Around the Helix: Cell and Gene Therapy Company Updates – March 19, 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. Longeveron's Cell Therapy Lomecel-B Improves Cognitive Function in Mild Alzheimer Disease

New data from the now-completedphase 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), have been published in Nature Medicine.

2. SpliceBio Doses First Patient in Trial for Stargardt Gene Therapy SB-007

Barcelona-based genetic medicines company SpliceBio has dosed the first patient in the phase 1/2 ASTRA clinical trial (NCT identifier pending), which is evaluating SB-007, a dual adeno-associated virus (AAV) vector-based gene therapy intended to treat Stargardt disease.

3. Patient Dies of Acute Liver Failure After Treatment With Sarepta’s DMD Gene Therapy Elevidys

A patient has died of acute liver failure (ALF) after being treated with Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), a marketed adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD).

4. Novartis’ OAV101 IT Improves or Stabilizes Motor Function in Patients With Spinal Muscular Atrophy

Novartis’ OAV101 IT, an investigational intrathecally-delivered version of the marketed gene therapy onasemnogene abeparvovec (Zolgensma), has demonstrated the ability to improve patient scores on the Hammersmith Functional Motor Scale Expanded (HFMSE). The data, which come from the phase 3 STEER clinical trial (NCT05089656), were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 16-19 in Dallas, Texas.

5. BMS's Liso-Cel Approved for R/R Follicular Lymphoma in European Union

Bristol Myers Squibb (BMS)’s lisocabtagene maraleucel (liso-cel, marketed as Breyanzi), an autologous CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy, has received approval from the European Commission for an expanded indication for relapsed/refractory (r/r) follicular lymphoma in patients who have previously been treated with at least 2 lines of systemic therapy. The decision was based on data from the phase 2 TRANSCEND FL clinical trial (NCT04245839). Notably, liso-cel had previously received accelerated approval from the FDA in May 2024 for treating adults with r/r follicular lymphoma who have received 2 or more prior lines of systemic therapy.

6. Arbor Biotechnologies Rakes in $73.9 Million for Gene Editing Therapies

Arbor Biotechnologies has received $73.9 Million in a round of Series C financing. The funds will be put towards advancing the development of its gene editing therapies, including its lead candidate, ABO-101. ABO-101 is a lipid nanoparticle (LNP)-delivered Cas12i2-based gene editing approach currently being assessed in a clinical trial for the treatment of the rare genetic disease primary hyperoxaluria type 1.

7. Cellares and Cabaletta Bio Make Progress in Manufacturing Partnership

Cabaletta Bio and Cellarres have successfully manufactured multiple batches of resecabtagene autoleucel, (rese-cel, previously known as CABA-201), Cabaletta’s investigational CAR-T that is intended to treat autoimmune diseases, with the use of Cellares' Cell Shuttle, an automated manufacturing platform. “Through our partnership with Cellares, our teams have successfully achieved proof of concept for the ability to automate the rese-cel cellular drug substance manufacturing process," Gwendolyn Binder, PhD, the president of science and technology at Cabaletta Bio, said in a statement. "I believe that the potential increase in capacity, meaningful reduction in costs, and rapid global technology transfer offers a potential solution for the global scale out of rese-cel for patients with autoimmune disease."