Around the Helix: Cell and Gene Therapy Company Updates – October 30, 2024

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. Sangamo Reaches Accord With FDA on Plans for Accelerated Approval Pathway for Fabry Disease Gene Therapy ST-920

Sangamo Therapeutics has emerged from a Type B interaction with the FDA having come into alignment with the agency on plans to pursue an accelerated approval pathway for isaralgagene civaparvove (ST-920), an investigational adeno-associated virus vector-based gene therapy product intended to treat Fabry disease.

2. Ocugen Gets Green Light to Move Onto Phase 2 Portion of Trial Evaluating Stargardt Gene Therapy OCU410ST

Ocugen has received clearance from a Data and Safety Monitoring Board (DSMB) to move onto the phase 2 portion of the phase 1/2GARDian clinical trial (NCT05956626), which is evaluating OCU410ST, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of Stargardt disease.

3. Intellia’s CRISPR Gene Editing Therapy NTLA-2002 Appears to Eliminate Hereditary Angioedema Attacks in Some Patients Following One-Time Treatment

Intellia Therapeutics’ NTLA-2002, an investigational CRISPR/Cas9-based gene-editing therapy that is delivered systemically as a single-dose and is being evaluated in a phase 1/2 clinical trial (NCT05120830) for the treatment of hereditary angioedema (HAE), has eliminated HAE attacks in some patients through their most recent follow-up, according to data the company is presenting at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting, held October 24 to 28, in Boston, Massachusetts.

4. Immatics’ TCR-T Therapy Shows Good Phase 1 Safety and Survival Data, Phase 3 Plans Confirmed

Treatment with the investigational TCR-based autologous cell therapy, ACTengine IMA203 (Immatics), has been shown to demonstrate a response rate exceeding 50% while maintaining a favorable tolerability profile across all dose levels tested, according to newly presented data. Additionally, the company reported progression-free survival (PFS) and overall survival (OS) data for the first time among patients with melanoma in the trial.

5. March Biosciences Rakes in $28.4 Million to Support Development of CD5-Directed CAR-T

The funds, which the company raised in a Series A round of financing, will be put towards carrying out a phase 2 clinical trial in relapsed/refractory CD5-positive T-cell lymphoma for MB-105, the company's investigational autologous CD5-directed chimeric antigen receptor T-cell (CAR-T) therapy. Notably, March Biosciences also announced that it has partnered with the cell therapy venture studio Volnay Therapeutics to assist with establishing scalable manufacturing for MB-105.

6. Abeona Therapeutics Resubmits BLA to FDA for Epidermolysis Bullosa Gene Therapy Pz-Cel

Following the complete response letter (CRL) that the company received from the FDA earlier this year for the biologics license application (BLA) that the company submitted to the agency for prademagene zamikeracel (pz-cel; EB-101), an investigational autologous gene-corrected epidermal sheet therapy for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB), Abeona has resubmitted the BLA for the therapy. “We have incorporated the agency’s feedback and are confident that our resubmission package addresses all the Chemistry Manufacturing and Controls items identified in the CRL, including observations from the completed Pre-License Inspection of our manufacturing facility," Vish Seshadri, the chief executive officer of Abeona, said in a statement.

7. Capsida Biotherapeutics Garners FDA Orphan Drug Designation for Encephalopathy Gene Therapy CAP-002

Capsida Biotherapeutics has received orphan drug designation from the FDA for CAP-002, an investigational gene therapy intended to treat developmental and epileptic encephalopathy caused by syntaxin-binding protein 1 (STXBP1) mutations. CAP-002, which is currently in investigational new drug application (IND)–enabling studies, utilizes a capsid that has been engineered by the company with the intent of enabling brain-wide neuronal expression, while cutting down on tropism for the cells of the liver seen in many AAV capsids used for gene therapy products.

8. RiboX's Circular RNA Therapy RXRG001 Cleared for Phase 1/2a Trial in Radiation-Induced Xerostomia and Hyposalivation

RiboX Therapeutics has received clearance of an IND application from the FDA for RXRG001, an investigational circular RNA therapy, enabling a phase 1/2a clinical trial that the company will refer to as the SPRINX-1 Study, in patients with radiation-induced-xerostomia and hyposalivation. RiboX stated that this is the first IND clearance the FDA has granted for any circular RNA therapy.