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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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Evaxion Biotech A/S | January 23, 2023
Evaxion Biotech A/S, a leading clinical-stage biotechnology firm focused on the development of AI-driven immunotherapies, recently announced that FDA has granted fast-track designation for the Company's customized cancer therapy, EVX-01, in conjunction with KEYTRUDA®.
In December 2022, Evaxion gained FDA clearance to proceed with its phase 2b clinical test, where patients with metastatic melanoma are given EVX-01 in combination with KEYTRUDA®. In addition, Evaxion obtained fast-track designation for the vaccine candidate on January 17, 2023. The fast track is developed to expedite FDA's assessment of innovative, novel medications with the potential to meet an unfulfilled medical need.
EVX-01, a peptide-based immunotherapy for cancer, is Evaxion's most advanced clinical asset. The program generates a unique medicine for each patient based on gene analysis of their malignancies and matching with their immune system. The Company's proprietary AI platform, PIONEER, helps facilitate this process.
Per Norlén, CEO at Evaxion, commented," We are extremely pleased that our cancer vaccine candidate EVX-01 has received the FDA fast track designation, as it enables a potentially faster approval of the vaccine. This is first and foremost to the benefit of the patients. And it is a great validation of our AI platform, PIONEER, and our drug development candidate."
(Source – Globe Newswire)
The ongoing Phase 2b clinical trial is being done at sites in Europe, the United States, and Australia. It is conducted in partnership with Merck, which supplies its PD-1 inhibitor KEYTRUDA®. The trial was launched in Australia when the first patient was enrolled in September 2022.
About Evaxion Biotech A/S
Evaxion is a clinical-stage AI-immunology™ platform company using AI, engineering expertise and drug development knowledge to identify and develop novel immunotherapies for the treatment of various cancers, bacterial diseases and viral infections. It has utilized artificial intelligence to build a diverse pipeline of candidate immunotherapies that target two of the most critical unmet medical needs in the world, cancer and infectious diseases. Its three proprietary AI platforms include PIONEER, EDEN and RAVEN.
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MEDTECH, INDUSTRIAL IMPACT
Twist Bioscience Corporation | February 13, 2023
Twist Bioscience Corporation, a company that helps customers succeed by providing high-quality synthetic DNA on its silicon platform, recently announced technology early access to the Twist enhanced Whole Genome Sequencing (eWGS) solution for applications related to non-human gnomics at the Advances in Genome Biology and Technology 2023 General Meeting conducted in Hollywood, Florida. eWGS is a unique solution that allows researchers to collect simultaneous low-pass whole genome data as well as in-depth coverage of specific areas in a high-throughput and cost-effective workflow.
The eWGS workflow begins with a modified Twist 96-plex library preparation kit, which prepares 96 samples in a single tube for sequencing. Following that, a single-tube 96-plex enrichment on an aliquot of the library is performed using a customized panel to enrich further targets that require additional coverage. The original and enhanced libraries are then merged into a single sequencer-ready pool with low whole genome coverage (0.5x to 4x) and high target-specific coverage. Researchers can tailor the coverage differential between low-pass and high-pass sites to their own needs. Preparing and enriching 96 samples for sequencing at once in a single pool enables a high-throughput workflow and may save costs by utilizing fewer materials and resources.
Twist Bioscience’s co-founder and CEO Emily M. Leproust commented, “In agricultural genomics, researchers constantly have to make a tradeoff between cost and insight. Deep sequencing across entire genomes is cost prohibitive, forcing researchers to turn to less costly approaches like genotyping by sequencing, microarrays, or low-pass WGS. These methods impose limits on the resolution, accuracy, or flexibility of experiments.” She added, “With our eWGS solution, researchers can now get all the information they need without compromising on cost or data, enabling our customers to push new boundaries in trait selection, breeding and other applications.”
(Source – Business Wire)
About Twist Bioscience Corporation
Founded in 2013, Twist Bioscience is a leading synthetic biology and genomics firm that has created a proprietary DNA synthesis platform to industrialize biological engineering. It assists researchers in the Life Sciences who are transforming the world for the better. Scientists from several sectors, including health, agriculture, data storage and industrial chemicals, employ its oligo pools, synthetic genes, and NGS target enrichment to improve human lives and the planet's sustainability. Its technology eliminates inefficiencies and enables cost-effective, high-throughput, quick, accurate DNA synthesis and sequencing. The company provides both the quantity and quality that researchers need to quickly capitalize on future prospects.
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