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Here are 1-3 brief analogies for CRISPR Therapeutics (CRSP):

• The Moderna of gene editing. (Like Moderna pioneered mRNA technology, CRISPR is a leader in a new, revolutionary platform for medicine.)
• The Tesla of genetic medicine. (Similar to how Tesla disrupted the auto industry with EVs, CRSP is leading a disruptive, high-potential technology in disease treatment.)
• The Intel of genetic code editing. (CRSP is developing the foundational "software" or "toolkit" for rewriting genetic code, much like Intel provides the foundational chips for computing.)

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• Casgevy (exagamglogene autotemcel): An approved gene-edited cell therapy for treating sickle cell disease and transfusion-dependent beta-thalassemia.
• Allogeneic CAR-T Cell Therapies: "Off-the-shelf" gene-edited cell therapies in development for various cancers, including B-cell malignancies and certain solid tumors.
• In Vivo Gene Editing Programs: Therapeutic candidates where CRISPR gene editing is delivered directly into the patient's body to treat genetic diseases like Transthyretin Amyloidosis.

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• Vertex Pharmaceuticals (VRTX): Vertex Pharmaceuticals is CRISPR Therapeutics' key partner for the co-development and global commercialization of Casgevy™ (exagamglogene autotemcel), a gene therapy approved for sickle cell disease and transfusion-dependent beta-thalassemia. Under their agreement, Vertex leads the global commercialization efforts, and CRISPR Therapeutics receives a 40% share of net profits and losses from Casgevy sales in the U.S. and Europe. This profit-sharing arrangement makes Vertex Pharmaceuticals a primary source of revenue for CRISPR Therapeutics from its flagship product.

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Samarth Kulkarni, Ph.D. Chief Executive Officer and Chairman of the Board

Samarth Kulkarni, Ph.D., has served as the Chief Executive Officer of CRISPR Therapeutics since 2017, having joined the company in 2015 in its early stages. As President and Chief Business Officer, he was instrumental in shaping the company's strategy and establishing key partnerships with biopharma companies like Vertex and Bayer. Under his leadership as CEO, CRISPR Therapeutics developed the world's first approved gene-edited treatment for sickle cell disease and transfusion-dependent beta thalassemia. Before joining CRISPR, Dr. Kulkarni was a Partner at McKinsey & Company, where he held a prominent role in the Pharmaceutical and Medical products practice and co-led the biotech practice. He currently serves on the Boards of Directors for Black Diamond Therapeutics, Centessa Pharmaceuticals, Repare Therapeutics, and Oruka Therapeutics, and previously chaired the Board of Directors of Casebia Therapeutics, a joint venture between CRISPR Therapeutics and Bayer.

Raju Prasad, Ph.D. Chief Financial Officer

Raju Prasad, Ph.D., assumed the role of Chief Financial Officer at CRISPR Therapeutics on March 14, 2023. Prior to his tenure at CRISPR, Dr. Prasad spent nine years at William Blair & Company, from March 2014 to March 2023, where he rose to the position of Partner in January 2021. As a senior biotechnology analyst at William Blair, he specialized in covering small-cap, mid-cap, and large-cap companies, and spearheaded the firm's initiative to expand coverage into cell therapy, gene therapy, and gene editing. His earlier career included roles as a research associate at the University of North Carolina at Chapel Hill's Gillings School of Global Public Health and as an independent consultant for the U.S. Environmental Protection Agency. Dr. Prasad also contributes to the advisory board of Portal Innovations, a life sciences venture development engine.

James R. Kasinger General Counsel and Secretary

James R. Kasinger has served as the General Counsel and Secretary of CRISPR Therapeutics since 2017. Before joining CRISPR Therapeutics, Mr. Kasinger held the position of General Counsel and Secretary at Intellia Therapeutics, Inc. from 2015 to 2017. His experience also includes serving as Vice President, Associate General Counsel at Sarepta Therapeutics, Inc. from 2013 to 2015, and as Executive Vice President, General Counsel, and Secretary at AMAG Pharmaceuticals, Inc. from 2007 to 2013. He began his legal career as a corporate and securities associate at Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C. Mr. Kasinger holds a B.A. from Duke University and a J.D. from Boston College Law School.

Naimish Patel, M.D. Chief Medical Officer

Naimish Patel, M.D., became the Chief Medical Officer of CRISPR Therapeutics in 2024. Prior to this role, Dr. Patel served as Executive Vice President, Head of Development at CRISPR Therapeutics. Before joining CRISPR, he was the Chief Medical Officer at Gritstone bio, Inc. from 2018 to 2021. His career also includes leadership positions in clinical development at companies such as AbbVie, Inc., where he was Vice President and Global Head of Immunology Clinical Development from 2016 to 2018, and Genentech, Inc., where he held various roles from 2012 to 2016, including Head of Clinical Development for Immunology, Infectious Diseases, and Ophthalmology. Dr. Patel received his M.D. from the University of Texas Southwestern Medical Center and completed his residency in Internal Medicine at Stanford University Medical Center.

