The cell and gene therapy landscape continues to evolve rapidly, marked by regulatory milestones, promising clinical data, and strategic industry consolidations. Several companies have recently announced significant developments in their respective pipelines.
Regulatory Updates
Abeona Therapeutics has resubmitted its Biologics License Application (BLA) to the FDA for prademagene zamikeracel (pz-cel; EB-101), an autologous gene-corrected epidermal sheet therapy, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). This follows a complete response letter (CRL) received earlier in the year. The resubmission aims to address the FDA's previous concerns and potentially bring this novel therapy closer to approval for patients with RDEB, a debilitating genetic skin disorder.
RiboX Therapeutics received FDA clearance for its Investigational New Drug (IND) application for RXRG001, a circular RNA therapy. This clearance enables a Phase 1/2a clinical trial, named the SPRINX-1 Study, in patients suffering from radiation-induced xerostomia (RIX) and hyposalivation, conditions that severely impact quality of life post-radiation therapy.
HuidaGene Therapeutics' HG202, a CRISPR/Cas13 RNA-editing therapy targeting neovascular age-related macular degeneration (nAMD), has also received IND clearance from the FDA, paving the way for a Phase 1 clinical trial. This represents a significant step forward in the development of gene editing therapies for ophthalmic diseases.
CARsgen Therapeutics announced the removal of clinical holds on trials for its BCMA-directed CAR-T therapy zevorcabtageneautoleucel, its Claudin18.2-directed CAR-T satricabtageneautoleucel (satri-cel), and its GPRC5D-directed CAR-T CT071. These holds, initially placed in late 2023 due to CMC concerns, have now been lifted, allowing the trials to proceed. Notably, the ELIMYN18.2 clinical trial is evaluating satri-cel in heavily pretreated Claudin18.2 (CLDN18.2)-positive advanced gastric/gastroesophageal (GC/GEJ) or pancreatic cancer (PC).
Clinical Trial Advancements
Nanoscope Therapeutics presented data from its Phase 2b RESTORE clinical trial (NCT04945772) at the American Academy of Ophthalmology 2024 Annual Meeting, demonstrating that MCO-010 (sonpiretigene isteparvovec), an ambient-light activatable multi-characteristic opsin (MCO) gene therapy, improved visual acuity in patients with retinitis pigmentosa (RP) compared to placebo. This suggests a potential new treatment option for RP, a group of inherited retinal diseases that lead to progressive vision loss.
Strategic Acquisitions and Collaborations
Lyell Immunopharma has entered into an agreement to acquire ImmPACT Bio, a company developing CD19/CD20-directed CAR-T therapy IMPT-314 for third-line large B-cell lymphoma patients who have not previously received CAR-T therapy. Following the acquisition, Lyell will prioritize the development of IMPT-314 and LYL119, its ROR1-targeted CAR T product, while discontinuing development for some of its other cell therapy candidates. This strategic move reflects Lyell's focus on advancing promising CAR-T therapies for hematological malignancies.
EvolveImmune and AbbVie have established a collaboration and option-to-license agreement. EvolveImmune will leverage its T-cell engager platform EVOLVE in conjunction with AbbVie's oncology expertise to develop 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," said Jonathon Sedgwick, PhD, the senior vice president and global head of discovery research at AbbVie.
