Japanese pharma and biotech friend Takeda is set to spend nearly $1 billion in backloaded payments for a deal with early-stage startup Carmine Therapeutics.

The pair will target rare diseases using gene therapies out of the biotechs red blood cell extracellular vesicles (RBCEVs) platform, which it calls Regent and was first published in Nature back in 2018 (with the startup officially launching a year later).

An initial focus is non-viral gene therapies rather than the traditional path of adeno-associated virus (AAV)-based gene therapies that can lead to unwanted side effects.

De-risking the Development of Biotherapeutics Using Early Stage In Vitro Expression and Genetic Characterisation Tools

There is a high attrition rate during the development of biotherapeutics impacting the high cost of development. Early identification of the preferred expression host for manufacturing, along with lead candidate screening and material supply can help to reduce both attrition rates and cost.

The biotech said its 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.

The deal could be worth as much as $900 million, with Takeda leading Carmine's convertible note seed plus funding round by extending a $5 million convertible loan to the biotech. Theres an upfront payment and extra research funding cash, though details of dollar amounts here were not given.

Takeda nabs an option to license the programs following the completion of preclinical proof of concept studies; from here, it would be responsible for clinical development and sales.

Carmine Therapeutics was founded last year by Esco Ventures X, a life science fund, professor Harvey Lodish of the Whitehead Institute at MIT and Singaporean professors Minh Le and Jiahai Shi.

Lodish was a founding member of several big biotechs, including Genzyme (which was snapped up by Sanofi).

XQ Lin, founding CEO of Carmine Therapeutics and managing partner of Esco Ventures, said: 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.

This also marks another, albeit very early venture into gene therapies for Takeda. In April, German CRO-biotech hybrid Evotec allied with Takeda to expand into gene therapy research. The move saw Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.

Takeda acquired a gene therapy center in Orth an der Donau, Austria, through its purchase of Shire, which picked up the site two years earlier in its takeover of Baxalta. Throughout the series of changes in ownership, which began when Baxter spun out Baxalta in 2014, a team at the site has worked on gene therapies.

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, added Takeda's rare disease 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.

Excerpt from:
Takeda pens $900M biobucks pact with Carmine for non-viral gene therapy work - FierceBiotech

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