CRISPR Therapeutics Updates Business and Reports Q1 2024 Financials

CRISPR Therapeutics, a biopharmaceutical company specializing in gene-based medicines for critical illnesses, has reported its financial results for the first quarter of 2024. The company has shown significant advancements in its portfolio, particularly with the launch of CASGEVY™ (exagamglogene autotemcel, or exa-cel), which is approved in multiple countries for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Notable Achievements:
- CASGEVY™ has seen successful regulatory approvals in the U.S., Great Britain, the EU, Saudi Arabia, and Bahrain. Regulatory submissions have also been completed in Switzerland and Canada, with Canada granting priority review.
- Vertex Pharmaceuticals is leading the global development, manufacturing, and commercialization of CASGEVY™ in collaboration with CRISPR Therapeutics.
- More than 25 authorized treatment centers globally have been activated, and multiple patients have already had cells collected for treatment.
- Vertex has secured agreements with commercial and government health insurance providers in the U.S., as well as reimbursement arrangements in Saudi Arabia, Bahrain, and France for eligible patients.

Pipeline Expansion:
- CRISPR Therapeutics is advancing next-generation CAR T product candidates, CTX112™ and CTX131™, targeting CD19 and CD70 respectively. These candidates are undergoing clinical trials across multiple indications.
- The company's in vivo gene editing product candidates, CTX310™ and CTX320™, are targeting ANGPTL3 and Lp(a) respectively and are in ongoing clinical trials.
- CRISPR Therapeutics has expanded its preclinical programs with new candidates utilizing lipid nanoparticle (LNP) delivery to the liver for refractory hypertension and acute hepatic porphyria (AHP).

Research and Development:
- The company continues to develop its targeted conditioning program using an anti-CD117 (c-Kit) antibody-drug conjugate (ADC) through preclinical studies.
- Research efforts are underway to enable in vivo editing of hematopoietic stem cells, which could eliminate the need for conditioning and expand treatment possibilities beyond SCD and TDT.

Immuno-Oncology and Autoimmune Diseases:
- CTX112™ and CTX131™ are part of CRISPR Therapeutics’ next-generation allogeneic CAR T candidates. CTX112™ is being developed for both oncology and autoimmune indications, with a Phase 1/2 trial ongoing for CD19 positive B-cell malignancies.
- CTX131™ is currently in a clinical trial for solid tumors, with plans to initiate trials for hematologic malignancies.

In Vivo Programs:
- CRISPR Therapeutics has a proprietary LNP platform for delivering CRISPR/Cas9 to the liver. The first two in vivo programs, CTX310™ and CTX320™, target cardiovascular disease and are in ongoing clinical trials.
- New preclinical programs, CTX340™ and CTX450™, are utilizing this LNP delivery system targeting refractory hypertension and acute hepatic porphyria respectively.

Regenerative Medicine:
- CTX211™, an allogeneic, gene-edited stem cell-derived therapy for Type 1 Diabetes (T1D), is in a Phase 1 clinical trial. CRISPR Therapeutics aims to develop a beta-cell replacement product that does not require chronic immunosuppression.

Corporate Developments:
- Christian Rommel, Ph.D., has been proposed for election to the Board of Directors, bringing extensive experience in drug development.
- In February 2024, CRISPR Therapeutics secured approximately $280 million through a direct offering to institutional investors.

Financial Highlights:
- The company reported $2.1 billion in cash, cash equivalents, and marketable securities as of March 31, 2024, up from $1.7 billion at the end of 2023. This increase was driven by proceeds from the direct offering, a milestone payment from Vertex, and other financial activities.
- R&D expenses decreased to $76.2 million from $99.9 million year-over-year, primarily due to reduced external research and manufacturing costs.
- General and administrative expenses fell to $18 million from $22.4 million, mainly due to lower employee-related and stock-based compensation expenses.
- Net loss for the first quarter of 2024 was $116.6 million, compared to $53.1 million for the same period in 2023.

In summary, CRISPR Therapeutics has made significant strides in advancing its diverse pipeline of gene-based therapies while maintaining a strong financial position. With ongoing and upcoming clinical trials, the company is poised to potentially broaden the reach and impact of its transformative treatments.