RARE Daily

Myeloid Enters Exclusive Gene-Editing Option and Research Collaboration with Prime Medicine

March 31, 2022

Rare Daily Staff

Rare cancer focused biotech Myeloid Therapeutics has entered into an exclusive option and research collaboration agreement with gene-editing biotech Prime Medicine focused on accelerating the development of Myeloid’s proprietary RNA-based, retrotransposon mediated gene-insertion technology, RetroT, which has applications in gene editing and delivery and may be complementary to Prime Editing. 

“At Myeloid, we have built one of the deepest RNA-based technology platforms focused on multiple therapeutic modalities, including our newest technology announced today for gene editing, RetroT,” said Daniel Getts, CEO of Myeloid. “This partnership highlights the broad applicability of our R&D engine to indications beyond cancer.”

Retrotransposons are genetic elements that replicate through reverse transcription of an RNA transposition intermediate. They contribute to structural changes and more importantly to gene regulation. Myeloid’s RetroT delivers genetic sequences and integration enzymes in a single mRNA strand. This breakthrough technology offers the potential to deliver gene-sized pieces of DNA into the genome, which are larger than currently possible with existing gene editing technologies. RetroT holds the potential to significantly expand the type and scope of genetic errors that can be reversed in situ. 

Myeloid and Prime will collaborate to optimize Myeloid’s RetroT technology, with the goal of extending its utility to multiple cell types, increasing its efficiency, and expanding its programmability so that it can be directed to pre-specified target sites within the genome. If successful, this technology could be used alongside prime editing to transform and vastly simplify the delivery of both ex vivo and in vivo cell therapies for the treatment of human diseases. 

“We are committed to expanding our gene editing toolbox with new, uniquely positioned approaches that may complement Prime Editing,” said Keith Gottesdiener, CEO of Prime Medicine. “We look forward to partnering with the extremely talented team at Myeloid – including many experts in retrotransposon biology – to advance this novel technology, with the goal of expanding the applicability of Prime Editing to more broadly address human diseases.”

Under the terms of the agreement, Myeloid received an upfront payment of $45 million. Myeloid is also eligible to receive significant milestone and option exercise payments and sales-based royalties. The companies will work together to address key elements required for translating Myeloid’s RetroT technology into human clinical products. Upon completion of these efforts, Prime will have the exclusive option to gain control of the intellectual property estate to expand upon its existing technology platform. 

Photo: Daniel Getts, CEO of Myeloid Therapeutics

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