Vertex Pharmaceuticals

Reinventing gene therapies with next-generation technologies

Human Gene Editing

<div>
 
 CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, the company had 18 drug candidates in its pipeline. 
 
 
 In a milestone event in November 2023, the company&rsquo;s gene-editing therapy exagamglogene autotemcel, CASGEVY 
 <a href="https://sp-edge.com/updates/24093">
 (exa-cel)
 </a>
 , developed with Vertex Pharmaceuticals to treat sickle-cell disease and beta-thalassemia, received the world&rsquo;s first authorization from the UK's Medicines and Healthcare products, Regulatory Agency (MHRA). In December 2023, it received FDA approval for patients aged 12 and older. The drug for beta-thalassemia patients is also nearing its market entry in the US, with the FDA&rsquo;s decision expected on March 30, 2024.&nbsp;
 
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured strategic collaborations with several companies to co-develop and co-commercialize gene editing medicines that include 
 <a href="https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-and-crispr-therapeutics-amend">
 Vertex Pharmaceuticals
 </a>
 (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and 
 <a href="https://viacyte.com/press-releases/crispr-therapeutics-and-viacyte-announce-strategic-collaboration-to-develop-gene-edited-stem-cell-derived-therapy-for-diabetes/">
 ViaCyte
 </a>
 (September 2018) to develop VCTX210, a regenerative medicine for diabetes.
 
 
 Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.&nbsp;&nbsp;
 
 
 
 
 
 Funding and financials
 
 
 
 
 Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.
 
 
 
 
 
</div>

<div>
 
 Arbor Biotechnologies specializes in gene editing to develop novel therapeutics for a variety of indications, starting with liver-related diseases. It operates an AI-powered biodiscovery platform to discover novel Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) enzymes, which are sequences of repeating genetic code. To do this, its platform deploys computational search techniques, genome sequencing, gene synthesis, and high-throughput screening. This can eventually be turned into drugs or even applied to agriculture and other industrial uses. 
 
 
 As of March 2024, the company had five drug discovery programs, one was in IND-enabling stage, while two were in the lead optimization stage, and two were in discovery stages. In 2018, the company announced the discovery of Cas13d, a new type of gene editing enzyme that helps to alter RNA to treat genetic diseases.&nbsp;
 
 
 Key customers and partnerships
 
 
 Most recently, in 
 <a href="https://www.globenewswire.com/news-release/2024/01/03/2803274/0/en/4DMT-and-Arbor-Biotechnologies-Establish-Partnership-to-Co-Develop-and-Co-Commercialize-Next-Generation-Genetic-Medicines-for-CNS-Diseases.html">
 January 2024
 </a>
 , the company announced a strategic collaboration with 4D Molecular Therapeutics that would focus on advancing new AAV-based gene-editing therapies for central nervous system (CNS) diseases with high unmet medical needs in both rare and common disease populations.
 
 
 The company entered into a strategic partnership worth USD 1.2 billion with Vertex Pharmaceuticals in 
 <a href="https://www.biospace.com/article/vertex-and-arbor-strike-a-1-2-billion-deal-for-gene-editing-programs/">
 August 2021
 </a>
 to develop ex vivo therapies (gene editing done in a specialized laboratory and returned to the body). The duo originally partnered in 
 <a href="https://investors.vrtx.com/news-releases/news-release-details/vertex-and-arbor-biotechnologies-establish-collaboration">
 2019
 </a>
 to develop proteins for gene-edited therapies. The companies expanded this partnership in 
 <a href="https://arbor.bio/arbor-biotechnologies-announces-expanded-strategic-partnership-with-vertex-now-extending-to-precision-gene-editing-using-reverse-transcriptase/">
 January 2023
 </a>
 , to give Vertex Pharmaceuticals the rights to Arbor&rsquo;s novel precision editing technology for up to three diseases.
 
 
 Arbor also entered into a non-exclusive licensing agreement with 
 <a href="https://www.globenewswire.com/news-release/2022/02/08/2380680/0/en/EdiGene-and-Arbor-Biotechnologies-Announce-Worldwide-Non-exclusive-License-Agreement-to-Advance-Ex-Vivo-Engineered-Cell-Therapy-Programs.html">
 EdiGene Inc
 </a>
 . in February 2022 to leverage Arbor&rsquo;s platform for ex vivo oncology therapies and a strategic research and non-exclusive licensing agreement with 
 <a href="https://investors.tcr2.com/news-releases/news-release-details/tcr2-therapeutics-and-arbor-biotechnologies-establish">
 TCR2 Therapeutics
 </a>
 in January 2022 to develop cell therapies for the latter.&nbsp;
 
 
 Funding and financials
 
 
 In its latest Series B funding round in November 2021, which was led by Ally Bridge Group, TCG Crossover, and Temasek, Arbor Biotechnologies raised USD 215 million. The proceeds of the round were earmarked to fuel the company&rsquo;s clinical development pipeline. 
 
