Behind the BioTech Startup Building Gene-Editing Tools to Cure Famously Hard-to-Cure Diseases and Make Billions of Dollars

Gene editing is one of the most exciting areas in healthcare, and several public and private companies are trying to crack the code. Scribe Therapeutics, a biotech startup focused on gene-editing tools to treat tough diseases, is attracting attention from major pharmaceutical firms and venture capitalists.

Crispr pioneer and Nobel Laureate Jennifer Doudna Ph.D. co-founded Scribe in October 2018. Benjamin Oakes Ph.D., David Savage, and Brett Staahl Ph.D. are the other cofounders of the company.

Notably, Scribe works on “CasX” which is a different set of proteins for Crispr systems. It has also developed a platform called “Crispr-by-design” which can tackle multiple diseases. Instead of removing cells from the body for editing and then returning them, it focuses on editing genes within the body.

According to Dr. Joshua Modell who’s an assistant professor at John Hopkins School of Medicines, CasX is better than Cas9 as they are smaller which makes them more apt for certain applications.

He also added that CasX proteins might be more selective and only impact the desired part of the DNA.

Scribe has been in the news lately for the big deals that it has signed with pharma majors.

Gene-Editing Company Signs Deals Worth Over $4 Billion

Scribe has signed deals with pharma majors that total around $4 billion. The most recent such deal is with Prevail which is a wholly owned subsidiary of Eli Lilly and is working on genetic medicines for Parkinson’s disease and other such neurodegenerative conditions.

As part of the deal which is valued at $1.5 billion, Prevail would provide $75 million as an upfront payment and equity investment. Prevail will also provide R&D funding and additionally pay Scribe some royalties on future sales.

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Scribe has also expanded its deal with pharma giant Sanofi to around $2.2 billion. The previous agreement was signed last year and was valued at around $1 billion with $25 million as an upfront payment.

The new alliance could bring in $1.2 billion as success-based payments to the gene-editing company, including a $40 million upfront payment.

Regarding the expanded partnership, Oakes stated, “We are consistently impressed by the knowledge, thoroughness, and urgency that Sanofi has shown in our current ex vivo collaboration, along with their swift progress and innovation in non-viral delivery technologies. We are excited to broaden our efforts into new areas with significant unmet needs.”

The first such deal that Scribe announced was in October 2020 with Biogen which included an upfront payment of $15 million and upto $400 million based on meeting pre-decided milestones.

Scribe Therapeutics Funding and Valuation

Scribe Therapeutics raised $20 million in the Series A funding round which was led by Andreessen Horowitz. It was followed by a Series B funding of $100 million in March 2021. Avoro Ventures led the round, which valued the company at $300 million.

After the funding round, Avoro Ventures’ partner Behzad Aghazadeh joined the company’s board and termed Scribe’s platform “quite unique compared to Cas9 systems.”

Aghazadeh added that Oakes has “really thought out and engineered his way to really addressing the challenges that other gene-editing companies still face.”

According to data from Pitchbook, VCs have invested around $3.3 billion in the industry since 2019. That however might not provide the correct funding picture as multiple gene-editing and genomics companies went public over the last three years – primarily through special purpose acquisition company mergers (SPACs).

ARK Invest, which is led by Catie Wood even has an ETF named ARK Genomics Revolution ETF (NYSE: ARKG) that invests in genomic companies. The ETF meanwhile trades at around a third of its all-time highs that it hit in 2021. Wood nonetheless sees digital wallets and genomics as two of the most promising trends over the next decade.

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