Carmine and Takeda Collaborate on Rare Disease Gene Therapies

Carmine Therapeutics and Takeda Pharmaceutical entered into a research collaboration to discover, develop, and commercialize transformative non-viral gene therapies for two rare disease targets using Carmine’s technology.

In addition, Takeda has committed a $5 million convertible loan in support of the development of Carmine’s novel Regent platform.

Under the terms of the agreement, Carmine will receive an upfront payment, research funding support, and is eligible for more than $900 million in total milestone payments plus tiered royalties. Takeda has an option to license the programs following the completion of preclinical proof of concept studies and would be responsible for clinical development and commercialization.

Carmine’s CEO XQ Lin says the deal provides funding to advance its wholly-owned programs and further develop its proprietary Regent platform, which leverages red blood cell extracellular vesicles (RBCEVs), first published in Nature Communications in 2018. An initial focus is non-viral gene therapies. Compared to adeno-associated virus (AAV)-based gene therapy, RBCEV-based gene therapy has the potential for repeat dosing, a significantly larger transgene payload capacity in excess of 11KB, and enhanced bio-distribution in selected tissues through RBCEV surface modification.

“At Takeda, we’re expanding our foundation in gene therapy by establishing a network of innovative partners like Carmine Therapeutics who are pursuing non-viral vector approaches that could leapfrog today’s technology,” said Madhu Natarajan, head of Takeda’s Rare Diseases Drug Discovery unit. “Developing alternative gene therapy delivery vehicles like the Regent platform that could address the challenges of AAV gene therapy is critical to one day delivering next-generation cures for rare diseases.”

Photo: Madhu Natarajan, head of the Rare Diseases Drug Discovery Unit at Takeda

Author: Rare Daily Staff