A firm that launched last year to develop therapies using single-letter CRISPR/Cas9 gene editing has raised another round of financing.
Cambridge, Massachusetts-based Beam Therapeutics said Wednesday that it had closed a $135 million Series B funding round. New participants include Redmile Group, Cormorant Asset Management, GV, Altitude Life Science Ventures and undisclosed investors. Existing investors that also took part include F-Prime Capital, ARCH Venture Partners, Eight Roads Ventures and Omega Funds.
The company launched last May, emerging from stealth mode with an $87 million Series A funding round led by ARCH and F-Prime.
Beam’s technology is designed to edit the genome at the letter of the single letter – A (adenine), G (guanine), T (thymine) and C (cytosine) – rather than the more common method of cutting DNA and RNA strands. The thinking is that because more than half of the genetic errors associated with disease result from a single-letter change among the billions of nucleobases that form the human genome, eliminating errors at the single-letter level can precisely modify the genome. That, in turn, could prevent, modify or cure a wide range of diseases.
CRISPR/Cas9 technology has come a long way on multiple fronts since Beam’s launch last year. In September, a federal court ruled in favor of the Massachusetts Institute of Technology and Harvard University’s Broad Institute in a patent interference lawsuit between that organization and the University of California Berkeley.
And in November, Chinese researcher He Jiankui came under fire for allegedly using CRISPR/Cas9 technology to modify the genomes of twin baby girls in order to make them resistant to HIV infection, leading to questions about the ethics surrounding the technology.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Most recently, the Food and Drug Administration gave the green light to the first ever clinical trial involving a therapeutic developed using CRISPR/Cas9, namely a Phase I/II study of Vertex Pharmaceuticals and CRISPR Therapeutics’ CTX001 in sickle cell disease.
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