Japanese pharmaceutical giant Takeda Pharmaceutical has entered into a research collaboration agreement with US-based Carmine Therapeutics for the discovery, development and commercialisation of rare disease therapies.

The partners intend to leverage Carmine’s REGENT technology, based on red blood cell extracellular vesicles (RBCEVs), to develop non-viral gene therapies for two rare disease targets.

RBCEV-based gene therapy is said to have multiple benefits when compared to adeno-associated virus (AAV)-based gene therapy, including potential for repeat dosing and larger transgene payload capacity.

Takeda also agreed to provide a $5m convertible loan to support the development of Carmine’s REGENT platform.

Carmine Therapeutics founding CEO XQ Lin 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 platform, and advance our wholly-owned programmes.”

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Under the deal, Takeda will make an upfront payment and provide research funding support to Carmine, which is eligible to receive more than $900m in milestone payments plus tiered royalties.

The agreement provides Takeda with an option to licence the programmes after completion of pre-clinical proof of concept studies and would carry out clinical development and commercialisation.

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.”

In March, Takeda signed a multi-target alliance with biotechnology company Evox Therapeutics to work on protein replacement and mRNA therapies for rare diseases.