Scribe Therapeutics

Scribe Therapeutics

Develops and licenses CRISPR-based genetic medicines

Overview

Scribe Therapeutics develops gene-editing therapies using CRISPR-based technologies. Its primary work is to create and license a proprietary CRISPR genetic medicine platform to treat genetic diseases, including in vivo gene editing. The company also develops viral and non-viral delivery methods to enable editing inside the body, aiming for highly specific genome targeting and allele-specific interventions. This focus on precise delivery and allele-targeted editing differentiates Scribe Therapeutics from others who may offer broader gene-editing tools or external delivery methods. Revenue comes from partnering with healthcare providers, research institutions, and pharmaceutical companies to co-develop gene therapies and by licensing its technology. The company’s goal is to advance genetic medicine by providing precise, partner-enabled gene therapies and delivery platforms that can treat diseases caused by specific gene variants.

About Scribe Therapeutics

Simplify's Rating
Why Scribe Therapeutics is rated
B-
Rated B on Competitive Edge
Rated B on Growth Potential
Rated C on Differentiation

Industries

Biotechnology

Healthcare

Company Size

51-200

Company Stage

Series B

Total Funding

$140M

Headquarters

Alameda, California

Founded

2017

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Simplify's Take

What believers are saying

  • Sanofi partnership worth $1.2B+ validates XE platform across multiple disease indications.
  • STX-1150 entering clinic mid-2026 targets $10.1B genetic medicine market by 2026.
  • Single-dose PCSK9 silencing differentiates from existing therapies requiring repeated administration.

What critics are saying

  • STX-1150 hepatic toxicity or off-target editing emerges in human clinical trials.
  • Editas and CRISPR Therapeutics capture PCSK9 market with earlier clinical programs.
  • FDA rejects epigenetic silencing class as insufficiently reversible for preventative use.

What makes Scribe Therapeutics unique

  • DeepXE AI platform predicts CRISPR guide potency, accelerating therapeutic design precision.
  • STX-1150 achieves durable LDL-C control via epigenetic silencing without permanent DNA changes.
  • XE platform demonstrates >75% liver editing in primates with no detectable off-target effects.

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Funding

Total Funding

$140M

Above

Industry Average

Funded Over

3 Rounds

Series B funding is typically for startups that have proven their business model and need more funding to expand rapidly—often by entering new markets or adding more products. Investors are usually venture capital firms that specialize in later-stage investments.
Series B Funding Comparison
Above Average

Industry standards

$35M
$45M
Linktree
$65M
Substack
$100M
ClickUp
$100M
Scribe Therapeutics

Benefits

Health Insurance

Growth & Insights and Company News

Headcount

6 month growth

1%

1 year growth

1%

2 year growth

6%
Generate Biomedicines
Jan 29th, 2026
Generate:Biomedicines welcomes Aarif Khakoo, MD, MBA, as chief scientific officer

Generate:Biomedicines welcomes Aarif Khakoo, MD, MBA, as chief scientific officer. 01.29.2026 SOMERVILLE, Mass., Jan. 29, 2026 - Generate:Biomedicines ("Generate") today announced the appointment of Aarif Khakoo, MD, MBA, as chief scientific officer. A physician-scientist and experienced biotechnology leader, Dr. Khakoo brings deep expertise in building and scaling platform-driven drug generation engines, with a track record of advancing medicines from discovery through early clinical development. In this role, he will lead scientific and strategic application of The Generate Platform to discover and develop therapeutics across the company's pipeline. "Aarif is a highly respected scientific leader with a rare combination of translational insight, platform vision, and operational rigor," said Mike Nally, chief executive officer of Generate:Biomedicines. "His experience building and scaling innovative discovery engines and advancing programs that translate into the clinic will be instrumental as we continue to expand the capabilities of The Generate Platform and advance our growing pipeline of protein therapeutics." Dr. Khakoo joins Generate from Scribe Therapeutics, where he served as chief scientific officer and head of research and development, leading platform development, biology discovery, and translational research for a next-generation genome editing and epigenetic silencing platform. Previously, he was head of drug development and chief medical officer at Calico Life Sciences, where he both oversaw multiple investigational new drug and clinical trial applications filings and advanced a pipeline of programs across immuno-oncology, neurodegeneration, and kidney disease, while chairing the Calico - AbbVie Joint Steering Committee. Earlier in his career, Dr. Khakoo spent seven years at Amgen, where he founded and led the cardiometabolic disorders therapeutic area and contributed to the discovery and advancement of multiple novel clinical candidates, including programs now in late-stage development and one that has received FDA approval. He also served as head of Amgen's South San Francisco site, overseeing its growth to an R&D hub of approximately 600 employees and chairing Amgen's Early Program Review Committee, which guided the company's early development portfolio. Before transitioning to industry, Dr. Khakoo served on the cardiology faculty at The University of Texas MD Anderson Cancer Center, leading translational research on heart failure and cancer therapy-related cardiotoxicity. He is a board-certified physician, adjunct clinical faculty member at Stanford University, and author of numerous peer-reviewed publications. About Generate:Biomedicines Generate is a clinical-stage generative biology company founded at the intersection of machine learning, biological engineering, and medicine, advancing a new era of programmable biology to engineer better medicines for patients, faster. The Generate Platform integrates computational design with large-scale biological experimentation, enabling the creation of novel, optimized therapeutics that address historically undruggable and hard-to-drug targets, as well as known targets in new and more effective ways. The platform has enabled the generation of a broad pipeline of therapies across multiple therapeutic areas and protein-based modalities, addressing health challenges out of reach of traditional approaches. Founded in 2018, Generate is leading a fundamental shift from drug discovery to drug generation. Learn more at www.generatebiomedicines.com or follow us on X, LinkedIn, and YouTube.

