Around the Helix: Cell and Gene Therapy Company Updates – September 18, 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. ProKidney Puts Focus on US Trial for T2D and CKD Cell Therapy Rilparencel

ProKidney has announced a new strategic focus on its phase 3 REGEN-006 (PROACT 1) clinical trial (NCT05099770), which is evaluating rilparencel (also known as REACT), a renal autologous cell therapy, in patients in the United States who have type 2 diabetes (T2D) and chronic kidney disease (CKD).

2. Oncternal Therapeutics Axes B-Cell Lymphoma Trial for CAR-T Therapy ONCT-808

Oncternal Therapeutics has made the decision to discontinue its phase 1/2 ONCT-808-101 clinical trial (NCT05588440) evaluating ONCT-808, an investigational chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of relapsed/refractory aggressive B-cell lymphoma.

3. Vironexis Biotherapeutics’ AAV Vector-Based Immunotherapy VNX-101 Cleared for US Trial in Acute Lymphoblastic Leukemia

Vironexis Biotherapeutics has received clearance of its investigational new drug application from the FDA for VNX-101, an adeno-associated virus (AAV) vector-based gene therapy, enabling a phase 1/2 clinical trial in CD19+ acute lymphoblastic leukemia.

4. BridgeBio Pharma Drops Development of Congenital Adrenal Hyperplasia Gene Therapy BBP-631

BridgeBio Pharma has made the decision to discontinue development of BBP-631, an investigational AAV 5 vector-based gene therapy that was being evaluated in the phase 1/2 ADventure clinical trial (NCT04783181) for the treatment of congenital adrenal hyperplasia.

5. HAYA Therapeutics and Lilly Team Up to Tackle Metabolic Diseases

Under a new collaboration agreement, the 2 companies will apply HAYA's regulatory genome discovery platform for the purpose of identifying targets for RNA-based therapeutics aimed at treating chronic metabolic conditions, including obesity. The platform is specifically geared towards identification of disease-relevant long noncoding RNA sequences and creation of associated targeted therapeutics.

6. eGenesis Snags $191 Million for Genetically Modified Porcine Kidney Transplant Solution

The funds were raised in a round of Series D financing following the company's performance of the first successful xenotransplant of a porcine kidney into a human patient, which was carried out under the FDA's Expanded Access pathway, earlier this year. eGenesis will use the funding for further development of EGEN-2784, a porcine kidney that features a number of genetic modifications intended to prevent rejection after transplant into human patients.

7. Voyager Therapeutics to Receive $3 Million Milestone Payment Following Selection of Capsid for Further Development by Neurocrine

Neurocrine Biosciences, via selection by a collaborative joint steering committee, 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 receive a $3 million milestone payment later this year. The capsid is the third to be selected as part of an ongoing collaboration between the 2 companies.

8. Atsena Therapeutics Garners Orphan Drug Designation for X-linked Retinoschisis Gene Therapy

The FDA has granted orphan drug designation to Atsena Therapeutics’ ATSN-201, an investigational AAV gene therapy, for the treatment of X-linked retinoschisis associated with mutations in the RS1 gene. “We are honored to receive the FDA’s Orphan Drug Designation for ATSN-201, which was also recently granted Rare Pediatric Disease designation," Patrick Ritschel, MBA, the CEO of Atsena Therapeutics, said in a statement. "These designations mark a significant inflection point for the potential of this ocular gene therapy in an inherited retinal disease that currently has no available treatments."