Within just six months of coming out of stealth mode, Scribe Therapeutics, co-founded by Jennifer Doudna, the 2020 Nobel Prize Winner, bagged another $100 million through Series B financing on March 31st.
The funds will be channeled towards developing a pipeline of therapeutics for neurodegenerative diseases and others with high unmet needs, the company said. Furthermore, it will be used to advance Scribe’s custom-made gene editing and delivery technologies.
The Series B financing round was led by Avoro ventures and Avoro Capital Advisors along with OrbiMed Advisors and Andreessen Horowitz. Behzad Aghazadeh of Avoro Ventures and Avoro Capital Advisors and Carl L. Gordon of OrbiMed Advisors will join Scribe’s board of directors.
“Scribe is applying deep, multidisciplinary engineering expertise to develop the custom tools and applications needed to confront genetic diseases,” said Behzad Aghazadeh. “We are pleased to support Scribe’s accomplished leadership team in their continued growth of the platform and their dedication to fulfilling the promise of CRISPR gene editing.”
The CRISPR-Cas9 tool developed by Jennifer Doudna and Emmanuelle Charpentier has been commercialized at an unprecedented speed in the past few years. However, the Cas9 protein has limitations like off-target effects and low efficiency. To overcome this, the CRISPR-Cas tool has been tweaked endlessly to generate new tools. The Cas12 protein, for instance, offers high editing efficiency. CasX, an improved enzyme, is small, compact, and offers advantages of both Cas9 and Cas12.
Scribe spun out from Doudna’s lab the day after she was awarded the Nobel Prize. The company is developing best-in-class genetic medicine technologies by honing enzymes, specifically CasX, for greater efficacy, specificity, and deliverability.
According to the co-founder, Dr. Benjamin Oakes, Scribe will help create “novel CRISPR technologies with the necessary therapeutic attributes needed to overcome the existing challenges of gene editing and delivery technologies.”
Making CRISPR Enzymes from Scratch
To elaborate on this, the company is building CRISPR enzymes from scratch. Instead of fishing for new Cas proteins from the microbial genome, it is making changes to the existing Cas protein, specifically to CasX, in the lab, which generates an endless repertoire of CasX variants.
These variants are selected for high genome-editing efficacy, small size, and high specificity to the target sequence. Since CasX is less than 1000 amino acids in size, it can be easily packed in a delivery vector.
To date, Scribe’s “Crispr by design” platform has engineered CasX enzymes found in nature into millions of novel variants with ideal therapeutic attributes to enable highly effective and specific in vivo applications. For example, its X-editing (XE) technology is an engineered molecule that offers researchers to edit living cells with greater specificity, activity, and deliverability than currently available genome editing technology.
“Scribe’s engineering approach has revolutionized industry expectations for blueprinting and creating CRISPR-based therapies,” said Vijay Pande, General Partner at Andreessen Horowitz. “By accelerating the shift from an artisanal discovery approach to a fully industrialized one, the company is poised to fundamentally transform how we treat and manage genetic diseases at scale.”
The company is already testing its battery of CRISPR-CasX tools on diseases of significant unmet need, starting with a pipeline of neurodegenerative disorders. Recently, it made a $15 million pact with Biogen to create therapeutics for ALS.
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