Beam Therapeutics

Unlike first-generation approaches that introduce double-strand breaks, Beam’s base editing allows precise edits through direct chemical conversion of one base to another to address the point mutations that underlie many human diseases.

Industry Overview: The dawn of a new era in gene editing with CRISPR

CRISPR gene editing ushered in a new era of both modifying and understanding the genome. Prior editing approaches such as zinc finger nucleases, TALENs, and meganucleases are more cumbersome, costly, and time-consuming, as they require significant protein engineering for adaptation to the corresponding target sequence. In contrast, CRISPR uses a guide RNA to direct the active protein to specific genomic sequences through base pairing with the target location. Once in place, CRISPR cuts DNA and then allows natural DNA repair mechanisms to take over.

The Opportunity: Understanding use cases – gene disruption vs. specific gene editing

First-generation editing technologies, including CRISPR, rely on introducing double strand breaks into the genome. While precise edits could theoretically be achieved through homology-directed repair (HDR) or non-homologous end joining (NHEJ), these processes are much less efficient, and as such, traditional editing approaches are more suited for gene disruption than for specific and precise gene editing. Safety is also a lingering concern with first-generation editing approaches due to the potential for chromosomal rearrangements, as insertions and deletions occur with high frequency.

CRISPR sparked a revolution for new, next generation gene editing technologies that allow scientists to more efficiently and precisely modify the genome.

Why We Invested: Supporting the next wave of gene editing technologies

At F-Prime, we understand that breakthrough technologies require time to develop, which is why we continue to seek out innovators pioneering new approaches who embrace both the excitement and the challenges associated with advancing nascent modalities alongside us.

F-Prime recognized the transformative therapeutic potential of DNA editing and made early investments in companies focused on furthering CRISPR-based platforms such as Caribou. We also realized the power of technologies that could achieve direct single nucleobase editing to significantly expand the overall utility of this entirely new therapeutic category.

Our affiliate FBRI, which is comprised of a team dedicated to supporting research efforts to identify breakthrough discoveries, funded David Liu’s early base editing work. Dr. Liu is a renowned gene editing expert, scientific co-founder of Editas and Harvard and Broad Institute researcher. His team developed a DNA base editing technology that enabled more precise edits through direct chemical conversion of one base to another compared to approaches that introduce double-strand breaks. This discovery paved the way for the second generation of gene editing technologies and ultimately led to the creation of Beam Therapeutics.

Source: Beam Therapeutics

To facilitate the progress of this tremendously promising research, we invited the company to work out of F-Prime’s incubator office space with biopharma executive, John Evans, leading the team as CEO. Meanwhile, F-Prime’s President and Managing Partner, Stephen Knight, M.D., and Partner, Jessica Alston, Ph.D., helped to establish the company by assisting with licensing and IP discussions, and other members of the F-Prime team supported the company with recruiting key management hires, developing a strategic business plan and ultimately preparing the company for its public offering.

Founded in 2017, Beam Therapeutics raised nearly $223 million privately before going public in February 2020. They also announced a collaboration with Pfizer in January 2022. Today, Beam Therapeutics is a leader in harnessing base editing to develop precision genetic medicines that provide lifelong cures to patients suffering from serious diseases.

Learn more about Beam’s approach and vision for base editing in this video.

Our Focus: F-Prime continues to invest in biotech leaders

As we did with Beam, we’re constantly searching for the next breakthrough to invest in; sometimes, we build companies from scratch which we’ve done over 30 times and counting.

The convergence of science, technology, and healthcare in ways that achieve groundbreaking advancements gives rise to inspiring businesses and great investment opportunities.

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