Cell therapy weekly: Fast Track designation for solid tumor CAR-T therapy
This week: Ray Therapeutics (CA, USA) received Regenerative Medicine Advanced Therapy (RMAT) designation for its gene therapy candidate for retinitis pigmentosa, A2 Biotherapeutics (CA, USA) received Fast Track designation for its CAR-T therapy for solid tumors, and LumaCyte’s (VA, USA) analytical method was included in the newly published ISO global standard for gene delivery systems.
The news highlights:
RMAT designation for retinitis pigmentosa gene therapy
The FDA has granted RMAT designation to Ray Therapeutics’ lead candidate for the treatment of retinitis pigmentosa, RTx-015. The therapy utilizes an AAV.7m8 viral vector, delivered as a single intravitreal injection, to introduce a gene encoding a highly light-sensitive protein to retinal ganglion cells, aiming to restore visual function.
“Receiving RMAT designation is a critical step as we move along the continuum of progress toward bringing hope to patients with advanced vision loss,” said Paul Bresge, CEO and Co-Founder of Ray Therapeutics. “Our mission has always been to restore functional sight. This designation will help us work more closely with the FDA to accelerate the delivery of this potentially transformative therapy to the patient community.”
Fast Track designation for solid tumor CAR-T therapy
A2 Biotherapeutics has been granted Fast Track designation by the FDA for its CAR-T therapy A2B543, which is currently under investigation in Arm 2 of the EVEREST-2 clinical study.
A2B543 is a specialized therapy using engineered autologous Tmod™ cells. These cells include a CAR activator targeting MSLN (a tumor marker), a blocker targeting HLA-A*02 to protect healthy cells, and a membrane-tethered IL-12 booster that activates only when tumor antigens are detected. The booster enhances the therapy’s effectiveness and durability while minimizing the risks of widespread IL-12 toxicity.
“Receiving Fast Track designation for A2B543 from the US FDA is a key milestone for A2 Bio in accelerating this promising precision CAR T-cell therapy for cancer patients,” said Jim Robinson, CEO of A2 Bio. “We are deeply committed to patients facing cancer and other devastating diseases, and we believe in the potential for A2B543 to address unmet needs in cancer therapy.”
Analytical method included in global standard for gene delivery systems
LumaCyte announced that the analytical principle underlying its Radiance® platform technology, optical and fluidic force-based measurements, has been included in the newly published International Organization for Standardization (ISO) global standard for gene delivery systems.
“Precision in viral vector quantification is foundational to patient safety, product comparability, and a stable supply chain,” said Renée Hart, President and Chief Business Officer of LumaCyte. “The inclusion of optical and fluidic force measurements in this ISO standard reflects the industry’s growing need for standardized, advanced analytics to improve process control, reduce variability, and support confident decision‑making from development through commercial manufacturing.”
The full ISO standard, ISO 16921‑2:2026: Biotechnology — Gene delivery systems — Part 2: Quantification methods for viral vectors, is available through the ISO website: https://www.iso.org/standard/86188.html