Beam Therapeutics Files for IPO, Pursuing Treatments Using Base Editing

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Beam Therapeutics CEO John Evans and co-founders David Liu, PhD, Keith Joung, MD, PhD, and Feng Zhang, PhD. The developer of treatments based on base editing has filed for an initial public offering. [Beam Therapeutics]

Beam Therapeutics has disclosed plans to go public, submitting plans for an initial public offering (IPO) that would raise up to $100 million for the year-old company founded by gene editing pioneers to develop new treatments based on base editing.

“If existing gene editing approaches are “scissors” for the genome, our base editors are “pencils,” erasing and rewriting one letter in the gene,” Beam declared in its S-1 registration statement, filed on Friday.

Beam’s base editing technology is designed to underpin a potential new class of precision genetic medicines that target a single base in the genome—without making the double-stranded breaks in DNA that are carried out by existing gene editing technologies from CRISPR to zinc fingers, arcuses, and TAL nucleases.

“This approach uses a chemical reaction designed to create precise, predictable, and efficient genetic outcomes at the targeted sequence, which we believe will dramatically increase the impact of gene editing for a broad range of therapeutic applications,” Beam said of its base editing technology.

David R. Liu, PhD, of the Broad Institute of Harvard and MIT, as well as Harvard University and the Howard Hughes Medical Institute, co-founded Beam along with CRISPR pioneer Feng Zhang, PhD, and Keith Joung, MD, PhD, of Massachusetts General Hospital and Harvard Medical School.

Beam licenses foundational base editing IP from Harvard University, the Broad Institute, MIT, and Editas Medicine across multiple platforms. In May 2019, Beam and Japanese biotech Bio Palette announced an exclusive cross-license agreement around each company’s base editing intellectual property (IP).

And earlier this month, Beam launched a collaboration with Prime Medicine to research and develop a novel gene editing technology recently developed by Liu and his team of researchers at Broad Institute. The agreement gives Beam the exclusive right to pursue the technology for the creation and correction of single-base transition mutations (for example, A-to-G or C-to-T) as well as the treatment of sickle cell disease—though Beam added that it is not currently using the IP licensed from Prime Medicine in any of current programs.

“We believe we will be able to rapidly advance our portfolio of novel base editing programs by building on the significant recent advances in the field of genetic medicine,” Beam added.

Three pipelines

Beam organizes its treatment programs by delivery modality into three distinct pipelines. Furthest along in lead optimization phases are five hematology and oncology programs using electroporation for efficient delivery to blood cells and immune cells ex vivo. In earlier research phases are programs using lipid nanoparticles (LNPs) for nonviral in vivo delivery to the liver and potentially other organs in the future; and adeno-associated viral vectors (AAV) for viral delivery to the eye and central nervous system, or CNS.

Beam has yet to spell out how much in net proceeds it expects to generate from the IPO—which would depend on how much money the company ultimately agrees to raise—or how much it would set aside toward the purposes outlined in the registration statement.

Those purposes include:

  • Continued research and development of Beam’s portfolio of base editing programs, including preclinical studies and advancement through potential preclinical proof-of-concept for the company’s three delivery modalities.
  • IND-enabling studies and potential initiation of clinical studies for unspecified current programs.
  • Continued advancement of Beam’s platform technologies and discovery-stage research for other potential programs.

Since its founding last year Beam raised $222 million in capital—strong enough for the company to rank third among private companies listed in GEN’s A-List of “Top 10 Companies Leveraging Gene Editing in 2019,” published August 12.

In its registration statement, Beam listed $126.8 million in cash, cash equivalents, and marketable securities as of June 30. However, Beam also acknowledged that it accumulated a deficit of $156.2 million since its inception on January 25, 2017, with net losses of $31.5 million for the first six months of this year and $116.7 million for 2018.

Beam declared itself the first company to pursue development of new therapies using CRISPR base-editing technology when it announced in May 2018 that it raised $87 million in Series A financing led by F-Prime Capital Partners and ARCH Venture Partners.

In March of this year, Beam raised an additional $135 million through a Series B financing that attracted several new investors—including GV, the venture capital arm of Alphabet launched as Google Ventures in 2009. At the time, Beam said it would use the proceeds to advance development of next-generation CRISPR technologies, expand its pipeline of base editing programs, and extend its scientific and technical leadership.

Joining GV in the Series B were new investors that included Redmile Group, Cormorant Asset Management, Altitude Life Science Ventures, plus Series A investors that included F-Prime and ARCH Venture Partners, as well as Eight Roads Ventures, and Omega Funds.

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