CRISPR Therapeutics AG (C2RS34)

Overview

CRISPR Therapeutics AG is a pioneer in the field of gene editing, leveraging its sophisticated Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform to develop innovative gene-based medicines for a variety of serious human diseases. Founded in 2013 and based in Zug, Switzerland, CRISPR Therapeutics is at the forefront of medical research in gene editing technology, particularly CRISPR/Cas9, which enables precise and targeted modifications to genomic DNA. With a focus on addressing unmet medical needs in hemoglobinopathies, immune-oncology, autoimmune diseases, in vivo treatments, and type 1 diabetes, the company is dedicated to advancing a diverse portfolio of therapeutic programs aimed at providing transformative treatments for patients worldwide.

Products and Services

• CASGEVY

An ex vivo CRISPR/Cas9 gene-edited cell therapy, CASGEVY is CRISPR Therapeutics’ lead product candidate designed to treat patients with transfusion-dependent beta-thalassemia, severe sickle cell disease, and other hemoglobinopathies. This innovative treatment edits a patient's hematopoietic stem and progenitor cells to induce high levels of fetal hemoglobin in red blood cells, potentially alleviating symptoms of these severe genetic diseases.

• CAR T Cell Therapies: CTX112 and CTX131

CRISPR Therapeutics is also developing CAR T cell therapies, including CTX112, targeting CD19, and CTX131, targeting CD70. These therapies are designed for the treatment of various oncology and autoimmune indications, utilizing engineered T cells to recognize and attack cancer cells or modulate immune responses in autoimmune diseases.

• In Vivo Gene Editing: CTX310 and CTX320

In a groundbreaking approach to cardiovascular disease treatment, CRISPR Therapeutics offers CTX310 and CTX320. These in vivo gene editing therapies target angiopoietin-like protein 3 and lipoprotein, respectively, disrupting the validated targets to address underlying causes of cardiovascular conditions.

• VCTX211

VCTX211 represents an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate focused on the treatment of Type 1 Diabetes (T1D). This advanced therapeutic aims to offer a novel approach to managing T1D by addressing the immune system's role in the disease's development and progression.

In addition to its innovative product pipeline, CRISPR Therapeutics AG has formed strategic partnerships with leading companies in the biotech sector, including Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. These collaborations are instrumental in advancing CRISPR/Cas9 and other gene editing technologies, furthering the company's mission to provide groundbreaking treatments for diseases with significant unmet need.