CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Caris Life Sciences | March 09, 2023
Caris Life Sciences®, a leading molecular science and technology firm and Incyte Corporation have recently announced a strategic research partnership to advance precision medicine approaches for Incyte's oncology pipeline.
Caris' innovative technology integrates data from whole exome sequencing, protein analysis, whole transcriptome sequencing and proprietary AI models and signatures to enhance patient outcomes through personalized medicine development. The partnership will initially apply Caris' data and analytics tools, comprehensive molecular liquid and tissue profiling services, and clinical trial enrollment program capabilities spans two therapeutic programs, with the option to expand to four total programs.
Incyte will use Caris' data insights and analytics capabilities to uncover novel biomarkers and enhance clinical positioning strategies for its oncology initiatives. Patients enrolling in Incyte-led clinical trials will be subjected to longitudinal testing with Caris' comprehensive tissue and liquid molecular profiling tests for drug candidates developed as part of the agreement. Incyte will also use Caris' biomarker-driven patient selection for clinical trials, with the possibility of collaborating on creating companion diagnostics for the partnership's programs.
Caris Life Sciences President and Chief Scientific Officer David Spetzler, MS, Ph.D., MBA, commented, "This partnership with Incyte will leverage Caris' leading molecular science and technology solutions to support Incyte's oncology research and development efforts." He further emphasized, "The aggregate strength of our platform, which provides patient level DNA and RNA data both in tissue and blood, may help to better identify and predict patient response to therapy, which in turn may accelerate clinical trial enrollment, optimize clinical positioning and potentially enhance technical and regulatory success."
(Source – PR Newswire)
About Caris Life Sciences
Caris Life Sciences® is a leading molecular science firm that utilizes advanced technologies to revolutionize healthcare. Its suite of molecular profiling offerings, which assess RNA, DNA and proteins, enables patients, researchers and physicians to improve outcomes and save lives by revealing a molecular blueprint. Additionally, it is advancing precision medicine through the use of advanced AI and machine learning algorithms by utilizing its proprietary analytics engine, DEAN™, to develop a better understanding of the molecular mechanisms of cancer. This information helps physicians to better identify and predict patient response to therapy and supports researchers and biopharmaceutical companies in research development, clinical trials, and target identification. With headquarters in Irving, Texas, the company has offices in several locations worldwide, providing services throughout the US, Europe, Asia, and other international markets.
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CELL AND GENE THERAPY, DIAGNOSTICS
Businesswire | March 28, 2023
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics’ gene editing technology, known as CRISPR/Cas9, to accelerate the development of Vertex’s hypoimmune cell therapies for type 1 diabetes (T1D).
“We have multiple programs in our T1D portfolio including VX-880 and VX-264, which are in the clinic, as well as our hypoimmune program, in preclinical development,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “Having successfully demonstrated clinical proof of concept in T1D in our VX-880 program, we are excited to deepen our relationship with CRISPR Therapeutics with this agreement, which will allow us to further accelerate our goal of generating fully differentiated, insulin-producing hypoimmune islet cells for T1D.”
“We are pleased to expand our long and successful relationship with Vertex with this collaboration which fully leverages our gene editing platform to develop hypoimmune cell therapies for T1D,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “In parallel, we continue to expand our capabilities in regenerative medicine and advance our existing allogeneic gene-edited cell therapy programs.”
Under this agreement, Vertex will pay CRISPR Therapeutics $100 million up-front for non-exclusive rights to CRISPR Therapeutics’ technology for the development of hypoimmune gene-edited cell therapies for T1D. CRISPR Therapeutics will be eligible for up to an additional $230 million in research and development milestones and receive royalties on any future products resulting from this agreement.
CRISPR and ViaCyte, Inc., which was acquired by Vertex in 2022, will continue to collaborate on their existing gene-edited allogeneic stem cell therapies, using ViaCyte cells, for the treatment of diabetes under the terms of their collaboration. A Phase 1/2 study of VCTX211, an allogeneic, gene-edited, stem cell-derived product candidate for T1D, which originated under the CRISPR Therapeutics and ViaCyte collaboration, has been initiated and is ongoing. CRISPR Therapeutics will not obtain any interest in Vertex’s pre-existing pipeline of T1D products, including VX-880 and VX-264.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, and alpha-1 antitrypsin deficiency.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune’s Best Workplaces in Biotechnology and Pharmaceuticals and Best Workplaces for Women.
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INDUSTRY OUTLOOK
Globenewswire | May 15, 2023
TriLink BioTechnologies a Maravai LifeSciences company and global provider of life science reagents and services, has launched its newest cap analog, CleanCap® M6 . The latest CleanCap analog is expected to help developers and researchers maximize the impact of their mRNA therapeutics and vaccines while reducing overall manufacturing costs, bringing life-changing medicines to market faster.
With the introduction of the CleanCap M6 technology, legacy capping methods like enzymatic and ARCA may be a thing of the past. By combining the modification of 3’OMe on the m7-Guanosine and the additional methyl modification on the +1 Adenosine, the CleanCap M6 analog is expected to improve mRNA potency with the highest protein expression of any CleanCap analog yet. Combined with technology’s single-pot reaction capabilities that shrink manufacturing times and increase mRNA yields with a purity profile of >95%, CleanCap analogs are novel mRNA Cap structures, which may improve the benefits of an mRNA therapeutic or vaccine.
“TriLink’s portfolio of CleanCap mRNA capping technology is expected to be the new go-to capping method in the very near future - it is the future,” shared Kate Broderick Ph.D., Chief Innovation Officer at Maravai LifeSciences. “What excites me most about this novel analog is its ability to make mRNA an even more powerful therapeutic than we have seen previously.”
The company’s first CleanCap analog launched in 2017 and this capping technology has been incorporated in one of the first commercially approved COVID-19 vaccines. With the introduction of CleanCap M6, TriLink has developed its most robust analog to date. Studies have shown that the innovative product increases mRNA expression by 30% or more versus enzymatic capping methods and is estimated to reduce manufacturing costs by 20-40%.
“mRNA is clearly an emerging therapeutic modality where mRNA vaccines and therapeutics are expanding rapidly,” added Drew Burch, EVP of TriLink’s Nucleic Acid Products division. “CleanCap M6, and the entire CleanCap portfolio, joins a strong roster of synthesized nucleic acids, NTPs, and more that we expect to continue to grow.”
In addition to the CleanCap mRNA capping portfolio, TriLink has scale-up expertise and unique mRNA, oligonucleotide & plasmid production capabilities for companies focused on therapeutic, vaccine, diagnostic breakthroughs, leaning on its 25-plus years of experience to provide superior CDMO services.
Data and customer insights garnered from a market assessment conducted by a third-party consulting firm on behalf of TriLink BioTechnologies in November 2022.
About Maravai
Maravai is a leading life sciences company providing critical products to enable the development of drug therapies, diagnostics, and novel vaccines. Maravai’s companies are leaders in providing products and services in the fields of nucleic acid synthesis and biologics safety testing to many of the world’s leading biopharmaceutical, vaccine, diagnostics, and cell and gene therapy companies.
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