Jon Terrett, Ph.D. Head of Research

Jon Terrett, Ph.D., serves as the Head of Research at CRISPR Therapeutics. Dr. Terrett brings extensive experience in drug discovery and development to the company. Prior to CRISPR Therapeutics, he held various leadership roles in research at other biotechnology and pharmaceutical companies. Notably, he was Vice President, Oncology Research at Merck KGaA from 2012 to 2016. Before that, he spent several years at Celltech R&D (now UCB Pharma), where he held positions of increasing responsibility, including Head of Oncology Research. Dr. Terrett earned his Ph.D. in Chemistry from the University of Cambridge.

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The key risks to CRISPR Therapeutics' business are primarily centered around the complex intellectual property landscape, challenges in commercializing its approved therapy, and inherent risks associated with its clinical development pipeline.

1. Intellectual Property (IP) Disputes and Licensing Complexities: CRISPR Therapeutics operates within a highly intricate and evolving intellectual property framework, characterized by "patent thickets" and ongoing legal disputes over foundational CRISPR-Cas9 technology. These disputes, notably between institutions like the University of California, Berkeley, and the Broad Institute, create significant uncertainty regarding licensing rights and access to key technologies. Navigating these overlapping patents can lead to delays, increased legal costs, and potential infringement risks, thereby impacting the company's ability to develop and commercialize therapies efficiently. Obtaining necessary licensing rights can be prohibitively expensive, involving large upfront fees and annual maintenance fees, which represent substantial sunk costs, especially given the low success rates in pharmaceutical commercialization.
2. Commercialization Challenges and Financial Losses: Despite the groundbreaking approval of Casgevy, the world's first CRISPR-based therapy, its commercialization has been slower than initially anticipated. The complex logistics of cell collection and infusion for this ex vivo therapy have presented patient adoption hurdles, limiting its revenue recognition. CRISPR Therapeutics continues to operate at a significant net loss, with substantial ongoing investment required for its clinical pipeline and commercial infrastructure. This high cash burn and negative earnings per share projections represent a significant financial challenge, raising questions about the company's timeline to profitability.
3. Clinical Trial Risks, Efficacy, and Long-term Safety: As a biotechnology company focused on novel gene-editing therapies, CRISPR Therapeutics faces inherent risks in the clinical development process, including uncertainties regarding the safety and efficacy of its product candidates. Specifically for CRISPR technology, concerns exist about potential long-term safety implications, such as unintentional genetic mutations and off-target effects. Studies have indicated that the CRISPR method, while effective, is not always safe, and cleaved chromosomes may not recover, potentially compromising genomic stability and possibly promoting cancer in the long run. Delays in clinical trial data, regulatory approvals, or adverse events can significantly impact the development timelines and investor confidence.

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The emergence and advancement of alternative, potentially more precise gene-editing technologies, such as prime editing and base editing. These platforms, being actively developed by competitors (e.g., Beam Therapeutics, Verve Therapeutics), offer the potential for superior safety and efficacy compared to CRISPR Therapeutics' primary CRISPR/Cas9 platform, posing a threat to its long-term competitive advantage in gene therapy development.

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CRISPR Therapeutics (CRSP) has several key products and pipeline candidates with significant addressable markets:

• Casgevy (exa-cel): This gene-edited therapy, developed in collaboration with Vertex Pharmaceuticals, is approved for eligible patients 12 years and older with sickle cell disease (SCD) or transfusion-dependent beta-thalassemia (TDT). The initial addressable patient population is estimated to be approximately 7,000 TDT patients and 25,000 SCD patients in the U.S. and Europe. The total target addressable market is estimated at nearly 60,000 patients globally. The market opportunity for Casgevy could be as high as $48 billion (based on 32,000 patients at $1.5 million per patient), with CRISPR Therapeutics' share being $19.2 billion. It is considered a "multibillion-dollar" opportunity. Future advancements, such as targeted conditioning, aim to significantly expand this patient population.
• CTX310 (Cardiovascular Disease - ANGPTL3): This investigational therapy targets ANGPTL3 for cardiovascular diseases. In the U.S. alone, more than 40 million patients are affected by elevated LDL, severely elevated triglycerides, or both, indicating a substantial addressable market. The broader cardiovascular drug market is projected to grow from $228.9 billion in 2025 to $359.6 billion in 2035 globally.
• CTX320 (Cardiovascular Disease - Lp(a)): Targeting lipoprotein(a) or Lp(a), elevated levels of which affect up to 20% of the global population. This program is considered to have "blockbuster potential" due to Lp(a)'s high atherogenicity and the ease of identifying eligible patients.
• CTX112 (Immuno-Oncology and Autoimmune Diseases - Anti-CD19 allogeneic CAR T): This is a next-generation allogeneic CAR T cell therapy targeting CD19+ malignancies and autoimmune diseases. While specific market sizing for CTX112 is not available, it aims to improve upon earlier programs like CTX110. For comparison, other CAR T therapies have reported list prices between $410,000 and $475,000 per dose.
• CTX131 (Immuno-Oncology - Anti-CD70 allogeneic CAR T): This next-generation allogeneic CAR T therapy targets CD70, expressed on various solid tumors and hematologic malignancies. An earlier version, CTX130, was projected to reach annual revenue of $16 million globally by 2040. CTX131 aims for enhanced potency.

