Around the Helix: Cell and Gene Therapy Company Updates – January 29, 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. First Patient Dosed in Phase 3 Trial for Intellia Therapeutics’ Hereditary Angioedema Gene Editing Therapy NTLA-2002

The first patient has been dosed in Intellia Therapeutics’ pivotal phase 3 HAELO clinical trial (NCT06634420) evaluating NTLA-2002, an investigational CRISPR/Cas9-based gene-editing therapy that is delivered systemically as a single-dose, for the treatment of hereditary angioedema (HAE).

2. AskBio Makes Progress in Clinical Evaluation of Parkinson Disease Gene Therapy AB-1005

Asklepios BioPharmaceutical(AskBio) has begun the process of randomly assigning enrolled participants in the sham-controlled phase 2 REGENERATE-PD clinical trial (NCT06285643) evaluating AB-1005 (AAV2-GDNF), an investigational adeno-associated virus vector serotype 2 (AAV2) gene therapy, for the treatment of Parkinson disease (PD).

3. Allogene Therapeutics’ ALLO-329 Cleared by FDA for Phase 1 Clinical Trial in Rheumatology Indications

Allogene Therapeutics’ ALLO-329, an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, has received clearance of an investigational new drug (IND) application from the FDA for a phase 1 rheumatology basket study.

4. Sarepta’s DMD Gene Therapy Elevidys Shows Sustained Benefit in Patients Who are Ambulatory

Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), a marketed adeno-associated virus (AAV) vector-based gene therapy for patients with Duchenne muscular dystrophy (DMD), has shown sustained benefit in patients who are ambulatory who were treated in the phase 3 EMBARK clinical trial (NCT05096221), according to new results from part 2 of the study.

5. Atalanta Therapeutics Rakes in $97 Million

The funding, which the company will put towards phase 1 clinical trials for 2 of its RNA interference (RNAi) therapies, was obtained in a round of Series B financing coled by EQT Life Sciences and Sanofi Ventures. “Importantly, this Series B will support a path to the clinic for 2 programs for serious neurological diseases that today lack disease-modifying therapies—KCNT1-related epilepsy and Huntington disease—and will anchor our growing franchise of investigational medicines for Huntington disease," Alicia Secor, MBA, the president and CEO of Atalanta, said in a statement. "We are diligently progressing these programs toward IND submissions next year so that we can start our phase 1 trials and reach patients who are waiting.”

6. March Biosciences Garners FDA Orphan Drug Designation for CD5-Directed CAR-T MB-105

March Biosciences' MB-105, an investigational CD5-directed chimeric antigen receptor T-cell (CAR-T) therapy being evaluated in an ongoing phase 1 clinical trial (NCT03081910) for relapsed/refractory (r/r) CD5-positive T-cell lymphoma, has received orphan drug designation from the FDA. "The MB-105 Phase 1 trial has shown promising safety and efficacy signals in r/r T-cell lymphoma patients," Sarah Hein, PhD, the cofounder and CEO of March Biosciences, said in a statement. "This designation further validates our development strategy as we prepare to initiate our phase 2 clinical trial in early 2025.”