Around the Helix: Cell and Gene Therapy Company Updates – November 6, 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. Abeona Therapeutics Resubmits BLA to FDA for Epidermolysis Bullosa Gene Therapy Pz-Cel

Following the complete response letter (CRL) that Abeona Therapeutics 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.

2. 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 (RIX) and hyposalivation.

3. Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial

Nanoscope Therapeutics’ MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy, has improved visual acuity in patients with retinitis pigmentosa (RP) in comparison to patients who received a placebo in data from the phase 2b RESTORE clinical trial (NCT04945772). The data were presented at the American Academy of Ophthalmology 2024 Annual Meeting, held October 18 to 21 in Chicago, Illinois.

4. HuidaGene Therapeutics’ CRISPR/Cas13 RNA-Editing Therapy HG202 Gets Greenlight for US Trial in Macular Degeneration

HuidaGene Therapeutics’ HG202, an investigational CRISPR/Cas13 RNA-editing therapy intended to treat neovascular age-related macular degeneration (nAMD), has received clearance of an investigational new drug (IND) application from the FDA, enabling a phase 1 clinical trial.

5. FDA Removes Clinical Holds Previously Placed on 3 of CARsgen's Cell Therapies

The FDA has removed clinical holds from trials for CARsgen's BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy zevorcabtageneautoleucel, its Claudin18.2-directed CAR-T satricabtageneautoleucel(satri-cel), and its GPRC5D-directed CAR-T CT071. CGTLive has previously provided coverage of the ELIMYN18.2 clinical trial, which is evaluating satri-cel in heavily pretreated Claudin18.2 (CLDN18.2)-positive advanced gastric/gastroesophageal (GC/GEJ) or pancreatic cancer (PC). The holds were originally placed in late 2023.

6. Lyell Immunopharma Bringing ImmPACT Bio Into the Fold

Lyell Immunopharma has agreed to purchase ImmPACT Bio, which is developing CD19/CD20-directed CAR-T therapy IMPT-314 for the treatment of patients with third line large B-cell lymphoma who have not previously received CAR-T. After the acquisition Lyell will continue to prioritize the development of IMPT-314 and LYL119, its ROR1-targeted CAR T product. On the other hand, Lyell announced that it will discontinue development for some of its other cell therapy candidates.

7. AbbVie and EvolveImmune Team Up to Tackle Various Cancers With T-Cell Engager Platform

Under a new collaboration and option-to-license agreement, EvolveImmune will apply its T-cell engager platform EVOLVE in conjunction with AbbVie's expertise in oncology with the aim of developing therapeutic antibodies for both blood cancers and solid tumors. "AbbVie is dedicated to advancing the understanding of devastating diseases like cancer and investing in groundbreaking technologies and therapeutic platforms, to deliver novel treatments for patients with high unmet needs," Jonathon Sedgwick, PhD, the senior vice president and global head of discovery research at AbbVie, said in a statement. "We are excited to collaborate with the talented team at EvolveImmune to further advance their novel T-cell engager platform technology."