Carmine, Takeda Collaborate to Discover and Develop Rare Disease Gene Therapies

Carmine Therapeutics has signed a research collaboration agreement with Takeda to discover, develop and commercialize non-viral gene therapies for two rare disease targets using Carmine's REGENT technology, based on red blood cell extracellular vesicles. In addition, Takeda has committed a $5M 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 over $900M in total milestone payments plus tiered royalties. Takeda has an option to license the programs following the completion of pre-clinical proof of concept studies and would be responsible for clinical development and commercialization.

"We are pleased to enter this collaboration with Takeda, a recognized global leader in rare disease therapies, slightly more than a year since Carmine was created and incubated by Esco Ventures X. This provides Carmine with significant funding to further develop our REGENT(TM) platform, and advance our wholly-owned programs," XQ Lin, Founding CEO of Carmine Therapeutics and Managing Partner of Esco Ventures, said.

Carmine Therapeutics is developing a novel class of therapeutics based on its REGENT technology 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 Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan. "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."