CRISPR Therapeutics has outlined its strategic priorities and 2024 outlook, emphasizing its achievements in 2023, including the first-ever approval of a CRISPR-based gene-editing therapy, CASGEVY. The company is set to focus on clinical trials across various therapeutic areas, such as oncology, autoimmune, cardiovascular, and diabetes. CASGEVY received regulatory approvals in the U.S., Great Britain, and Bahrain for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of March 30, 2024, for CASGEVY in TDT. CRISPR Therapeutics also highlighted next-generation research in hemoglobinopathies, immuno-oncology, autoimmune diseases, and in vivo gene editing programs. The company’s cash position is robust, starting 2024 with approximately $1.9 billion. Additionally, CRISPR Therapeutics provided an update on the ViaCyte collaboration, manufacturing capabilities, and the operational status of its GMP facility. The company aims to continue its focus on resource efficiency and return on invested capital.
Table: Main Points from CRISPR Therapeutics Press Release
| Key Highlights | Details |
|---|---|
| Landmark Achievements in 2023 | – First-ever approval of a CRISPR-based gene-editing therapy, CASGEVY. – Entry into clinic with in vivo therapies. |
| Strategic Priorities for 2024 | – Drive forward programs and expand pipeline for gene editing therapies. – Focus on clinical trials in oncology, autoimmune, cardiovascular, and diabetes. – Continuous innovation on the platform with next-generation gene editing and delivery technologies. |
| CASGEVY (exa-cel) | – Regulatory approvals for CASGEVY in the U.S., Great Britain, and Bahrain for SCD and TDT. – Positive opinion from EMA’s CHMP for CASGEVY. – PDUFA action date of March 30, 2024, for CASGEVY in TDT. – Additional regulatory submissions under review in Switzerland, Saudi Arabia, and planned for Canada. – Completion of enrollment in two global Phase 3 studies of CASGEVY in patients 5 to 11 years old with SCD or TDT. – Vertex’s efforts to establish a network of authorized treatment centers (ATCs) and agreements for access to CASGEVY. |
| Hemoglobinopathies | – Two next-generation research focuses to expand addressable population with SCD and TDT. – Advancement of internal targeted conditioning program through preclinical studies. – Ongoing research for in vivo editing of hematopoietic stem cells, supported by a $14.5 million grant from the Bill & Melinda Gates Foundation. |
| Immuno-Oncology and Autoimmune Disease | – Ongoing clinical trials for next-generation allogeneic CAR T candidates, CTX112 and CTX131, targeting CD19 and CD70, respectively. – Plans to initiate a clinical trial of CTX112 in systemic lupus erythematosus (SLE) in the first half of 2024. – Expansion of CTX131 trials into hematologic malignancies. – Expected clinical update in 2024 for next-generation candidates. |
| In Vivo Gene Editing Programs | – Advancement of in vivo gene editing programs (CTX310 and CTX320) using lipid nanoparticle delivery for cardiovascular diseases. – Phase 1 clinical trial ongoing for CTX310 targeting ANGPTL3. – Initiation of Phase 1 clinical trial for CTX320 targeting Lp(a). – Expectation to nominate additional in vivo programs targeting rare and common diseases in mid-2024. |
| Regenerative Medicine | – ViaCyte’s opt-out from collaboration for gene-edited stem cell therapies for diabetes. – CRISPR Therapeutics’ commitment to advancing a Phase 1 clinical trial for CTX211 for the treatment of Type 1 Diabetes (T1D). – Continued collaboration with Vertex for non-exclusive rights to CRISPR/Cas9 technology for T1D. |
| Manufacturing | – Fully operational GMP facility in Framingham, MA. – Support for production of investigational therapies with potential benefits of lower cost, increased flexibility, and greater scalability. |
| Cash Position and Operating Efficiencies | – Strong financial position with approximately $1.9 billion in cash, cash equivalents, and marketable securities. – Focus on resource efficiency and return on invested capital. |
Sources: CRISPR Therapeutics Press Release, CRISPR Therapeutics Website
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