PharmiWeb.com
Dec 15th, 2025
Scribe Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference

Scribe Therapeutics to present at the 44th Annual J.P. Morgan Healthcare Conference. ALAMEDA, Calif.-(BUSINESS WIRE)-#CRISPR-Scribe Therapeutics Inc. (Scribe), a genetic medicines company pioneering next-generation in vivo CRISPR-based medicines designed to be safe, durable, and effective enough to become standard-of-care treatments for prevalent diseases, today announced that Benjamin Oakes, Ph.D., Chief Executive Officer of Scribe, will present at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026 at 8:30 a.m. PT in San Francisco, CA. About Scribe Therapeutics Scribe Therapeutics is revolutionizing medicine by developing optimized in vivo CRISPR-based genetic medicines designed to become standard of care treatments for patients suffering from highly prevalent diseases, starting with cardiometabolic disease. The company is on a mission to build the first CRISPR-based therapeutics that are effective and safe enough to transform everyone's lifetime risk for disease. Scribe's CRISPR by Design(TM) approach engineers bacterial immune systems into a premier suite of genome and epigenome editing tools built for unique molecular advantages in activity, specificity, and deliverability, enabling the creation of therapies with a broader therapeutic window and safe for use as a preventative treatment. The company's lead candidate, STX-1150, is a novel liver-targeted therapy designed to epigenetically silence the PCSK9 gene, resulting in significant and durable reduction of LDL-C levels. To broaden and accelerate the impact of its engineered CRISPR technologies for patients, Scribe has formed strategic collaborations with world-leading pharmaceutical companies including Sanofi and Eli Lilly. Co-founded by Nobel Prize winner Jennifer Doudna and backed by leading life sciences investors, Scribe is engineering the future of genetic medicine. To learn more, visit www.scribetx.com. Media Contact: Thermal for Scribe Therapeutics [email protected]

Business Wire
Mar 7th, 2025
Scribe Therapeutics Presents Data on its Epigenetic Long-Term X-Repressor Technology at the 2025 Keystone Symposia on Precision Genome Engineering Conference

Scribe Therapeutics presents data on its Epigenetic Long-Term X-Repressor technology at the 2025 Keystone Symposia on Precision Genome Engineering conference.

Business Wire
Jan 9th, 2025
Scribe Therapeutics to Present at the 43rd Annual J.P. Morgan Healthcare Conference

Scribe Therapeutics to present at the 43rd Annual J.P. Morgan Healthcare Conference.

Business Wire
May 10th, 2024
Scribe Therapeutics Presents Data Demonstrating Highly Potent Gene Editing In Vivo, Including Saturated Editing Of Hepatocytes In Nhps, By An Engineered Xe Genome Editor; Showcases Novel Epigenetic Editing Capabilities At 2024 Asgct Annual Meeting

ALAMEDA, Calif.--(BUSINESS WIRE)--Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, presented data on its X-Editor (XE) and Epigenetic Long-Term X-Repressor (ELXR) technologies at the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, MD, and virtually.The data described in the company’s oral presentations and poster session showcase the dramatic potency, safety, and specificity of the novel XE genome editing and ELXR epigenetic editing platforms and the company's developments in successfully advancing these technologies into large animal models, including non-human primates.“ A major challenge in the field has been to achieve saturating levels of genome editing, at therapeutically relevant doses, with molecules that have better safety and delivery characteristics than CRISPR-Cas9—this has not been possible to date,” said Benjamin Oakes, Ph.D., co-founder and Chief Executive Officer of Scribe. “ For the first time, Scribe has demonstrated that the holistic engineering of a wholly unique and highly potent CRISPR enzyme can enable this goal. We have demonstrated that our XE platform can achieve editing rates of greater than 75% in the liver of NHPs, theoretically targeting all hepatocytes, while not displaying any detectable off-target effects in primary human cells.”“ Furthermore, we were thrilled to unveil a taste of the engineering behind Scribe’s ELXR epigenetic editing platform and present data elucidating the effectiveness of our comprehensive engineering approach compared to previous methodologies,” Dr. Oakes said. “ We believe this positions Scribe as the only company to have engineered, from the ground up, novel CRISPR genome editing and epigenetic editing platforms that can meet and often exceed the field’s expectations regarding therapeutically relevant potency and specificity.”Additional highlights on the developments of the XE platform and application in vivo include:Presentation: Engineering CasX to Create a Gene Editor with Potent Activity in Non-Human PrimatesUsing a variety of holistic engineering approaches, Scribe has created X-Editor molecules that are >100 steps in sequence space removed and >100-fold more potent than the naturally occurring CRISPR predecessorsXE achieves at or near-saturating levels of editing across a broad range of tissue types, including the eye, CNS, cardiac and skeletal muscle, and liver using either AAV or LNPXE is highly potent in non-human primate studies, achieving >75% editing in the liver and saturating editing at <1.5mg/kgWhen tested at different sites, XE is highly specific, demonstrating no detectable off-target editing at doses 10-fold higher than the effective dose in primary human hepatocytesScribe continues to expand the XE platform by engineering molecules with customizable genomic targetability by modifying the PAM recognition of the XE platformPresentation: AAV-Mediated Delivery of a Novel CasX-Editor Molecule Achieves Allele-Specific and Potent Editing of P23H Rhodopsin in a Mouse Model of Retinitis PigmentosaXE and AAV cargo engineering enables safe and selective XE delivery and editing in rod photoreceptors when administered subretinallyScribe demonstrates for the first time the use of XE to achieve allele-specific editing of the P23H.RHO locus, in an in vitro system and a phenotypically relevant in vivo model

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