CRISPR Therapeutics, Inc.

CRISPR Therapeutics announced its senior management will present at the 43rd Annual J.P. Morgan Healthcare Conference on January 14, 2025. The company, known for its transformative gene-based medicines, has a diverse portfolio and has achieved historic approvals for CRISPR-based therapies, including CASGEVY™ for sickle cell disease and beta thalassemia.

Curevo Vaccine, Myriad Genetics, Vericel Corporation, and CRISPR Therapeutics will present at the 43rd Annual J.P. Morgan Healthcare Conference on January 15, 2025. PacBio ships Vega systems to Berry Genomics. PrognomiQ appoints Joel Kaufman as CFO. Vor Bio appoints Erez Kalir to its Board.

Vertex Pharmaceuticals partners with Orna Therapeutics to develop in vivo gene therapies for sickle cell disease and beta thalassemia, paying $65M upfront with potential for $635M more. This collaboration aims to improve on current CRISPR therapy Casgevy by enabling gene editing inside the body, avoiding toxic preconditioning and lengthy manufacturing.

Capsida Biotherapeutics receives a $40M license payment from AbbVie for a neurodegenerative disease program, with potential for more milestones and royalties. The collaboration leverages Capsida's AAV technology for targeted CNS therapy delivery, aiming to overcome traditional AAV challenges. Capsida also plans to advance its own CNS programs into clinical trials by mid-2025.

CRISPR Therapeutics and Vaxcyte are compared based on analyst recommendations, dividends, earnings, risk, valuation, institutional ownership, and profitability. CRISPR has higher revenue and earnings, more institutional ownership (69.2% vs. Vaxcyte's 96.8%), and a higher potential upside (89.99% vs. 71.71%). CRISPR beats Vaxcyte on 10 of 13 factors.

75% of C-suite executives in life sciences are optimistic about 2025, citing growth and innovation. AI in biopharma is expanding, with increased investments and partnerships. Cancer vaccines and gene editing are advancing, with late-stage trials and new therapies. VC and IPO markets are rebounding, and M&A activity is expected to increase. Multiomics tools are expanding, and spatial biology is consolidating. Regulatory changes under Trump 2.0 may impact biopharma and health policy.

In 2024, significant progress in gene and cell therapies for hematological disorders was noted, including Canada's approval of Casgevy for SCD and TDT, and Beqvez for hemophilia B. Despite FDA approvals, uptake of SCD gene therapies remained slow. Allo-HSCT showed positive outcomes for TDT, and a SCD patient died from busulfan-conditioning complications in a BEAM-101 trial.

The Global Cancer Gene Therapy Market is projected to grow from US$ 3.6 Billion in 2023 to US$ 16 Billion by 2033, at a CAGR of 16.1%. North America leads with a 36.5% market share. Innovations like CRISPR and CAR T-cell therapies, alongside AI in drug development, are driving growth.