Gene-editing pioneers launch genetic medicines company

Wednesday, 16 May, 2018

Gene-editing pioneers launch genetic medicines company

Scientific pioneers David R Liu, Feng Zhang and J Keith Joung have announced the launch of Beam Therapeutics, said to be the first company to pursue the development of new therapies using CRISPR base editing technology.

Beam Therapeutics will build on its co-founders’ work advancing CRISPR gene editing, developing precision genetic medicines that make edits to individual base pairs in the genetic code. The company’s research will focus on multiple DNA base editor platforms developed in Liu’s lab at Harvard University, as well as on the RNA base editor platform developed by Zhang at the Broad Institute of MIT and Harvard.

DNA is made up of billions of nucleobases (bases), each represented by a single letter (A, G, T, C), which are subsequently encoded in RNA messages for expression by the cell. Base editors are capable of precisely targeting and directly editing just one base out of billions within the genome, without cutting the DNA or RNA. Beam aims to use these technologies to generate a broad pipeline of precision genetic medicines that repair disease-causing point mutations, write in protective genetic variations or modulate the expression or function of disease-causing genes.

“Base editors are capable of making single-base changes with high efficiency and unprecedented control,” said Beam CEO John Evans. “Beam has assembled the key technologies in base editing and is dedicated to establishing base editors as a new therapeutic option for patients with serious diseases.”

Beam’s first licence agreement is with Harvard University for base editing technologies in the field of human therapeutics. This licence covers two base editing platforms developed in Liu’s lab in Harvard’s Department of Chemistry and Chemical Biology. The first is the C base editor (‘BE’), originally published in Nature in 2016, which features Cas9 linked to a cytidine deaminase to deliver programmable C-to-T or G-to-A edits in DNA. The second is the A base editor (‘ABE’), published in Nature in 2017, which features Cas9 linked to an evolved form of adenosine deaminase capable of editing DNA to deliver programmable A-to-G or T-to-C edits.

In a second agreement with the Broad Institute, Beam is able to acquire RNA base editing technologies from Zhang’s lab. This includes the RNA editor platform (‘REPAIR’), first published in Science in 2017, which features Cas13 linked to an adenosine deaminase to deliver single base A-to-G editing of RNA transcripts.

Both licences provide an initial period of exclusivity for human therapeutic use, after which time there is a mechanism to extend a licence to others on an individual gene target basis if the technology is not being actively developed for that target.

Finally, Beam has entered into a licensing and option agreement with Editas Medicine for exclusive rights to certain intellectual property licensed to Editas Medicine by Harvard, the Broad Institute and Massachusetts General Hospital (MGH), as well as to certain Editas Medicine technologies. Under this agreement, Beam has received an exclusive sublicence to patent filings by Harvard for base editing technologies developed in the Liu Lab and patent filings by MGH for CRISPR technology developed in the Joung Lab, as well as an exclusive option for future sublicensing of additional Cas9 patent families and Cpf1 patent families in the field of base editing. In return, Editas Medicine has received an equity stake in Beam and will be eligible for royalties on medicines utilising the related intellectual property and technologies.

Beam is launching with up to US$87 million in cumulative Series A funding for Beam and an affiliated entity, led by biotech venture capital firms F-Prime Capital Partners and ARCH Venture Partners. According to Robert Nelsen, Managing Director and Co-Founder of ARCH Venture Partners, Beam is “uniquely positioned to change how we treat and potentially even prevent diseases” thanks to its next-generation gene editing technology, world-class team and significant resources.

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