Around the Helix: Cell and Gene Therapy Company Updates – September 11, 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. Ractigen Garners Orphan Drug Designation for Muscular Dystrophy saRNA Therapy RAG-18

Ractigen Therapeutics has received orphan drug designation (ODD) from the FDA for RAG-18, its investigational small activating RNA (saRNA) that is currently in preclinical development for the treatment of cases of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy caused by any mutation in the DMD gene.

2. Biogen’s Nusinersen Shows Efficacy in SMA Compared With Sham Treatment at Experimental Higher Dose

Biogen’s nusinersen (Spinraza), a marketed antisense oligonucleotide therapy for the treatment of spinal muscular atrophy (SMA), has demonstrated efficacy compared with a sham treatment when evaluated at a higher dose than that approved for use by the FDA.

3. Cartesian Therapeutics Doses First Patient With mRNA CAR-T Descartes-15 in Trial for R/R Multiple Myeloma

Cartesian Therapeutics has dosed the first patient with Descartes-15, an investigational next-generation autologous mRNA-engineered chimeric antigen receptor T-cell therapy (CAR-T) therapy that targets B-cell maturation antigen (BCMA), in a phase 1 clinical trial (NCT06304636) evaluating the product for the treatment of relapsed/refractory (r/r) multiple myeloma (MM).

4. Allogeneic CAR-T Azer-Cel Achieves Complete Responses in Some Patients With Diffuse Large B-Cell Lymphoma

Azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, has demonstrated the ability to produce complete responses (CRs) in some patients with r/r diffuse large B cell lymphoma treated in a trial run by Imugene, which licensed the therapy from Precision BioSciences.

5. Novartis Licenses Another Capsid From Voyager

Novartis has licensed a novel capsid from Voyager Therapeutics for use in an undisclosed neurological target. Under the terms of the agreement, Voyager, which produced the capsid with its TRACER capsid discovery platform, will garner $15 million upfront and may receive up to $305 million in later payments. “Novartis is a global leader in gene therapy, and we view their continued selection of our IV-delivered, blood-brain barrier-penetrant capsids as strong validation of our TRACER capsid discovery platform,” Alfred W. Sandrock, Jr., MD, PhD, the chief executive officer of Voyager, said in a statement.

6. BridgeBio Pharma Garners FDA RMAT Designation for BBP-812

BridgeBio Pharma’s BBP-812, an investigational adeno-associated virus serotype 9 (AAV9) vector-based gene therapy intended to treat Canavan disease, has received regenerative medicine advanced therapy (RMAT) designation from the FDA. The gene therapy is currently being evaluated in the the phase 1/2 CANaspire clinical trial (NCT04998396), and the agency made its decision to grant the designation based on data from the study.

7. Genprex Announces Intent to Spin Out its Diabetes Gene Therapy Program to New Company

Genprex, which is currently developing gene therapy programs in both oncology and diabetes indications, has stated its intent to launch a separate company, to be dubbed NewCo, to handle continued development of its diabetes program. “Genprex’s diabetes program has received the attention of several potential investors, partners, collaborators, and pharmaceutical companies, and we believe separating it from our oncology programs could help increase interest, expedite clinical development and unlock the value of our novel diabetes program,” Ryan Confer, the president and chief executive officer of Genprex, said in a statement.

8. TransCode Snags NCI Grant for RNA Therapy Program in Solid Tumors

The National Cancer Institute (NCI) of the National Institutes of Health has awarded a $2 million Direct to Phase II Small Business Innovation Research grant to TransCode to support assessment of TTX-MC138, one of the company's investigational RNA therapies, in a phase 1/2 clinical trial for patients with advanced solid tumors.