Takeda, Carmine Ink $900 Million Pact for Non-Viral Gene Therapy
By Ruchi Jhonsa, Ph.D.
Gene therapy has long-held promise to correct a variety of genetic diseases and defects. However, currently, these therapies do not apply to all organs and are limited by inherent targeting biases of current delivery vehicles.
In 2018, a group of scientists published a study that harnessed a natural mechanism used by the cells for exchanging RNA or communicating with other cells. This mechanism is based on extracellular vesicles (EV) that were first discovered in mast cells as a means of transporting microRNA and mRNA in between the cells and were later discovered in other cell types, including red blood cells.
Extracellular vesicles are formed by budding of the plasma membrane and play a significant role in the transfer of cell receptors, cytokine release, and genetic transfer. As they are found naturally in the body, it is believed that they will be able to overcome most cellular barriers and drug delivery hurdles such as cytotoxicity and immunogenicity found in classical delivery methods. Very recently, the effectiveness of this strategy was demonstrated in the delivery of silencing RNA to cancer cells, neurons, and blood cells.
This strategy formed the foundation of a newbie in the biotech industry, Carmine Therapeutics. After its launch in 2019, the company started working on its published technology that utilizes red blood cell extracellular vesicles as a delivery vehicle for gene editing cargo. EVs produced by RBCs are biocompatible, readily available, and are highly amenable to genetic manipulations. Looking at the potential of the delivery vehicle, Takeda pharmaceutical signed a research collaboration with the company to discover, develop and commercialize gene therapies for two rare disease targets using Carmine’s REGENT technology short for Red Cell EV Gene Therapy.
XQ Lin, Founding CEO of Carmine Therapeutics and Managing Partner of Esco Ventures, commented, “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 programs.”
Under the agreement, Takeda is offering an undisclosed upfront payment and research funding support to Carmine along with an over $900 million in milestone payments and tiered royalties. In addition, it is offering a $5 million loan for the development of the REGENT platform that, depending upon the companies can either be repaid or converted to equity at a future date. After the completion of preclinical studies and depending upon the success of the programs, Takeda can also license the programs. If that happens, Takeda will be responsible for the clinical development and commercialization of the programs.
Takeda’s Expansion in Gene Therapy Arena
Takeda’s biggest gene therapy deal happened last year when it acquired Shire for a whopping $62 billion. With the acquisition, it got hold of Shire’s diverse mid- and late-stage pipeline, enriched in gene therapy programs along with a gene therapy center in Orth an der Donau in Austria. Takeda also entered the deal with Germany based Evotec to support its growing research stage gene therapy discovery programs last year. The alliance leverages Evotec’s growing gene therapy capabilities and its broader drug discovery platform.
This year the pharma giant made a deal with Codexis to research and create gene therapies for rare disorders, including lysosomal storage disorders and blood factor deficiencies. Overall, the company is progressing in the field, but it is still behind other players who already have their drugs in clinical trials. Takeda’s hemophilia A program is currently in Phase 1, with the hemophilia B candidate about to join it in human testing- well behind the leaders.
“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.”
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