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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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.
About QIAGEN
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.
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AI, DIAGNOSTICS
Genomic Vision | March 08, 2023
On March 7, 2023, Genomic Vision, a leading biotech company that develops products and services for the analysis and control of genome changes, launched FiberSmart®, an AI-based technology for automating the quantification and detection of fluorescent signals on combed DNA molecules.
Initially available for the analysis of Replication Combing Assays (RCA), Genomic Vision's proprietary method for directly visualizing DNA replication kinetics at the single molecule level. FiberSmart uses advanced AI methods to detect, visualize, and analyze DNA replication kinetics up to 3x more accurately and up to 10x faster than Genomic Vision's existing software solutions.
The technology offers a simple and user-friendly interface, facilitating quick analysis of DNA replication signals to deduce essential parameters describing replication kinetics.
It is compatible with Genomic Vision's FiberVision® and FiberVision-S® scanners for the RCA assay of the company's proprietary DNA combing technology with various potential applications, including in gene and cell therapy quality control.
FiberSmart has been successfully tested and validated by AstraZeneca and the Fritz Lipmann Institute in Germany.
Genomic Vision's CEO Aaron Bensimon said, "The launch of FiberSmart® is an important milestone for Genomic Vision as we bring the benefits of powerful AI technology to our users, who can now perform faster and more accurate genomic analysis seamlessly. Our proprietary DNA combing technique has multiple potential applications, particularly in the cell and gene therapy space, where highly accurate genomic analysis is paramount to ensure robust quality standards are met. With the launch of this software we are making it easier for users to exploit the full potential of our proprietary advanced genomic analysis technique."
(Source – Business Wire)
About Genomic Vision
GENOMIC VISION is a leading biotechnology company specializing in the analysis of genome modifications, with a focus on ensuring their quality and safety in genome editing technologies and biomanufacturing processes. It utilizes advanced nanotechnology for DNA analysis to develop cutting-edge diagnostic and drug discovery solutions for cancer and acute diseases at the intersection of genome dynamics and human diseases. The company's approach employs Molecular Combing Technology, a powerful technique that directly visualizes single DNA molecules to detect and quantify changes in the genome landscape and their contribution to pathology.
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