The overall global CRISPR gene editing market, which encompasses these therapies, was estimated at $5.72 billion in 2024 and is projected to reach approximately $26.22 billion by 2034, with a compound annual growth rate (CAGR) of 16.54%. North America held the largest share of the global CRISPR technology market in 2024.

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CRISPR Therapeutics (CRSP) is poised for significant future revenue growth over the next 2-3 years, primarily driven by the commercialization of its gene-editing therapies and the advancement of its robust clinical pipeline. The key drivers include:

1. Global Commercialization and Expansion of Casgevy (exa-cel)

   Casgevy, a one-shot gene therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), developed in partnership with Vertex Pharmaceuticals, is the most immediate and significant revenue driver. Approved in late 2023 and early 2024 in the United States and Europe, Casgevy is expected to generate over $100 million in revenues for Vertex in 2025, with significant growth projected for 2026. CRISPR Therapeutics receives a 40% share of the net revenues from Casgevy. Future growth will be driven by expanding patient access and treatment centers, increasing the number of eligible patients, including pediatric populations, and potentially developing a gentler preconditioning regimen that could broaden the addressable patient population.

2. Advancement and Potential Commercialization of Allogeneic CAR-T Cell Therapies

   CRISPR Therapeutics boasts a pipeline of wholly-owned allogeneic (off-the-shelf) CAR-T cell therapies in oncology, including CTX110, CTX112, CTX130, and CTX131. CTX110, targeting CD19+ B-cell malignancies, has shown encouraging preliminary data with efficacy and durability comparable to approved autologous CAR-T therapies and holds a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, potentially accelerating its review. Similarly, CTX130, which targets CD70 for T-cell lymphomas and solid tumors (such as renal cell carcinoma), has demonstrated promising anti-tumor activity and a tolerable safety profile, including a reported complete response in a solid tumor setting. The progression of these candidates through clinical trials and potential regulatory approvals over the next 2-3 years represents significant new product launches and revenue streams.

3. Progression of In Vivo Gene Editing Programs for Common Diseases

   The company is advancing several in vivo gene editing programs, which involve delivering gene-editing tools directly into the body. Key programs include CTX310 and CTX320, targeting ANGPTL3 and Lp(a) respectively, for cardiovascular diseases. CRISPR Therapeutics expects to move multiple in vivo programs into clinical trials within the next 12-15 months. Additionally, new preclinical programs utilizing lipid nanoparticle (LNP)-mediated delivery to the liver for conditions like refractory hypertension and acute hepatic porphyria are being explored. The successful advancement and early positive data from these programs could open up vast new markets and collaborations, contributing substantially to future revenue growth.

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Share Issuance

• CRISPR Therapeutics announced an underwritten public offering in June 2020, which was priced at approximately $450.0 million in gross proceeds, excluding any exercise of the underwriters' option to purchase additional shares.
• In February 2024, the company announced a registered direct offering for the sale of approximately $280 million of its common shares to a select group of institutional investors.
• As of October 2025, CRISPR Therapeutics filed a prospectus supplement to offer and sell up to $600 million worth of common shares from time to time through Jefferies LLC.

Inbound Investments

• CRISPR Therapeutics' largest funding round was a Post IPO round for $900 million in April 2021, which included Vertex Pharmaceuticals as an investor.
• The company has established strategic collaborations with leading companies, including Vertex Pharmaceuticals, for the co-development and commercialization of gene-based medicines like Casgevy, with Vertex now leading global development, manufacturing, and commercialization and splitting program costs and profits 60/40 with CRISPR Therapeutics.
• In December 2023, ARK Investment Management invested in CRISPR Therapeutics during a Post IPO funding round.

Outbound Investments

• In 2025, CRISPR Therapeutics entered into a strategic collaboration with Sirius Therapeutics to co-develop and commercialize novel small interfering RNA (siRNA) therapies for thromboembolic disorders and other serious diseases, sharing costs and profits equally.

Capital Expenditures

• CRISPR Therapeutics supports its immuno-oncology and autoimmune disease efforts with a wholly-owned U.S. manufacturing facility located in Framingham, MA, enabling the production of clinical and commercial-stage good manufacturing practice (GMP) materials for its allogeneic cell therapy programs.
• The company anticipates that its existing cash, cash equivalents, and marketable securities will fund operating expenses and capital expenditures for at least the next 24 months.