Axovant Releases Positive Interim Updates for Parkinson's and Tay-Sachs Gene Therapy Trials

Biospace.com | March 11, 2019

Axovant has been busy, releasing interim results from two different clinical trials, one for Tay-Sachs, the other for Parkinson’s disease. For Parkinson’s disease, Axovant released positive interim results from the first cohort of its SUNRISE-PD Phase II clinical trial of AXO-Lenti-PD. AXO-Lenti-PD is a gene therapy that delivers three genes, tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase, by way of a single lentiviral vector. These genes code for a group of enzymes required for dopamine synthesis. The cohort involved two patients with advanced Parkinson’s disease. They both received a single administration of the lowest dose of the therapy. It was well-tolerated, and no serious adverse events were observed. At three months after receiving the therapy, improvements of an average of 55 percent from baseline were observed, based on the Unified Parkinson’s Disease Rating Scale (UPDRS) Total OFF score. “Our focus in this first cohort of the SUNRISE-PD study was on the safety and tolerability of AXO-Lenti-PD, as well as the evaluation of efficacy using well-validated, objective measures,” stated Gavin Corcoran, Axovant’s executive vice president of Research and Development. “These early data support the safety of the lowest dose of AXO-Lenti-PD, similar to what was observed with the earlier generation construct, ProSavin, and also suggest substantially greater biological activity than the highest dose of ProSavin previously tested. These findings are highly encouraging, and we look forward to advancing to higher dose cohorts where we will explore the full clinical potential of AXO-Lenti-PD in patients with Parkinson’s disease.”

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Introducing the Anbio ADL i1910: An Elegant CLIA Analyzer for Comprehensive Clinical Testing

PRNewswire | July 12, 2023

Anbio, a leading provider of innovative diagnostic solutions, proudly presents the Anbio ADL i1910, a fully automated, compact, and affordable CLIA analyzer designed to transform the landscape of clinical diagnostics. With its cutting-edge technology and versatile capabilities, the ADL i1910 offers a comprehensive range of qualitative and quantitative testing options for analytes in human serum, plasma, whole blood, and urine samples. The ADL i1910 utilizes a direct chemiluminescence method based on acridinium ester, ensuring accurate and reliable results across a wide array of applications. Whether it's hormone detection, myocardial disease screening, infectious disease diagnostics, or tumor-related antigen analysis, the ADL i1910 delivers exceptional performance and meets the diverse needs of modern healthcare professionals. Key Features of the Anbio ADL i1910 Extensive Assay Menu: The ADL i1910 boasts an impressive range of 49 developed assays, allowing for both qualitative and quantitative testing of analytes. This comprehensive immunodiagnostic solution enables healthcare providers to access critical information efficiently. High Throughput: With a remarkable throughput of up to 120 tests per hour, the ADL i1910 significantly enhances laboratory productivity. Rapid turnaround times enable healthcare providers to make timely and informed decisions, ultimately improving patient care. Compact Footprint: Anbio understands the importance of laboratory space. The ADL i1910 has been meticulously designed to occupy minimal space, making it suitable for laboratories of all sizes. Its small footprint ensures that valuable space can be optimized for other essential equipment. User-Friendly Interface: The ADL i1910 features an intuitive graphic user interface with a large color touch screen, simplifying operation and minimizing the learning curve. The user-friendly design ensures ease of use for both experienced professionals and new users, enhancing overall efficiency. "Anbio is proud to introduce the ADL i1910, a breakthrough CLIA analyzer that revolutionizes clinical diagnostics," said Jack Davis, Chief Business Officer at Anbio. "With its advanced technology, extensive assay menu, and impressive throughput, the ADL i1910 provides healthcare professionals with a reliable and efficient solution for a wide range of diagnostic applications." The Anbio ADL i1910 is set to transform the field of clinical diagnostics, empowering healthcare providers with a cost-effective and comprehensive solution. About Anbio Biotechnology Anbio Biotechnology is devoted to making contributions for human health and has never stopped our goal to innovate in life sciences. Driven by continuous technical development and integration, resulting from close cooperation with prestigious institutes across the world. Anbio always strives to provide total solutions in clinical diagnosis field, by offering extensive diagnostic products including laboratory and point-of-care products.

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Cabaletta Bio and WuXi Advanced Therapies Announce Expansion of GMP Manufacturing Agreement to Include CABA-201

