Beam, Verve stockpile cash as investors bank on gene editing progress – BioPharma Dive

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Dive Brief:

  • Biotechnology companies developing gene editing medicines are capitalizing on surging stock prices and investor enthusiasm for the field to raise hundreds of millions of dollars in new funding. 
  • On Tuesday, Beam Therapeutics and Verve Therapeutics, two high-profile biotechs based in Cambridge, Massachusetts, each announced new investments. Beam said it expects to receive $260 million through the sale of nearly 2.8 million shares to a group of private investors, while Verve closed a Series B funding round that will add $94 million to its cash holdings. 
  • Beam, which this year expects to ask the Food and Drug Administration to begin studies of its lead gene editing therapy in humans, anticipates using the new funds to support clinical testing as well to pursue “strategic partnerships.” Verve is earlier in development, but similarly expects the cash it raised to help advance its lead gene editing treatment. 

Dive Insight:

Recent months have brought a series of setbacks for companies developing gene replacement therapies, which aim to replace missing or faulty genes with a functional copy. Several of the most advanced treatments have hit regulatory roadblocks at the FDA, while others have had new safety concerns arise or disappointing data read out.

Those hurdles, however, haven’t appeared to deter investors from pouring money into developers of gene editing treatments — a field that, while fast emerging, remains years behind the older technology used in gene replacement. 

CRISPR Therapeutics, Editas Medicine and Intellia Therapeutics, a trio of early biotech adopters of CRISPR gene editing, are now worth nearly $25 billion combined after rapid run-ups in their stock prices. In early December, Intellia capitalized on those stock gains to raise $200 million selling stock at a price of $36.50 per share. Just over a month later, Intellia shares trade at $83 apiece. 

Beam, which remains some distance away from treating a patient with one of its medicines, is now worth a lofty $6.5 billion after its share price rose by 560% since an initial public offering last February. (Verve is a private company.) 

Both Beam and Verve use a different, more precise form of gene editing than the CRISPR-based approach used by CRISPR Therapeutics, Editas and Intellia. Know as base editing, the technique allows Beam and Verve to change a a single “letter” in a genetic sequence without cutting DNA. 

Beam’s focusing first on advancing a base editing treatment for sickle cell disease and beta thalassemia, but also has an early-stage pipeline aimed at several liver and eye diseases as well as two types of cancer. The company expects to file an application with the FDA in the second half of this year to begin human testing of its first treatment. 

Verve, meanwhile, just selected its first drug candidate, which targets an inherited form of high cholesterol that can lead to early heart disease. Verve’s treatment, dubbed VERVE-101, uses base editing technology licensed from Beam to change a single letter in a gene called PCSK9, inactivation of which has been proven to lower LDL cholesterol. 

At the J.P. Morgan Healthcare Conference earlier this month, the biotech unveiled new data showing its treatment led to durable reductions in both PCSK9 protein and LDL cholesterol levels in monkeys, results that support its preparations for human testing. Verve is targeting 2022 for initiation of its first clinical study. 

The $94 million in new fundraising will help the company get there, building on a $63 million Series A2 financing round just seven months ago. 

Existing investors Wellington Management Company and Casdin Capital were joined in the latest round by a slate of nine venture funds, several of which also just committed to buying shares in Beam, too. 

Putting together the Series B funding was the product of dozens of Zoom conference calls over the past three months, Sekar Kathiresan, Verve’s CEO, wrote on Twitter

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