Around the Helix: Cell and Gene Therapy Company Updates – December 17, 2025

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. FDA Approves Waskyra as First Gene Therapy for Wiskott-Aldrich Syndrome

The FDA has approved etuvetidigene autotemcel (Waskyra; Fondazione Telethon ETS) as the first cell-based gene therapy approved for the treatment of Wiskott-Aldrich syndrome (WAS). Its indication is specifically for pediatric patients aged 6 months and older and adults with a mutation in the WAS gene for whom hematopoietic stem cell transplantation (HSCT) is appropriate but a suitable HLA-matched related donor is unavailable.

2. MDA 2026 to Bring Keynote Insights and Evolving Neuromuscular Care

Each year, the Muscular Dystrophy Association (MDA) holds its Clinical and Scientific Conference, which has evolved into one of the most influential meetings in the world focused entirely on neuromuscular disease. In the lead up to the keynote announcement for 2026's conference, CGTLive's sister site, NeurologyLive®, interviewed Sharon Hesterlee, PhD, MDA's president and chief executive officer. Hesterlee shared her insight on the keynote's significance, spoke on the design of the conference, and discussed her reasoning on why clinicians will see 2026's meeting as more essential than ever.

3. Evaluating Gene Editing Therapy Reni-Cel for Severe Sickle Cell Disease

Investigational sickle cell disease gene editing candidate renizgamglogene autogedtemcel (reni-cel, previously known as EDIT-301) was evaluated in the phase 1/2 RUBY clinical trial (NCT04853576). At the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, CGTLive® spoke to Rabi Hanna, MD, the chairman of the Division of Pediatric Hematology & Oncology and BMT at Cleveland Clinic Children's, shortly before he presented new data from RUBY at the conference.

4. What the Field of Cell and Gene Therapy Needs Most in the Current Moment

Deborah Phippard, PhD, shared her thoughts with Renier Brentjens, MD, PhD, on the importance of big wins for those in cell and gene therapy research. She pointed to examples like mainstream papers such as The New York Times covering gene therapy research breakthroughs and the huge advance in sickle cell disease treatment provided by the FDA's approval of 2 new gene therapies in 2023.

5. New Company Focused on Logic-Gated CAR-T Unveils Itself

Link Cell Therapies, a company focused on the development of logic-gated chimeric antigen receptor T-cell (CAR-T) therapies for solid tumor and blood cancer indications, has come out of stealth. Notably, the company received Series A financing from investors including Johnson & Johnson Innovation and Bristol Myers Squibb. “Link is advancing a technology that we developed while at Stanford University that allows for logic-gated CAR-T cell control," Link cofounder Robbie Majzner, MD, of the Dana-Farber Cancer Institute and Harvard Medical School, said in a statement. "The Link-based CAR activates and kills target cells only when a combination of antigens is colocalized on the tumor, thereby bypassing normal tissues that express only one of those targets."

6. FDA Lifts Clinical Hold on Tenaya's MYBPC3-Associated Hypertrophic Cardiomyopathy Gene Therapy Trial

The FDA has lifted a clinical hold that it had previously placed on the phase 1b/2a MyPeak-1 clinical trial (NCT05836259), which is evaluating TN-201, an investigational adeno-associated virus (AAV)-vector based gene therapy, for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). The hold was originally place by the FDA in November 2025 when Tenaya reached out to the agency explaining its intent to make a protocol amendment to MyPeak-1. Tenaya now states that all concerns leading to the clinical hold have been addressed and that it will resume dosing in the trial once it implements the protocol amendments.