Developing Precision Genetic Medicines
Gene Therapy at Beam Therapeutics
Beam Therapeutics is developing precision genetic medicines through base editing. Founded by leading scientists in CRISPR gene editing, Beam is pursuing therapies for genetic diseases using its proprietary base editing technology, which can make precise edits to single base pairs in DNA and RNA.
Over half of the genetic errors associated with disease result from a single-letter change in the billions of nucleobases (“bases”) that form the human genome. In other cases, certain natural genetic variations in DNA are known to protect against diseases.
Beam’s foundational technologies are capable of editing the genome at the level of a single letter – A, G, C, T – without cutting the DNA or RNA strands. By precisely modifying the genome to eliminate errors or write in protective changes, Beam’s novel approach to base editing may help prevent, modify and potentially cure a wide range of diseases affecting patients’ lives.
“Since the launch of Beam last year, we have made significant progress toward our goal of developing base editors as a new class of precision genetic medicines,” said John Evans, chief executive officer of Beam Therapeutics. “We now have 10 active programs underway and have expanded our team to more than 70 employees.”
Beam’s programs range from ex-vivo cell therapies to in-vivo direct correction. In a burgeoning CAR-T program, the accuracy and precision of cell counting is essential to understand the intricate biological impact both antigen binding technologies and base editing have on emerging cell therapies.
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“We have evaluated all the competing cell counting technologies on the market, and the NucleoCounter® family is by far our favorite.
Easy to use, they provide our teams with reassurance that the count we get is the correct one, each and every time. For early companies that might need to leverage external manufacturing or research assistance, it is also critical for every step of the production process to be transferable, and the NucleoCounter® family has an extensive track record of seamless integration with external CMOs and CROs.
The NC-200™ also benefits from removing the majority of operator variability from the equation, so assays tend to be more consistent from scientist to scientist and also site to site, reducing numerous obstacles to product development in the long run. These time savings directly correlate to cost savings and faster times into the clinic, all justification for the investment in a robust counter like the NC-200™.
We also found the value in integrating the NucleoCounter® family as early in the research pipeline as possible, ensuring results from early research are accurate and reproducible when products make the critical transition from research to development.
Not only are the NucleoCounter® instruments a must-have for anyone in the cell and gene therapy market, but ChemoMetec’s customer support is also a unique breath of fresh air.”
Aaron Edwards, Principal Research Associate, Beam Therapeutics