CRISPR Therapeutics develops curative gene‑editing medicines using CRISPR/Cas9 technology, creating both ex vivo cell therapies and in vivo lipid‑nanoparticle delivery approaches. Its pipeline targets serious genetic, hematologic, metabolic, oncologic and rare diseases, aiming to provide one‑time, disease‑modifying treatments for patients.
Funding
$280M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
ECFounders
Product
Problem
Patients with serious genetic, hematologic, metabolic, and oncologic diseases often lack curative treatment options, relying on lifelong therapies that manage symptoms but do not address the underlying genetic cause.
Solution
CRISPR Therapeutics applies CRISPR/Cas9 gene‑editing technology to develop a portfolio of curative medicines. The company creates both ex vivo and in vivo therapies that edit patient cells to correct disease‑causing genes or modulate disease pathways. Ex vivo programs edit autologous hematopoietic stem cells to reactivate fetal hemoglobin for sickle cell disease and β‑thalassemia, while allogeneic CAR‑T candidates incorporate edits that enhance potency and reduce exhaustion. In vivo approaches deliver Cas9 mRNA and guide RNAs via lipid nanoparticles to the liver, targeting genes such as ANGPTL3, Lp(a), AGT, ALAS1, and SERPINA1 for cardiovascular, metabolic, and rare diseases. The pipeline also includes immune‑evasive, stem‑cell‑derived beta‑cell replacements for type 1 diabetes and next‑generation conditioning agents to improve transplant safety. By leveraging proprietary editing platforms and delivery technologies, the company aims to provide one‑time, disease‑modifying treatments across a broad range of indications.
Target Audience
Primary customers are pharmaceutical partners and healthcare providers seeking gene‑editing therapeutics for patients with serious genetic, hematologic, metabolic, oncologic, and rare diseases.
Features
- Ex vivo CRISPR/Cas9 editing of autologous hematopoietic stem cells (exa‑cel) to induce fetal hemoglobin for sickle cell disease and transfusion‑dependent β‑thalassemia
- Allogeneic CRISPR‑edited CAR‑T cell therapies (CTX112 and related programs) with edits that boost potency and limit exhaustion for CD19‑positive cancers and autoimmune disorders
- In vivo lipid‑nanoparticle delivery of Cas9 mRNA/gRNA to the liver for gene knock‑down (e.g., ANGPTL3, Lp(a), AGT, ALAS1, SERPINA1) targeting cardiovascular, metabolic, and rare diseases
- Immune‑evasive, stem‑cell‑derived beta‑cell replacement therapies (CTX211, CTX213) for type 1 diabetes, employing gene edits to prevent immune rejection
- Next‑generation conditioning agents (e.g., CD117 ADC) designed to improve safety of hematopoietic stem cell transplantation
- Proprietary SyNTase™ editing platform for precise correction of point mutations such as the E342K SERPINA1 variant
- Broad disease focus spanning hemoglobinopathies, oncology, autoimmune disease, cardiovascular, metabolic, rare, and regenerative medicine areas