</div>

Artificial intelligence is identifying and testing new therapies faster than ever.

AI Drug Discovery

<div>
 
 Arbor Biotechnologies specializes in gene editing to develop novel therapeutics for a variety of indications, starting with liver-related diseases. It operates an AI-powered biodiscovery platform to discover novel CRISPR enzymes, which are sequences of repeating genetic code. To do this, its platform deploys computational search techniques, genome sequencing, gene synthesis, and high-throughput screening. This can eventually be turned into drugs or even applied to agriculture and other industrial uses. In 2018, the company announced the discovery of Cas13d, a new type of gene editing enzyme that helps to alter RNA to treat genetic diseases.&nbsp;
 
 
 As of February 2024, the company had five internal drug discovery programs, of which one was in the IND-enabling stage. It also has multiple collaborative programs under development focused on oncology and various other therapeutic areas.
 
 
 Key customers and partnerships
 
 
 The company has a long-standing partnership with Vertex Pharmaceuticals since
 <a href="https://www.businesswire.com/news/home/20190103005194/en/Vertex-and-Arbor-Biotechnologies-Establish-Collaboration-to-Discover-Novel-Proteins-to-Advance-Discovery-of-Gene-Editing-Therapies">
 January 2019
 </a>
 , which focused on developing gene-editing treatments for cystic fibrosis and four other diseases. The alliance was further extended in 
 <a href="https://www.biospace.com/article/vertex-and-arbor-strike-a-1-2-billion-deal-for-gene-editing-programs/">
 August 2021
 </a>
 and 
 <a href="https://sp-edge.com/updates/15741">
 January 2023
 </a>
 .&nbsp;
 
 
 Other partnerships included a collaboration with 
 <a href="https://www.prnewswire.com/news-releases/arbor-biotechnologies-announces-collaboration-with-ginkgo-bioworks-to-advance-the-discovery-and-development-of-precision-gene-editors-302011991.html">
 Ginkgo Bioworks
 </a>
 (December 2023) to leverage Ginkgo's R&amp;D services to further optimize Arbor's novel precision editors, a non-exclusive licensing agreement with 
 <a href="https://www.globenewswire.com/news-release/2022/02/08/2380680/0/en/EdiGene-and-Arbor-Biotechnologies-Announce-Worldwide-Non-exclusive-License-Agreement-to-Advance-Ex-Vivo-Engineered-Cell-Therapy-Programs.html">
 EdiGene
 </a>
 (February 2022) to leverage Arbor&rsquo;s platform for ex vivo oncology therapies, a strategic research and non-exclusive licensing agreement with 
 <a href="https://investors.tcr2.com/news-releases/news-release-details/tcr2-therapeutics-and-arbor-biotechnologies-establish">
 TCR2 Therapeutics
 </a>
 (January 2022) to develop allogeneic TRuC T-cell therapies using Arbor&rsquo;s CRISPR technology, and a license agreement with 
 <a href="https://www.lonza.com/news/2020-12-23-14-00">
 Lonza
 </a>
 (December 2020) to use Arbor&rsquo;s gene-editing technology for Lonza&rsquo;s bioproduction products and services.
 
 
 Funding and financials
 
 
 In its latest Series B funding round in November 2021, which was led by Ally Bridge Group, TCG Crossover, and Temasek, Arbor Biotechnologies raised USD 215 million. The proceeds of the round were earmarked to fuel the company&rsquo;s clinical development pipeline.
 
</div>

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Obsidian Therapeutics is a provider of cell and gene therapeutics used to treat tumors and skin cancer in patients. The company uses its proprietary cytoDRIVE platform to develop its therapeutics by using drug responsive domains (DRDs) to control protein functions using an FDA-approved small molecule. The platform uses Obsidian’s library of DRDs and biology engineering toolkit, which is used to optimize protein functionality in any cell type, including I/II membrane proteins, membrane-tethered cytokines, intracellular proteins, secreted proteins, and genome editing proteins.As of May 2024, the company had one lead candidate, OBX-115, a tumor-infiltrating lymphocyte (TIL) for treating melanoma and lung cancer. The company was planning to carry out three studies (a single center and two multicenter studies), all of which were in Phase I of clinical trials. The company was also developing a next generation TIL for an undisclosed application, which was still in discovery stages. The company also had two partnered programs, one with Bristol Myers Squibb and the other with Vertex, for two undisclosed applications.Key customers and partnershipsObsidian partnered with biotechnology company Vertex Pharmaceuticals in April 2022 to collaborate on discovering and developing therapies that regulate gene editing for treating serious diseases. Through the partnership, Obsidian would provide its cytoDRIVE platform so that Vertex could develop genetic therapies. Vertex would pay Obsidian USD 75 million in equity, upfront payments, and potential research milestone payments.Obsidian also extended its multi-year partnership with pharmaceutical company Bristol Myers Squibb in October 2022 to continue to develop cell therapies. Originally partnering in 2020, the partnership entailed Bristol Myers Squibb developing cell therapies while using Obsidian’s cytoDRiVE platform.Funding and financialsObsidian last raised funds in September 2021, where the company secured USD 115 million in a Series B funding round led by The Column Group Crossover Fund, along with participation from other venture capital and pharmaceutical firms including both Bristol Myers Squibb and Vertex Pharmaceuticals. The raised funds were set aside to advance its lead tumor infiltrating lymphocyte (TIL) program into clinics while also diversifying the therapeutic to target other lymphocytes.

