CRISPR Therapeutics AG

Lawsuit filed against Lonza, the Dutch manufacturer of the world's first CRISPR-based genome editing therapy, CASGEVY Second European action following UK litigation, enforcing ToolGen's rights to fundamental CRISPR-Cas9 RNP technology SEOUL, South Korea, Sept. 8, 2025 /PRNewswire/ -- ToolGen, Inc. (KOSDAQ: 199800), a global leader in genome editing technology, today announced that it has filed a patent infringement lawsuit against Lonza Netherlands B.V., a contract development and manufacturing organization (CDMO) that produces CASGEVY, the world's first approved CRISPR-based gene therapy. The case was filed with the District Court of The Hague. The lawsuit charges that Lonza, in its production of Vertex's CASGEVY at its Dutch facility, infringes ToolGen's European patent (EP 4 357 457) covering CRISPR-Cas9 RNP technology. CASGEVY, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is the world's first gene-editing therapy approved for sickle cell disease and beta-thalassemia. It has been authorized in the United Kingdom, United States, and European Union. Under a long-term supply agreement with Vertex, Lonza manufactures CASGEVY at its Netherlands site. This Dutch action follows ToolGen's UK lawsuit filed in April against parties involved in CASGEVY's sale and production. By targeting a key European production hub, ToolGen aims to reaffirm the importance of its foundational CRISPR-Cas9 RNP technology and strengthen protection of its intellectual property rights. "Behind the successful commercialization of CASGEVY lies the pivotal role of ToolGen's proprietary CRISPR-Cas9 RNP platform," said Jong Sang Ryu, CEO of ToolGen. "Toolgen filed this lawsuit because it cannot accept the unauthorized use of its proprietary technology. The lawsuit is not intended to restrict patient access to life-changing therapies, but to secure fair recognition and just compensation for the value of ToolGen's innovations. We remain open to exploring an amicable resolution of the dispute, ultimately fostering a sustainable and mutually beneficial ecosystem." Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Vertex Pharmaceuticals has tapped Enlaza Therapeutics' War-Lock platform to develop an improved conditioning agent for its blood disorder gene therapy, among other potential programmes. The deal, announced Tuesday, will provide Enlaza with $45 million between an upfront payment and equity investment, with Vertex on the hook for potentially more than $2 billion in R&D, regulatory and commercial milestones, plus royalties.Vertex markets CRISPR-edited gene therapy Casgevy (exagamglogene autotemcel) — developed in partnership with CRISPR Therapeutics — for sickle cell disease and beta thalassemia. Despite the product's slow uptake, Vertex has pegged the gene therapy as a potential blockbuster (see – Spotlight On: Vertex plots $1B+ sales trajectories for Casgevy, Journavx).Before taking Casgevy, patients currently are required to receive a chemotherapy conditioning regimen during a four-to-six-week hospital stay, which could pose a major barrier to treatment."Vertex is committed to developing transformative therapies for serious diseases…including certain autoimmune diseases and gentler conditioning for Casgevy," said Mark Bunnage, SVP of global research at Vertex. "We look forward to exploring the potential of Enlaza’s War-Lock platform to help us achieve these goals."Enlaza's covalent biologics platform is intended to enable irreversible protein binding, boosting a drug’s biologic effect without the need for long-term systemic exposure. War-Lock adds unnatural amino acids to virtually any protein drug, enabling them to form covalent bonds with their target. The biotech scored a $100-million series A last April, adding to its $61-million debut in late 2022.Along with the conditioning treatment, Enlaza is on tap to develop small format drug conjugates and T-cell engagers for certain autoimmune diseases."By partnering with Vertex…we gain a valued partner to help unlock the potential of covalent protein drugs, opening new markets and accelerating development of differentiated biologics with best-in-class potential," said Enlaza CEO Sergio Duron.Enlaza will be responsible for all research activities up until a development candidate is nominated, after which Vertex will subsequently lead all future R&D, manufacturing and commercialisation. It will also provide all R&D funding during the four-year collaboration.

iStock, Alona Horkova Vertex Pharmaceuticals commits $45 million upfront to leverage Enlaza Therapeutics’ War-Lock platform to create drug conjugates and T cell engagers for autoimmune diseases and gentler conditioning for sickle cell/beta thalassemia gene-editing therapy Casgevy. The immunology and inflammation space continues to fire on all cylinders as Vertex Pharmaceuticals and Enlaza Therapeutics locked in a front-loaded pact to develop small format drug conjugates and T cell engagers for autoimmune diseases and improved conditioning in patients with sickle cell disease or beta thalassemia who are treated with Vertex’s gene-editing therapy Casgevy. The deal will see Enlaza receive $45 million upfront, including an equity investment, and a potential $2 billion more in future milestone payments. Vertex is paying to leverage the company’s proprietary War-Lock platform, which creates highly specific warheads that covalently bind to drug targets of interest, according to a Tuesday press release announcing the collaboration. This “white-space” technology generates protein drugs that retain the selectivity of small format biologics while expanding the therapeutic window and enabling more effective therapies across numerous therapeutic modalities, according to Enlaza. So far, the platform “has demonstrated compelling promise in oncology, and this strategic collaboration marks a key inflection point as we expand into autoimmune indications,” Enlaza CEO Sergio Duron said in a prepared statement on Tuesday. On the Vertex side, Mark Bunnage, senior vice president of Global Research, said in a statement that the company “is committed to developing transformative therapies for serious diseases that fit within our unique R&D strategy including certain autoimmune diseases and gentler conditioning for Casgevy, the first and only approved CRISPR/Cas9 gene-edited therapy.” Casgevy—which Vertex developed with partner CRISPR Therapeutics— won FDA approval in December 2023 for sickle cell disease and for beta thalassemia in January 2024. La Jolla, California–based Enlaza, which bills itself as “the first covalent biologics platform company,” raised $100 million in series A financing in April 2024. This followed the company’s December 2022 launch, in which it closed on $61 million in seed financing to build a pipeline of covalent biologics with an initial focus on cancer. The biotech’s website does not currently list a pipeline. The immunology and inflammation (I&I) space —which includes autoimmune diseases—has seen a tremendous amount of investment activity during the past couple of years, with big names like Sanofi and Gilead making major splashes. In July alone, GSK bet up to $12 billion in a pact with China’s Hengrui Pharma to advance up to 12 novel therapies for respiratory, immunology and oncology indications, and Bristol Myers Squibb spun out a new company with five immunology assets and $300 million in funds from Bain Capital.