Carmine Therapeutics and Takeda will collaborate to discover and develop non-viral Rare Disease Gene Therapies Using Carmine’s novel REGENT platform
U.S. headquartered Biotech startup Carmine Therapeutics has signed a $900 million research collaboration agreement with Takeda Pharmaceutical to discover, develop and commercialize transformative non-viral gene therapies for two rare disease targets, according to a company statement released on Tuesday.
Japanese multinational pharmaceutical and biopharmaceutical company Takeda has also committed a $5M convertible loan to support the development of Carmine’s novel REGENT platform.
According to the terms of the agreement, Takeda will provide an undisclosed amount as upfront payment to Carmine, offer research funding support, and over $900 million in total milestone payments in addition to tiered royalties.
Carmine has raised over $9.4 million in seed funding in an oversubscribed round led by Esco Ventures from Esco Ventures and Takeda Ventures, since its inception in August 2019.
Takeda can choose to license the programs developed by Carmine after the completion of pre-clinical proof-of-concept studies, and would be responsible for clinical development and commercialization, according to the statement.
The 10-month-old startup was founded by Esco Ventures X, serial biotech entrepreneur and MIT Professor Harvey Lodish, who is also the Chair of the Scientific Advisory Board of Carmine, and Professors Minh Le and Jiahai Shi.
The startup was incubated in Singapore by Esco Venture, the corporate venture and incubation arm of Esco Group. The startup is heaquartered in Cambridge, Massachusetts with its research arm based in Singapore.
Managing Partner of Esco Ventures and Founder and CEO of Carmine Therapeutics Lin Xiang Qian said, “This collaboration leverages Takeda’s industry leading rare diseases expertise and resources, and Carmine’s REGENT platform, to bring transformative non-viral gene therapies to patients.”
The latest funding will allow Carmine to further develop its REGENT platform, and advance its wholly-owned programs, he added.
According to the statement, Carmine is a new class of genetic medicines based on red blood cell extracellular vesicles (RBCEVs) as allogeneic therapies.
The new technology leverages RBCEVs to deliver nucleic acid payloads using extracellular vesicles, which is a natural way to transfer information between cells. The startup is currently focussing on non-viral gene therapies.
Developed by Le at the National University of Singapore and Shi Jiahai at the City University of Hong Kong, the foundation work for this novel technology was first published in Nature Communications in 2018, and aims to revolutionize nucleic acid therapies.
RBCEV-based gene therapy has the potential for repeat dosage, can be externally engineered, at scale, to carry large nucleic acid payloads of at least 11 kilobases, and surface functionalized with targeting ligands to increase bioavailability in specific tissues and cell types.
Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan said, “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.”
“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,” he added.
Takeda has also entered into a strategic partnership with Sandiego-headquartered biopharmaceutical company Neurocrine Biosciences, which develops treatments for nerve and endocrine-related diseases and disorders, according to a report by Global Legal Chronicle.
Header Image courtesy of Carmine Therapeutics