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


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

CGTLive Around the Helix

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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social and use #AroundTheHelix!

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.

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