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.
INDUSTRIAL IMPACT, MEDICAL
Synthego | March 17, 2023
Synthego Corp., a leading genome engineering solutions provider, announced today the launch of its service partner ecosystem, CRISPR Discovery Partners, designed to accelerate drug discovery by providing a seamless research workflow from CRISPR editing to phenotypic data and downstream services. The ecosystem brings together high-value partners including Arctoris, BrainXell, Curia, PhenoVista, and Pluristyx to offer a coordinated solution for CRISPR-driven drug discovery.
The CRISPR Discovery Partner ecosystem creates an integrated workflow starting with Synthego's CRISPR Engineered Cells through functional readouts, providing researchers with a faster, more efficient, and more reliable way to move their discoveries forward. Researchers can access the full range of genome engineering services through Synthego, as well as downstream services including small molecule screening, cell-based assays, high content imaging, iPSC differentiation, and multi-omics through partners. The result is a streamlined drug discovery process that can help accelerate the development of life-saving treatments.
Synthego's CRISPR Discovery Partners include
Arctoris, a tech-enabled drug discovery service company, combines wet lab automation and machine learning with a world-class team of experienced drug discovery researchers to enable the accelerated development of small molecule, biologics, and cell therapy assets.
BrainXell provides differentiation services to generate high-purity, iPSC-derived human neurons, astrocytes, and microglia for research use with a focus on drug discovery. BrainXell also offers fibroblast reprogramming and select downstream assay services.
Curia, a global contract research, development and manufacturing organization (CDMO), offers products and services from discovery through manufacturing to help customers transform R&D results into real-world impact.
PhenoVista, a San Diego-based CRO, specializes in modern, human, cell-based in vitro assay development and analysis. Internationally recognized for developing and implementing both fully bespoke projects and pre-validated Ready-2-Go assay services, this award-winning research partner leverages high-content imaging to enable life scientists to accelerate their discovery of novel therapeutics and improve global health.
Pluristyx offers expertise and tools to support and accelerate the development and commercialization of revolutionary therapeutics to treat serious human diseases by providing unique products, consulting, and contract services, including cell line expansion and banking.
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms and products for science at scale. With its foundations in engineering disciplines, the company's platform technologies vertically integrate proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance basic research, target validation, and clinical trials. With its technologies cited in hundreds of peer-reviewed publications and utilized by thousands of commercial and academic researchers and therapeutic drug developers, Synthego is at the forefront of innovation enabling the next generation of medicines by delivering genome editing at an unprecedented scale.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Azenta Life Sciences | February 28, 2023
On February 27, 2023, Azenta Life Sciences, a leading provider of life sciences solutions, announced the launch of its latest automated cryogenic storage system, the Cryo Store PicoTM. This new product is designed to store high-value biological samples used throughout various stages of the life sciences industry, from research and development to clinical use.
Azenta is already known for providing innovative automated cryogenic solutions such as the BioStore™ III Cryo and the CryoPod™ Carrier, and the Pico is the latest addition to its lineup. The product offers automated cryogenic solutions that are configured for laboratory and clinical procedures.
The Pico offers a variety of features for cell and gene therapy developers and manufacturers, including end-stage deployment in hospitals and clinics. It offers controlled access and traceability to support regulatory compliance with a compact footprint and is also user-friendly for laboratory personnel.
Interested parties can order the Pico now, and a live demonstration will take place at the Society for Laboratory Automation and Screening (SLAS) 2023 International Conference in San Diego, California.
Pico's Product Manager Erica Waller commented, "Pico takes our proven automation capabilities and scales it down for the lab or the clinic, bringing the sample integrity and process rigor of automation closer to the point of use,"
(Source – PR Newswire)
About Azenta Life Sciences
Azenta is a leading life sciences solutions provider that helps to accelerate the development and delivery of impactful therapies to market. The company offers a comprehensive suite of cold-chain sample management solutions and genomic services for areas such as clinical research, drug development and advanced cell therapies. These solutions are trusted by some of the top biotech, pharmaceutical, academic, and healthcare institutions worldwide. Headquartered in Chelmsford, MA, Azenta has a global presence with operations across Europe, North America, and Asia. The company is committed to providing reliable, innovative, and high-quality solutions to meet the needs of its clients in the life sciences industry.