RARE Daily

Beam Therapeutics Raises $117.5 Million

October 1, 2020

Rare Daily Staff

Beam Therapeutics raised $117.5 million through a public offering of 5 million shares of its common stock at $23.50 a share.

In addition, Beam granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of common stock at the public offering price, less the underwriting discounts and commissions.

Founded by leading scientists in CRISPR gene editing, Beam Therapeutics is pursuing therapies for serious diseases using its proprietary base editing technology, which the company says can make precise edits to single base pairs in DNA and RNA to correct, modify, activate, silence, and edit genes at multiple sites at the same time without detectable translocations.

Beam is addressing a variety of targets using three delivery technologies that have been clinically validated for a specific target: electroporation for hematology and oncology targets, non-viral liquid nano-particles for delivery of RNA therapies targeting liver diseases, and AAV-vector gene therapy delivery for ocular and central nervous system disorders.

Beam’s preclinical pipeline is broad and includes compounds in development for rare diseases such as sickle cell disease, beta thalassemia, alpha 1 antitrypsin deficiency, glycogen storage disorder 1a, Stargardt disease, an inherited form of macular degeneration, and other central nervous system disorders.

In August in its second quarter 2020 earnings report, the company said that it would first target sickle cell disease, and announced the first two development candidates in its portfolio: BEAM-101, an adenine base editor (ABE) that reproduces single base changes observed in individuals with hereditary persistence of fetal hemoglobin in which elevated levels of fetal hemoglobin protect these individuals from the effects of sickle cell disease or beta-thalassemia; and BEAM-102, an ABE that directly corrects the causative mutation in sickle cell disease, converting it into a naturally-occurring human hemoglobin variant, Hb-G Makassar. Individuals with the Makassar variant have normal hematologic parameters and no evidence of hemoglobin polymerization or sickling of red blood cells.

“BEAM-101 and BEAM-102 are highly differentiated editing programs that may enable a one-time treatment option for patients with sickle cell disease,” said Giuseppe Ciaramella, president and chief scientific officer of Beam in a statement. “Both candidates are supported by promising preclinical data, and we are working to advance them to the next stage of development to assess the impact they could have in treating this devastating disease.”

Photo: Giuseppe Ciaramella, president and chief scientific officer of Beam

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