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Biotech Partners on Gene Editing Tech in $1.35B Deal

Gene therapy graphic

(Genome Research Limited, Flickr)

10 Jan. 2022. A biotechnology company is making its precise gene editing technology available to drug maker Pfizer for rare diseases, in a deal valued at $1.35 billion. Beam Therapeutics Inc. in Cambridge, Massachusetts and Pfizer Inc. in New York say the four-year agreement applies Beam’s process that edits highly granular genetic components to rare inherited diseases in the liver, muscles, and central nervous system.

Beam Therapeutics designs therapies for genetic diseases and blood-related cancers that edit and correct malfunctioning genes responsible for the disorders. The company’s technology edits malfunctioning genes at more precise and granular level than most others, aiming for pairs of nucleic acids called base-pairs that make up DNA. Humans have about 3 billion pairs of nucleic acids — adenine (A) with thymine (T), and cytosine (C) with guanine (G) — with the sequence of these nucleic acid pairs comprising a person’s DNA or genetic code. When mutations or errors occur in the these nucleic acids, the errors are transcribed into faulty instructions provided to cells with RNA and the proteins that result from those instructions.

One type of DNA error is called a point mutation, where a single base-pair is replaced by another base-pair. Point mutations, says Beam, make up at least half of DNA errors associated with disease, including neurodegenerative, metabolic, and blood disorders, as well as some types of hearing and vision loss. Beam Therapeutics says its technology is designed to correct those base-pair errors, using advances in the genome editing technique known as clustered regularly interspaced short palindromic repeats, or Crispr.

Spun-off from Harvard, MIT, and Editas Medicine

In addition, Beam Therapeutics designs its treatments to work inside the body, called in vivo. The company says its base-pair editing therapies can be delivered with nanoscale lipid or natural oil particles, benign adeno-associated viruses, or electroporation, a process using electric pulses to open cell walls. Different delivery methods, says Beam, are needed to reach different cell types. As reported by Science & Enterprise in May 2018, Beam Therapeutics is spun-off from labs at Harvard University and MIT, and licensing some of its technology from Crispr pioneering company Editas Medicine.

The agreement calls for Pfizer and Beam Therapeutics to collaborate on base-editing in vivo treatments with lipid nanoparticles and messenger RNA for three rare inherited diseases of the liver, muscles, and central nervous system, not yet in Beam’s pipeline. Beam will conduct initial research, while Pfizer retains an option to acquire exclusive rights to each therapy candidate. If Pfizer licenses any therapies, it will be responsible for their further development, regulatory approvals, and commercialization.

“We have a strong history in developing gene replacement therapies for rare diseases,” says Pfizer’s chief scientist Mikael Dolsten in a statement, “and we see this collaboration with Beam as an opportunity to advance the next generation of gene editing therapies, an exciting scientific frontier, potentially leading to transformation for people living with rare genetic diseases.”

Pfizer is providing Beam Therapeutics with an initial payment of $300 million. In addition, Beam is eligible for development, regulatory, and commercial milestone payments of up to $1.05 billion if all terms of the agreement are fulfilled, as well as royalties on sales of products from the collaboration. The parties may also extend the agreement for another year. Beam has an option to join with Pfizer in developing one of the therapy candidates licensed, for a fee paid to Pfizer, and profits divided about two-to-one between Pfizer and Beam.

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Disclosure: The author owns shares in Pfizer.

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