CELL AND GENE THERAPY, MEDICAL
Be The Match BioTherapies® | January 18, 2023
On January 17, 2023, Be The Match BioTherapies, a company that provides solutions for organizations that develop and commercialize cell and gene therapies, introduced its offering of CIBMTR® clinical research organization services (CRO services), which significantly reduces the time needed to launch and execute high-impact clinical trials.
These CRO services are the outcome of a collaboration between Be The Match BioTherapies and CIBMTR, resulting in expertise and access to unmatched research, sites, donors, partnerships, scientific and operational knowledge, and other resources.
CIBMTR CRO services offer unique capabilities such as
Established relationships with over 225 clinical locations domestically
Nearly 20 years of expertise in transplant and cellular therapy trials and study management, as well as clinically-trained staff
On-staff transplant physicians provide direct contact with patients and donors for clinical and research insights.
A link to the CIBMTR outcomes database for over 575,000 patients
The new CRO services offer turn-key solutions, including protocol development and approval oversight, site selection, project management, site start-up, research sample/laboratory coordination, data management, study monitoring, patient-reported outcomes, statistical analysis, and financial administration. For certain services, such as data analysis, surveys, site selection and administration, and sample management, businesses may instead rely on the expertise of CIBMTR CRO services.
About Be The Match BioTherapies®
Be The Match BioTherapies is the sole supplier of cell and gene therapy solutions with fully configurable services to assist the cell therapy supply chain. It is committed to accelerating patient access to life-saving cell and gene treatments by delivering the best-quality cellular source material from Be The Match Registry®, the most-varied registry of prospective blood stem cell donors in the world, with over 7 million potential blood stem cell donors. Through global collaborations with apheresis, bone marrow collection, and transplant centers, the firm creates, onboards, trains, and maintains extensive collection networks to advance cell treatments.
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.
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.
MEDTECH, INDUSTRIAL IMPACT
SOPHiA GENETICS and QIAGEN | March 06, 2023
SOPHiA GENETICS, a healthcare cloud-based software company, announced a new partnership with QIAGEN N.V. that will pair QIAseq reagent technology with the SOPHiA DDM™ platform to improve tumor analysis via next-generation sequencing (NGS).
Customers can utilize QIAseq panels with SOPHiA DDM™, a cloud-based data analysis platform to enable new research perspectives. The partnership will support the detection of somatic variants using QIAseq targeted DNA pro panels for homologous recombination repair. HRR is a biomarker test that helps identify tumors with specific mutations that may be sensitive to PARP inhibitors in oncology.
SOPHiA GENETICS's Set-Up Program, which aims to establish and demonstrate any test's analytical performance before it is carried out, will be integrated with QIAGEN's kits to support a range of applications. This partnership will allow customers to improve the design of new workflows using QIAseq technologies, while SOPHiA DDM™ will offer extensive applications for QIAGEN kits.
QIAGEN intends to expand its partnership with SOPHiA GENETICS to other analysis areas, such as utilizing custom solutions and QIAseq panels. This partnership is the first one in QIAGEN's QIAseq Platform Partnership.
Next-generation sequencing (NGS) can transform the detection and management of rare diseases and cancers. However, it also generates large, intricate, and messy datasets that are challenging to analyze. Identifying causal variants and getting valuable insights from NGS data requires specialized expertise, time, and numerous resources. The SOPHiA DDM™ Platform is a machine learning-based analytical tool that can efficiently call, annotate, and pre-classify variants from raw NGS data. In addition, it features user-friendly capabilities to facilitate prioritization, interpretation and reporting.
QIAGEN is a global provider of sample and assay technologies for academic and pharmaceutical research, molecular diagnostics, and applied tests. In addition, the firm offers a wide range of products and solutions for the isolation, purification, and analysis of DNA, RNA, and proteins, as well as automation and bioinformatics services. With over 35 countries and over 5,000 employees, QIAGEN serves customers in various fields such as human healthcare, animal health, and environmental testing. The company is headquartered in Venlo, Netherlands, and has offices and facilities across the globe.