Globenewswire | August 23, 2023

Cabaletta Bio, Inc. a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that it has entered into certain work orders relating to Good Manufacturing Practice (GMP) manufacturing under its existing master services agreement with WuXi Advanced Therapies (WuXi ATU), a global Contract Testing, Development and Manufacturing Organization (CTDMO). As part of the agreement, WuXi ATU will serve as a cell processing manufacturing partner for the planned global clinical development of CABA-201 in multiple indications, including potential late-stage clinical trials and commercial readiness activities for CABA-201. “We have had a successful collaboration with WuXi ATU over the past two years for the GMP compliant production of novel cell therapies. Based on this initial collaboration, we chose to expand our partnership to include WuXi ATU as a manufacturer for our CABA-201 clinical programs,” said Gwendolyn Binder, Ph.D., President of Science and Technology of Cabaletta. “WuXi ATU’s dedicated production capacity for CABA-201 supports our planned global expansion and commercial preparedness efforts and will enable us to dose patients in multiple clinical trials with separate parallel cohorts, while maintaining a capital-efficient manufacturing strategy.” Under the terms of the agreement, WuXi ATU will provide GMP manufacturing of CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, to support any of Cabaletta’s planned clinical trials, including the previously announced separate Phase 1/2 clinical trials of CABA-201 for the treatment of patients with systemic lupus erythematosus and idiopathic inflammatory myopathies, or myositis. In addition, WuXi ATU will continue to serve as the Company’s cell processing manufacturing partner for the MusCAARTes™ Phase 1 clinical trial of MuSK-CAART. “We are delighted to expand our partnership with Cabaletta to advance the development of CABA-201 for patients with autoimmune diseases,” said David Y. H. Chang, Ph.D., President and Chief Technology Officer of WuXi ATU. “We look forward to applying our expertise in cell and gene therapy manufacturing to better support our customers to bring potentially life-saving treatments faster to patients in need.” About Cabaletta Bio Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA™ platform encompasses two strategies: the CARTA (chimeric antigen receptor T cells for autoimmunity) strategy, with CABA-201, a 4-1BB-containing fully human CD19-CAR T, as the lead product candidate being evaluated in systemic lupus erythematosus and myositis, and the CAART (chimeric autoantibody receptor T cells) strategy, with multiple clinical-stage candidates, including DSG3-CAART for mucosal pemphigus vulgaris and MuSK-CAART for MuSK myasthenia gravis. The expanding CABA™ platform is designed to develop potentially curative therapies that offer deep and durable responses for patients with a broad range of autoimmune diseases. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA. About WuXi Advanced Therapies (WuXi ATU) As the advanced therapies business unit of WuXi AppTec, WuXi Advanced Therapies is a Contract Testing, Development and Manufacturing Organization (CTDMO) that offers integrated platforms to transform the discovery, development, testing, manufacturing, and commercialization of cell and gene therapies. Our services and solutions accelerate time to market and support customer programs around the world.

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Cell and Gene Therapy, Medical

Synthego and bit.bio Expand Strategic Partnership to Build a Platform for the Development of Safer and More Efficient Genetic Engineering of Human

prnewswire | August 25, 2023

Synthego, a leading provider of industrialized genome editing solutions that accelerate discovery to clinic journey, and bit.bio, the company coding human cells for novel cures, today announced a strategic partnership to build a platform centered on the implementation of synthetic circuitry in cells for therapeutic application. bit.bio is building a pipeline of cell therapies focused on areas of unmet clinical need. The partnership will apply Synthego's expertise in gene editing to bit.bio's opti-oxTM precision cell programming technology, with the aim of discovering and developing advanced genetic engineering solutions for cell therapies. Synthego will rapidly design, build, test and deliver an end-to-end optimized multiplexed genetic editing strategy. The implementation of the multiplexed opti-ox engineering approach will reduce the number of engineering steps required, with the potential of shortening clinical cell engineering and development timelines by up to 10 months. "bit.bio is the leading synthetic biology company focussed on human cells. We are thrilled to deepen our partnership with bit.bio to facilitate a leap forward in the development of synthetic biology-based cell therapies," said Paul Dabrowski, CEO at Synthego. "By coordinating our respective technologies, expertise and resources, we will unlock new opportunities for addressing critical challenges in the field by advancing the R&D cycle of innovative cell therapies." "Synthego are experts when it comes to industrialized cell engineering," stated Mark Kotter, CEO at bit.bio. "Teaming up with Synthego will boost our genetic engineering capabilities. We expect efficiency gains across bit.bio's platform which will allow us and our partners to deliver transformative therapies more rapidly to patients in need." About Synthego 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. About bit.bio bit.bio is a synthetic biology company focused on human cells that is advancing medicine (UN SDG9) and enabling curative treatments (UN SDG3). The company does this by industrializing the manufacture of human cells and making them more accessible. The company was spun out of the University of Cambridge in 2016 and has since raised approximately $200m from investors such as Arch Ventures, Foresite Capital, Milky Way, Charles River Laboratories, National Resilience, Tencent, Verition Fund and Puhua Capital. bit.bio's opti-ox™ precision cell programming and manufacturing technology enables conversion of induced pluripotent stem cells (iPSCs) into any desired human cell type in a single step. This can be achieved within days and at industrial scale, while maintaining exceptional purity and unparalleled consistency. Our discovery platform extends this approach to any desired cell type by identifying the transcription factor combinations that define cell states (including identity, cell subtype identity, maturity) using high throughput screens and advanced data analysis. We believe that opti-ox can revolutionize regenerative medicine similarly to how CRISPR is unlocking gene therapy. bit.bio's cell therapy pipeline is focused on serious diseases that currently lack effective treatments. Our preclinical research areas include liver, immunology and metabolic disease, and we have a collaboration with BlueRock Therapeutics (Bayer AG) to create therapeutics for autoimmune and inflammatory disorders. In addition, our extensive ioCells™ research cell product portfolio, which includes wild type and disease model cells, is opening up new possibilities for studying human biology and developing new medicines in both research and high throughput and high content drug discovery.

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