Cell therapy weekly: partnership to advance gene therapy for ADA-SCID
This week: AGC Biologics (Milan, Italy) has partnered with Rarity PBC (CA, USA) to support the development of its gene therapy for Adenosine Deaminase Severe Combined Immunodeficiency Disorder (ADA-SCID), Aurion Biotech (WA, USA) announced 12-month results from its Phase I/II trial investigating its corneal cell therapy, and ME Therapeutics (Vancouver, Canada) secured an exclusive licensing agreement to use the National Research Council of Canada’s (NRC) proprietary nanobody-based CD22 binder in CAR-T therapies.
The news highlights:
- Partnership to advance gene therapy for ADA-SCID
- Positive 12-month results for corneal cell therapy
- Exclusive licensing deal for nanobody-based CD22 binder
Partnership to advance gene therapy for ADA-SCID
AGC Biologics has entered into a partnership agreement with Rarity PBC to support the development and manufacturing of RDP-101, a gene therapy designed to treat ADA-SCID. This collaboration represents a significant milestone, as RDP-101 could become the first commercially available gene therapy in the United States specifically targeting this rare genetic condition.
ADA-SCID is an extremely rare and potentially fatal inherited disorder that severely weakens the immune system in infants, making them highly susceptible to serious infections. The condition affects approximately one to five babies per million births worldwide and represents about 15 percent of all SCID cases. Without proper treatment, the disorder is typically fatal.
Rarity’s innovative RDP-101 therapy works by extracting a patient’s own hematopoietic stem cells, genetically modifying them outside the body, and then reintroducing them to restore normal immune function. Clinical trial results have been remarkably promising, successfully treating 48 out of 50 children who participated in the studies.
Under this comprehensive agreement, AGC Biologics will handle all aspects of the manufacturing process, including development, Good Manufacturing Practice production, and validation activities. The company will utilize its specialized ProntoLVV™ adherent platform technology, which has previously supported other commercially successful gene therapy products, to manufacture the lentiviral vector component of the treatment.
Positive 12-month results for corneal cell therapy
Aurion Biotech, has announced encouraging 12-month results from its Phase I/II CLARA clinical trial. The study evaluated AURN001, which combines human corneal endothelial cells with a rho-kinase inhibitor called Y-27632, for treating patients with corneal edema caused by corneal endothelial dysfunction.
The study demonstrated a clear dose-dependent response, with the highest dose group showing the most significant improvements. After 12 months, 65 percent of high-dose recipients achieved substantial vision gains of 15 letters or more, compared to zero percent in the control group. High-dose patients experienced an average improvement of 12.5 letters in visual acuity and reduced corneal thickness by 23.2 micrometers. Successful responders saw their vision improve from 20/60 to 20/25 on eye charts, while also reporting significant improvements in quality-of-life measures related to daily visual functioning.
Exclusive licensing deal for nanobody-based CD22 binder
ME Therapeutics has secured an exclusive licensing agreement with the NRC. The agreement grants ME Therapeutics commercial rights to use the NRC’s proprietary nanobody-based CD22 binder for use in CAR-T therapy, including in vivo CAR-T and myeloid CAR (CAR-M).
The nanobody technology offers several advantages over traditional antibody treatments, including better stability and more flexible design options. Currently, this CD22 binder is being tested in a Phase I clinical trial involving both pediatric and adult patients to evaluate its safety and effectiveness as part of a CAR-T therapy approach.
“We are pleased to license this Canadian-made, publicly-owned nanobody-based CD22 binder to a Canadian company for use in next-generation CAR applications,” says Sue Twine, Director General of the NRC’s Human Health Therapeutics Research Centre. “This technology was developed through years of dedicated NRC research, with support from our Cell and Gene Therapy Challenge program. It is a compelling example of publicly funded research enabling innovative next generation tools that can support development of affordable, cutting-edge treatments for Canadian patients.”