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Scribe develops gene-editing therapies based on CRISPR-CasX, an RNA-guided genome-editing platform. The company was founded in 2018 by Jennifer Doudna, Benjamin Oakes, David F. Savage, and Brett Staahl—a group of genome and molecular engineers from UC Berkeley's Doudna Lab, Oakes Lab, and the Innovative Genomics Institute.
Scribe Therapeutics' first technology is X-Editing (XE) molecules, which are designed to improve upon existing CRISPR genome editing technology, particularly for application in the treatment of neurodegenerative disorders. The treatments are primarily focused on in vivo (inside the body) disease-causing mutation correction, but they can also be utilized ex vivo (outside the body).
CasX-based therapies may be applied in the treatment of neurological, ophthalmological, multisystem, muscle, and metabolic diseases; hematopoietic disorders; and with cell therapy.
Neurological diseases: Huntington's disease (HD), familial amyotrophic lateral sclerosis (familial ALS), spinal muscular atrophy (SMA), spinocerebellar ataxia (SCA), early-onset familial Alzheimer’s disease, Parkinson's disease, progressive supranuclear palsy (PSP), Dravet syndrome, Angelman syndrome, Friedrich ataxia, and Batten disease.
Ophthalmological diseases: Retinitis pigmentosa (RP), cone-rod dystrophy (CRD), Leber's congenital amaurosis, hereditary optic atrophy (HOA), Stargardt disease, choroideremia, glaucoma, best vitelliform macular dystrophy (BVMD), achromatopsia, and usher syndrome.
Multisystem, muscle & metabolic: Cystic fibrosis, Duchenne muscular dystrophy (DMD), myotonic dystrophy (DM), limb-girdle muscular dystrophy (LGMD), TTR amyloidosis, alpha-1 antitrypsin deficiency (A1AD), familial hypercholesterolemia, tyrosinemia, phenylketonuria, acute intermittent porphyria (AIP), hypertriglyceridemia, Hutchinson-Gilford progeria syndrome (HGPS), methylmalonic acidemia, propionic acidemia, and primary hyperoxaluria.
Hematopoietic disorders: Sickle cell disease (SCD), severe combined immunodeficiency (SCID), Fanconi anemia (FA), hemophilia A/B, chronic granulomatous disease (CGD), and Von Willebrand disease.
Cell therapy: CAR-T, NK, TiL, HSC, iPSC, and more. Scribe Therapeutics claims that modifications can be made to nearly any cell therapy product.
The company kept a low profile until it announced raising $20 million in series A financing led by Andreessen Horowitz on Oct. 6, 2020. The following day, on Oct. 7, 2020, Jennifer Doudna along with Emmanuelle Charpentier won the Nobel Prize in Chemistry for their creation of CRISPR-Cas9 gene editing.
As part of the Series A financing, Scribe entered into a research partnership with Biogen Inc. to develop and market CRISPR-based treatments for Amyotrophic Lateral Sclerosis (ALS), securing $15 million of funding upfront and a potential $400 million in future development and commercial milestone payments. The contract also included tiered, high single-digit to sub-teen royalties.
On Mar. 31, 2021 Scribe announced that it had raised $100 million in Series B financing. It was led by Avoro Ventures and Avoro Capital Advisors, along with OrbiMed Advisors and Andreessen Horowitz. Perceptive Advisors, funds and accounts advised by T. Rowe Price Associates, funds managed by Wellington Management, Menlo Ventures, RA Capital Management, and an undisclosed investment firm also joined the assembly of investors.
Along with the Series B financing Scribe's board of directors has been expanded by two members: Behzad Aghazadeh, managing partner at Avoro Ventures and Avoro Capital Advisors, and Carl L. Gordon, managing partner at OrbiMed Advisors.
