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.
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INDUSTRIAL IMPACT, MEDICAL
Prnewswire | March 30, 2023
Elemental Machines, developer of the Intelligent Operations Platform, has entered into a partnership with System-c bioprocess as an authorized reseller of Elemental Machines' hardware solutions, servicing the biopharmaceutical industry in France.
The partnership will allow Elemental Machines to expand its footprint further in European markets. System-c serves as a partner and distributor for benchmark brands that cover a range of bioprocessing products from screening to filtration and beyond.
Under the terms of the agreement, System-c will be an authorized reseller and distributor of Elemental Machines' suite of IoT-enabled sensors, which operators can use to collect and unify data across their biopharmaceutical facilities.
"We are excited to partner with System-c and leverage their expertise in bringing innovative technology solutions to their bio-pharma customers,"said Dan Petkanas, Director of Global Alliances at Elemental Machines. "Together we will be able to deliver much needed tools to support and speed drug discovery."
"We are thrilled to announce our partnership with Elemental Machines," said Yannick Carfantan, CEO of System-c. "Elemental Machines' leading technology platform for optimizing laboratory operations is going to give our company a new boost in our business development, not only for the French market but across Europe. Their measurement, information, alerting and data recovery technology opens new horizons for the control of laboratory equipment and R&D, pilot, and production departments enabling peace of mind for a number of actors in the biotech and biopharma industries. A winner for sure!"
About System-c bioprocess
As a partner and distributor for benchmark brands and continually searching for new innovative technologies, our product range covers all stages of the bioprocesses: from screening to filtration of the final product. Our solutions are always evolving in parallel with the new developments in life sciences, as well as the requirements for quality, safety and process productivity.
Established in 1988 System-c bioprocess has been created to guarantee their customers complete, easy-to-use and customizable solutions to ensure productivity, reliability, and continuous traceability for all bioprocesses. Experts in innovative solutions and technologies for cell culture and fermentation processes, we have been constantly working with researchers, technicians and engineers from the pharmaceutical, biotechnology and agrifood industries for more than 30 years.
About Elemental Machines
Elemental Machines' Intelligent Operations Platform combines the best of IoT technology with purpose-built software solutions to deliver actionable intel to operators in the life sciences and byond. The Platform simplifies, optimizes, and augments operations by connecting the physical and digital infrastructure. Science and technology leaders trust Elemental Machines' ecosystem of hardware and software to deliver actionable insights that accelerate their pace of innovation.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Rocket Pharmaceuticals, Inc | February 09, 2023
On February 7, 2023, Rocket Pharmaceuticals Inc., a renowned late-stage biotech firm advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet needs, announced that FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease, a life-threatening genetic cardiac disease with no disease-altering therapies available. RMAT status was granted based on favorable safety and effectiveness findings from the Phase 1 RP-A501 clinical trial and will provide further added benefits of intensive FDA guidance and accelerated evaluation throughout the program's development.
RMAT designation, the same as Breakthrough Therapy designation, offers the advantages of intensive FDA guidance on efficient drug development, such as the ability for early FDA interactions to discuss intermediate or surrogate endpoints, potential priority review of the Biologics License Application (BLA), possible ways to support accelerated approval and satisfy post-approval requirements, and other possibilities to expedite development and assessment. Rocket's RP-A501 program also has Orphan Drug (US) and Rare Pediatric designations.
The Phase 2 pivotal trial is scheduled to begin in the second quarter of 2023. As disclosed previously, the company plans to conduct a single-arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator.
The Phase 1 program's findings provide one of the most extensive investigational gene therapy datasets for any cardiac disease. In patients with Danon Disease treated in the Phase 1 study, RP-A501 was generally well tolerated, with indications of restored expression of the deficient LAMP2 protein and durable improvement or stability of clinical parameters.
Data show consistent and robust improvements in a variety of parameters, including protein expression, decreased autophagic vacuoles, troponin, brain natriuretic peptide (BNP), left ventricular (LV) mass and thickness, and enhanced NYHA class and Kansas City Cardiomyopathy Questionnaire (KCCQ/Quality of Life) measurements. Notably, these BNP and NYHA class improvements and stability contrast sharply with BNP rise and NYHA class worsening found in a representative sample of pediatric and adolescent natural history patients. Overall, the results indicate improvements and normalization in a variety of quantifiable parameters used by cardiologists in clinical practice to foster risk assessment and treatment decisions.
About Rocket Pharmaceuticals, Inc
Founded in 2015, Rocket Pharmaceuticals is a fully integrated clinical-stage firm developing an integrated and sustainable pipeline of investigational genetic therapeutics that have the potential to address the underlying causes of complex rare childhood diseases. Through its platform-agnostic approach, it can build the optimal therapy for each indication, possibly generating revolutionary options for patients suffering from rare genetic disorders. The company has a team of highly productive experts and is increasingly being recognized as a leading biotechnology innovator.
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