Cell therapy weekly: collaborations advancing ophthalmology, colorectal cancer and non-viral cell therapy manufacturing
This week: strategic partnerships have been announced, with one partnership aiming to develop and commercialize genetic medicines for ophthalmology and a late-stage company strengthening its solid tumor pipeline by acquiring a CAR-T therapy for colorectal cancer. Plus, Kytopen Corp. (MA, USA) and Excellos Inc. (CA, USA) are in talks to advance non-viral cell therapy manufacturing.
The news highlights:
- Gene therapy partnership to support and advance ophthalmology treatment
- Acquisition of promising CAR T-cell therapy for colorectal cancer
- Partnership exploring non-viral cell therapy manufacturing technologies
Gene therapy partnership to support and advance ophthalmology treatment
MeiraGTx (London, UK) has partnered with Eli Lilly and Company (IN, USA) to develop and commercialize genetic medicines for eye diseases. Under the deal, Lilly receives worldwide exclusive rights to MeiraGTx’s AAV-AIPL1 gene therapy, which treats Leber congenital amaurosis 4 (LCA4) – a severe inherited blindness condition. The one-time subretinal injection delivers functional genes to restore vision in pediatric patients, with early 2025 trial results showing all 11 children born blind gained vision after treatment.
The collaboration extends beyond AAV-AIPL1, giving Lilly access to MeiraGTx’s broader gene therapy platform for ophthalmology, including novel delivery systems, AI-generated promoters, and riboswitch technology that allows precise control of therapeutic proteins through oral medications. Alexandria Forbes, president and CEO of MeiraGTx stated:
“We are excited to be entering into this collaboration with Lilly in ophthalmology. MeiraGTx has been dedicated to treating patients with serious eye conditions since the company’s inception, and this collaboration with Lilly is a testament to our leadership in this field and the power of our broad toolkit of proprietary gene therapy technologies for both rare and prevalent ocular disease.”
Financially, MeiraGTx receives US$75 million up front, with potential milestone payments exceeding US$400 million, plus tiered royalties on licensed products. Beyond vision restoration, the treatment showed life-changing benefits in communication, behavior, learning and social integration for treated children.
Acquisition of promising CAR T-cell therapy for colorectal cancer
Lyell Immunopharma (CA, USA) has acquired global rights to LYL273, a novel CAR T-cell therapy targeting metastatic colorectal cancer and other guanylyl cyclase-C (GCC)-expressing cancers, from Innovative Cellular Therapeutics (MD, USA). The deal includes US$40 million up front plus 1.9 million Lyell shares, with potential additional payments totaling up to US$820 million in milestones and up to 1.85 million more shares.
LYL273 has shown promising early results in patients with refractory metastatic colorectal cancer, achieving a 67% overall response rate and 83% disease control rate in a Phase I US trial with manageable safety at the highest dose level to date. The therapy is enhanced with CD19 CAR expression and controlled cytokine release, designed to improve CAR T-cell expansion, immune cell infiltration and cancer cell killing in the hostile solid tumor microenvironment.
“We rarely see such deep and durable responses in colorectal cancer patients treated with multiple prior lines of chemotherapy. The outcomes in this initial cohort of heavily pre-treated patients are very encouraging,” said Benjamin L. Schlechter, lead investigator in the Phase I clinical trial. “Patients with metastatic colorectal cancer have a tremendous need for innovations like LYL273, and I look forward to partnering with the Lyell team as we work to rapidly deliver on the potential of this innovative cellular therapy for patients with advanced colorectal cancer.”
Partnership exploring non-viral cell therapy manufacturing technologies
Kytopen and Excellos are exploring a collaboration to advance non-viral cell therapy manufacturing technologies. The partnership will assess how Kytopen’s Flowfect Tx® platform integrates with Excellos’ donor-to-dose manufacturing model and deep cell characterization capabilities. Together, the companies aim to enhance process consistency, scalability and overall cell quality in developing next-generation cell-based therapies
“Kytopen’s Flowfect® technology represents an exciting advancement in non-viral cellular engineering, and we’re eager to explore how it could complement our donor-to-dose expertise,” said Tom VanCott, CEO of Excellos.
