CRISPR Corporate Update 44th Annual J.P. Morgan Healthcare Conference January 12, 2026 1 Forward-Looking Statements This presentation and related materials may contain statements regarding matters that are not historical facts and are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Suc ...

Core Insights - CRISPR Therapeutics will present at the 44th Annual J.P. Morgan Healthcare Conference on January 12, 2026, at 8:15 a.m. PT in San Francisco [1] Company Overview - CRISPR Therapeutics is a leading biopharmaceutical company focused on developing gene-based medicines for serious diseases, evolving from a research-stage organization to an industry leader [3] - The company achieved a historic milestone with the approval of CASGEVY (exa-cel), the world's first CRISPR-based therapy for sickle cell disease and transfusion-dependent beta thalassemia [3] - CRISPR Therapeutics has a diversified pipeline targeting hemoglobinopathies, oncology, regenerative medicine, cardiovascular and autoimmune diseases, and rare diseases [3] - The company is advancing its gene editing capabilities through the development of SyNTase™, a proprietary platform for precise and efficient gene correction [3] - Strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals, are established to enhance its impact [3] - The company is headquartered in Zug, Switzerland, with R&D operations in Boston, Massachusetts, and San Francisco, California [3]

Core Viewpoint - The National Advertising Division (NAD) is referring claims made by Maze Therapeutics regarding their drug MZE829 to regulatory authorities due to Maze's failure to respond substantively to NAD's inquiry [1][3]. Group 1: Company Overview - Vertex Pharmaceuticals and Maze Therapeutics are competitors in the development of clinical-stage treatments for APOL1-Mediated Kidney Disease (AMKD) [2]. - Maze Therapeutics has made express and implied claims about the efficacy of MZE829 compared to Vertex's drug candidate inaxaplin [2]. Group 2: Regulatory Actions - The NAD's inquiry was prompted by Vertex's request to review Maze's claims about MZE829 [2]. - Maze Therapeutics declined to participate in the NAD process, stating that it does not currently sell any product or advertise MZE829 [3]. - Due to Maze's non-participation, NAD will refer the matter to appropriate government agencies for further action [3].

CASGEVY & Hemoglobinopathies - CASGEVY, a CRISPR-Cas9 therapy for severe sickle cell disease and beta thalassemia, is approved in multiple jurisdictions, with >65 authorized treatment centers activated globally and ~90 patients having initiated cell collection as of May 1st, 2025[9, 23] - The addressable market for CASGEVY is approximately 60,000 severe patients in approved territories[19] - A new CMMI model aims to improve access and health outcomes for sickle cell disease, potentially reducing U S expenditures by $3 billion annually[21] CAR T Programs - CTX112, an allogeneic CAR T therapy, demonstrated an overall response rate (ORR) of 67% and a complete response rate (CR) of 50% in a Phase I/II trial (N=12) for relapsed or refractory B-cell malignancies[40] - Updated CTX112 data (N=25) showed cell expansion comparable to autologous CAR T therapies, with dose-dependent increases in AUC and Cmax[43, 46] - In a subset of patients receiving CTX112 post-T cell engager (TCE) therapy, the overall response rate was 100% (6 patients)[48] In Vivo Programs - Initial results from the CTX310 Phase 1 dose escalation trial (N=10) showed promising efficacy, with one patient at the 0 8 mg/kg dose level experiencing an 82% reduction in triglycerides from a baseline of 1073 mg/dL at day 30[62, 66] - Preclinical data for CTX320 in NHPs demonstrated ~70% editing of LPA and ~95% reduction in plasma Lp(a) sustained at 2 years at 2 mg/kg dose[73] - CTX320 has the potential to benefit >60 million U S patients with elevated Lp(a)[81] Financials & Outlook - The company has a strong balance sheet with approximately $1 86 billion[15]

Core Insights - Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) has experienced a modest stock price increase of 4% since mid-December, indicating a period of recovery after facing challenges [2]. Company Overview - Vertex Pharmaceuticals is based in Boston and is recognized as a significant player in the pharmaceutical industry [2]. - The company has been under the coverage of a biotech consultant with over 5 years of experience in the sector, who has produced detailed reports on more than 1,000 companies [2]. Investment Insights - The investment group Haggerston BioHealth offers resources for both novice and experienced biotech investors, including catalysts to monitor, buy and sell ratings, and forecasts for product sales across major pharmaceutical companies [2]. - The group also provides integrated financial statements, discounted cash flow analysis, and market-by-market analysis, which are essential for informed investment decisions [2].