Around the Helix: Cell and Gene Therapy Company Updates – March 12, 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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social (@CGT_Live on X, or CGTLive on LinkedIn) and use #AroundTheHelix!

1. Neurotech’s Encapsulated Cell Therapy Encelto Approved by FDA for Macular Telangiectasia Type 2

Neurotech Pharmaceuticals’ revakinagene taroretcel-lwey (also known as NT-501), an allogeneic encapsulated cell therapy implant, has been approved by the FDA for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel). It is to be marketed under the name Encelto.

2. Stem Cell Therapy CALEC Restores Corneal Damage in Trial Led by Massachusetts Eye and Ear Investigators

A therapy known as cultivated autologous limbal epithelial cells (CALEC) transplantation showed over 90% partial and complete success rates in repairing the corneal surfaces that had corneal damage believed to be irreversible, according to data from a phase 1/2 clinical trial (Clinicaltrials.gov registration: NCT02592330) that was published in Nature Communications. The study is led by investigators at the Massachusetts Eye and Ear in Boston, including Ula Jurkunas, MD, PhD, who is associate director of the Cornea Service at the Mass Eye and Ear and professor of Ophthalmology at Harvard Medical School, Boston.

3. News and Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month 2025

For Bleeding Disorders Awareness Month, which is observed annually in the month of March, the CGTLive® team has gathered some of our recent coverage of hemophilia gene therapies from the past few months below to offer a snapshot at some of the happenings related to therapies for these disorders. A notable trend has been the scaling back of production or discontinuation of approved products for these indications, but research and development of new products also remains ongoing.

4. Atsena Therapeutics’ X-Linked Retinoschisis Gene Therapy Snags Fast Track Designation

The FDA has granted fast track designation to Atsena Therapeutics' ATSN-201, an investigational adeno-associated virus (AAV) gene therapy, for the treatment of X-linked retinoschisis (XLRS). ATSN-201 is currently being evaluated in the phase 1/2 LIGHTHOUSE clinical trial (NCT05878860). This designation, along with the previously granted orphan Drug and rare pediatric disease designations, marks an important milestone in advancing the development of ATSN-201," Patrick Ritschel, MBA, the CEO of Atsena, said in a statement.

5. BMS Brings 2seventy bio Into the Fold

2seventy bio has signed an agreement to be purchased by its partner Bristol Myers Squibb (BMS). Currently, the companies own idecabtagene vicleucel (ide-cel, marketed as Abecma), an FDA-approved chimeric antigen receptor T-cell (CAR-T) therapy intended to treat multiple myeloma, together. “A year ago, 2seventy decided to exclusively focus on unlocking the value of Abecma, with the goal of delivering more time for people living with multiple myeloma and maximizing value for all stakeholders,” Chip Baird, MBA, the CEO of 2seventy bio, said in a statement. “The strategic rationale for this acquisition is clear and today’s announcement represents the culmination of the journey for 2seventy bio."

6. Longeveron's Lomecel-B Improves Cognitive Function

New data from the the phase 2a CLEAR MIND trial (NCT05233774), which is evaluating Longeveron's allogeneic bone marrow-derived medicinal signaling cell (MSC) formulation Lomecel-B (also known as laromestrocel) for the treatment of mild Alzheimer disease (AD), has been published in Nature Medicine. Among the findings reported were improvements in cognitive function, quality of life, and brain volume. “This Nature Medicine publication reinforces laromestrocel’s safety and efficacy as a potential treatment for mild AD and paves the way for more advances in utilizing cell therapy for AD,” Joshua Hare, MD, the founder and chief science officer of Longeveron, said in a statement.

7. AskBio Moves Onto Dosing Second Cohort in LGMD2I/R9 Gene Therapy Trial

Bayer subsidiary Asklepios BioPharmaceutical (AskBio) has dosed the first patient in the second cohort of the phase 1/2 LION-CS101 clinical trial (NCT05230459) evaluating AB-1003 (LION-101), its investigational AAV vector-based gene therapy intended to treat limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The patient's dosing comes after safety data from the trial's first cohort were reviewed by a data safety monitoring board, which gave a recommendation to proceed. The second cohort is using a higher dose level for the gene therapy than was used for the first cohort.