Arbor Biotechnologies is an operator of a bio-discovery company intended to provide human diagnostic development services. The company's platform employs a diverse set of technologies and techniques including artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, enabling drug developers to accelerate the discovery of proteins for improving human health and sustainability.

Kymera Therapeutics is a biotechnology company that specializes in the field of targeted protein degradation. It features a proprietary predictive modeling degradation platform with a novel small molecule modality to accelerate the drug discovery designed for disease-causing proteins.

Genomics plc aiming to lead the genomic transformation of healthcare, to develop and apply sophisticated analyses of massive integrated genomic and phenotypic datasets to learn about human biology and reveal the human wiring diagram They will lead the way through this challenge, using their expertise and experience to unleash the potential of genomics and set the standards by which organisations and patients can benefit from genomic data.

Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.

CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.

Vertex Pharmaceuticals is a biotechnology company committed to the discovery and development of breakthrough small molecule drugs for serious diseases. The Company's strategy is to commercialize its products both independently and in collaboration with major pharmaceutical companies. Vertex's product pipeline is focused on viral diseases, cystic fibrosis, inflammation, autoimmune diseases, cancer, and pain.

<div> Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) is a global biotechnology company focused on developing innovative therapies for serious diseases. Founded in 1989 and headquartered in Boston, Massachusetts, Vertex has become a leader in treatments for cystic fibrosis (CF) and is expanding into other therapeutic areas. Vertex's core business revolves around its CF franchise, which includes approved medicines like Trikafta/Kaftrio, Symdeko/Symkevi, Orkambi, and Kalydeco. These drugs target the underlying cause of CF and have established Vertex as the dominant player in this market. The company's pipeline includes next-generation CF therapies as well as investigational treatments for conditions such as sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, type one diabetes, and pain management. In 2023, Vertex received approval for Casgevy, a gene therapy for sickle cell disease and transfusion-dependent beta thalassemia developed in partnership with CRISPR Therapeutics. Vertex has demonstrated strong financial performance, with product revenue reaching USD 2.69 billion in Q1 2024, a 13% year-over-year increase. The company has reiterated its full-year 2024 revenue guidance of USD 10.55 billion to USD 10.75 billion. Vertex continues to invest heavily in research and development, spending USD 789.1 million in Q1 2024 alone. With a market capitalization of over USD 110 billion and a robust pipeline of potential new therapies, Vertex remains well-positioned for future growth in the biotechnology sector. </div>

Vertex Pharmaceuticals Shares Drop on Mixed Results in Drug Trial

Companies that develop novel therapeutics based on the CRISPR/Cas9 gene-editing technology

Companies that develop novel therapeutics based on other types of gene editing technologies including TALEN and ZFN

Companies that develop systems that enable the diagnosis of gene-edited therapies. Also includes systems that deliver gene-editing components into the human body

Companies that offer their gene-editing platforms to other pharma companies to develop therapeutics

Companies that offer support services to gene-editing companies, which include genomic research companies, gene-editing component manufacturing, and designing tools

ANALYST_QUICK_TAKE

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

<div>

Vertex Pharmaceuticals has selected Orsini as the distributor for their gene-editing therapy, CASGEVY (exagamglogene autotemcel), but financial details of the partnership were not revealed.
This therapy uses CRISPR/Cas9 gene-editing technology, aimed at treating patients aged 12 and above who are suffering from sickle cell disease with recurring vaso-occlusive crises or transfusion-dependent β-thalassemia.

</div> Analyst QuickTake: 
 CASGEVY (developed by Vertex and CRISPR Therapeutics) is the world’s first FDA-approved gene editing therapy, receiving FDA approval in <a href="https: 
//sp-edge.com/updates/24785"> December 2023 </a> and market authorization in the UK in <a href="https: 
//sp-edge.com/updates/24093"> November 2023 </a> . Other similar treatments include Bluebird Bio’s one-time gene therapy LYFGENIA (sickle cell disease) and beti-cel (Zynteglo) (beta thalassemia) and Beam Therapeutics’ BEAM-101, an investigational gene-editing therapy currently in clinical trials for sickle cell disease.

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