform, The Generate Platform, has cell therapy and T-cell engagers among its diverse modalities. The company will use the new funding to further advance the platform.

7. AIRNA Comes Out of Stealth

AIRNA, a newly launched company focused on developing therapeutics based on its RESTORE+ RNA editing platform, exited stealth furnished with $30 million from investors led by ARCH Venture Partners. “We now have the capability to precisely rewrite RNA’s genetic instructions, which creates the potential to address a wide range of new targets and diseases,” Rodger Novak, MD, AIRNA’s Board Chair, said in a statement.

8. ResVita Bio’s Engineered Cell Therapy Secures Funding Via NIAID Grant

Startup ResVite Bio is developing RVB-101, an engineered cell therapy intended to treat severe atopic dermatitis, which is the subject of a $250,000 National Institute of Allergy and Infectious Disease (NIAID) Phase 1 Small Business Innovation Research (SBIR) grant. The cell therapy is applied directly to the skin in a moisturizer formulation and meant to continuously secrete therapeutic proteins during temporary colonization of the relevant area.

9. China-based Companies Porton Advanced and BRL Medicine Team Up

Porton Advanced, a contract development and manufacturing organization (CDMO), will help push forward the development of BRL Medicine’s therapies, which include gene therapy BRL-101 and chimeric antigen receptor T-cell therapy BRL-201. Porton’s CDMO platform covers modalities including plasmids, cell therapy, gene therapy, oncolytic viruses, mRNA therapy, and bacterial therapy.