A Startup is Developing More Precise Gene Therapies

A company called Beam Therapeutics is developing a new form of genetic editing, called ‘base editing’, to treat certain genetic diseases. The full story can be read here, at MedCity News.

Genes are made up of base nucleotides that bond together to form a code. Base editing is a form of gene therapy in which one specific base is changed to another. This means that, hypothetically, certain genetic variations to bases that cause genetic diseases could be identified and replaced. For example, a team of researchers in Korea recently published a study that used base editing on mice with Duchenne muscular dystrophy. There are four types of base: adenine (A), guanine (G), thymine (T), and cytosine (C). All genes are formed by sequences of these four bases. Beam Therapeutics has already licenced two of its base editing techniques; one that allows C to be changed with T, or G for A, and another that swaps A to G or T to C.

The company’s co-founder and Harvard University academic, David Liu, developed the base editing technology. It is different from existing CRISPR gene editing techniques that cut larger sequences of DNA and RNA and are less precise.

The company has already received $87 million in funding. The venture capital firms F-Prime Capital Partners and ARCH Venture Partners have both made significant investments in Beam Therapeutics. In addition, the company has received investments from angel investors, long-term institution investors, and family offices. This Series A funding is likely to last the company for several years, but as the company grows it will need more and more money to carry on. The business currently has fifteen employees, and around ten more are expected to be hired.

Around half of genetic diseases are caused by single base changes, and, hypothetically, may benefit from this new technology. Beam Therapeutics is currently considering working on ten to fifteen diseases, but are not releasing information about which diseases these are in order to not prematurely raise people’s hopes. Over the next year, the company will work on getting a better sense of the effectiveness of base editing on the chosen diseases and then will progress to animal studies and pre-clinical trials.
Beam Therapeutics is currently the only company thought to be working on base editing, however, this is likely to change as other people carry out similar research.

Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

Share this post

Follow us