CELL AND GENE THERAPY
Astellas Pharma Inc. and Dyno Therapeutics, Inc. | December 02, 2021
Astellas Pharma Inc. and Dyno Therapeutics, Inc. announced an option and license agreement was signed on November 23 to develop next-generation adeno-associated virus vectors for gene therapy directed to skeletal and cardiac muscle using Dyno's CapsidMap™ platform.
Dyno's CapsidMap platform represents a transformative approach applying in vivo experimental data and machine learning to create novel AAV capsids – the cell-targeting protein shells of viral vectors – designed to optimize tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability. Unlike traditional approaches, CapsidMap is uniquely well-suited to simultaneously optimize capsids for delivery across multiple organs, with the goal of enabling more effective whole-body treatment for many diseases.
With the establishment of the Astellas Gene Therapies Center of Excellence following the 2020 acquisition of Audentes Therapeutics Inc., Astellas is a leader in genetic medicines, working alongside its world-renowned partners to build a portfolio of potentially life-changing gene therapies. This research collaboration combines Dyno's AI-powered AAV vector engineering capabilities with Astellas Gene Therapies global leadership in AAV-based pipeline assets.
"Through our efforts in gene therapy and the Astellas Gene Therapies Center of Excellence, Astellas strives to identify, develop and deliver transformative gene-based therapies for patients with genetic diseases who currently have few or no effective treatment options. Our principal focus is on developing adeno-associated virus delivered therapies for the treatment of well-defined serious diseases. We are dedicated to delivering novel approaches and utilizing new technologies that can deliver transformational value for patients."
Naoki Okamura, Chief Strategy Officer and Chief Financial Officer, Chief Business Officer at Astellas
"We are so happy to be partnering with Astellas, a world leader in developing gene therapies. Dyno and Astellas each bring unique strengths to this collaboration, together enabling more rapid creation of new therapies for patients with great unmet need," said Dyno's CEO and co-founder Eric D. Kelsic, Ph.D. "This partnership demonstrates the flexibility of Dyno's platform to precisely design the delivery properties of gene therapy vectors towards multi-organ and disease-specific profiles, applying the scientific insights we are rapidly learning across all our partnered and internal vector engineering programs using CapsidMap."
Under the terms of the agreement, Dyno will design novel AAV capsids with improved functional properties for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities, including manufacturing, of gene therapy product candidates using the novel capsids. Dyno will receive an $18 million upfront payment and be eligible to receive additional payments during the research phase of the collaboration as well as clinical and sales milestone payments and royalties for any resulting products. The aggregate potential value of future milestone and royalty payments to Dyno exceeds $235 million per product and over $1.6 billion in total value.
Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients.
About Astellas Gene Therapies
Astellas integrated its wholly owned subsidiary, Audentes Therapeutics, Inc. as of April 1, 2021 and established "Astellas Gene Therapies" within the organization as an Astellas Center of Excellence to develop genetic medicines with the potential to deliver transformative value for patients. Based on an innovative scientific approach and industry leading internal manufacturing capability and expertise, we are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and hope to also advance additional Astellas gene therapy programs toward clinical investigation. We are based in San Francisco, with manufacturing and laboratory facilities in South San Francisco and Sanford, North Carolina.
About Dyno Therapeutics
Dyno Therapeutics is a pioneer in applying artificial intelligence and quantitative in vivo experiments to gene therapy. The company's proprietary CapsidMap™ platform rapidly discovers and systematically optimizes Adeno-Associated Virus capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts.
Flamingo Therapeutics, Inc. | September 09, 2021
Flamingo Therapeutics, Inc., a biotechnology company pioneering RNA-targeting therapies in oncology, today announced that it has entered into an agreement with Ionis Pharmaceuticals to develop RNA-targeted therapies to treat various forms of cancer. This alliance is an expansion of an existing collaboration with Ionis on the FLAME™ discovery engine, following Flamingo's Series A financing in 2020.
The agreement and discovery alliance will leverage Flamingo's deep expertise in oncology and long non-coding RNAs (lncRNAs) in combination with Ionis' expertise in RNA-targeted drug discovery and development to advance candidates against targets considered undruggable with traditional approaches. Flamingo's pipeline will include three clinical-stage programs from Ionis, inhibitors of STAT-3, Androgen Receptor (AR) and IRF4, and one preclinical-stage program that targets MALAT1, a well-characterized lncRNA target. Flamingo will also advance its proprietary discovery engine, FLAME (Flamingo LncRNA Antisense Mining Engine), that addresses lncRNAs, a large and untapped class of disease-causing targets within the dark matter of the human genome.
"We're thrilled to strengthen our relationship with Ionis who has been at the forefront of RNA-targeted therapy for thirty years," said Michael Garrett, Chief Executive Officer of Flamingo. "We believe the combination of Ionis' leadership in RNA and our cutting-edge lncRNA discovery engine will expand the opportunities for applying RNA-targeted approaches to oncology."
"We are pleased that Flamingo is leveraging their core strengths in developing novel cancer medicines to advance innovative Ionis-discovered oncology therapies where there is an unmet need," said Brett P. Monia, Ph.D., Ionis' chief executive officer.
In connection with the alliance, Rob MacLeod, Ph.D., VP, Oncology Research & Development of Ionis, will serve as Chief Scientific Officer of Flamingo. Under the terms of the agreement, Ionis is eligible to receive milestone payments and royalties on future product sales of the STAT3, AR, IRF4 and MALAT1 programs. Flamingo retains full rights to its FLAME platform and all lncRNA programs outside of MALAT1.
Flamingo Therapeutics was founded by VIB, Ghent University, KU Leuven, the University of Michigan, Kurma Partners and PMV, based on pioneering work led by Professor Jean-Christophe Marine (VIB-KU Leuven), Professor Pieter Mestdagh (Ghent University) and Professor Arul Chinnaiyan (University of Michigan). These scientific teams broke new ground on non-coding RNA genes in cancer and were among the first to show a role for lncRNAs as disease drivers in oncology.
About Flamingo Therapeutics
Flamingo is pioneering RNA-targeted therapies for oncology with state-of-the art chemistries. Flamingo has the most advanced antisense RNA-targeting oncology portfolio with three clinical programs targeting undruggable transcription factors and splice variants. To support and expand its pipeline, Flamingo has a proprietary discovery engine, FLAME™ (Flamingo LncRNA Antisense Mining Engine), that addresses lncRNAs, a large and untapped class of disease-causing targets within the "dark matter" of the human genome. The Company was founded in 2020 based on pioneering work in the field of lncRNAs in oncology with our academic partners at VIB, KU Leuven, Ghent University and the University of Michigan. Flamingo has a discovery alliance with Ionis Pharmaceuticals and is supported by well-known biotechnology investors Kurma Partners and PMV. The company is headquartered in Belgium with additional operations in San Diego, CA.
Gator Bio | September 15, 2021
Gator Bio, Inc. announced today the launch of the GatorPlus, a next generation biolayer interferometry (BLI) instrument and two new biosensor products, Gator™ Flex SA Kit and Gator™ AAVX probe. The GatorPlus adds to the currently available GatorPrime instrument; and the new Gator™ Flex SA Kit and Gator™ AAVX probe expand the off the shelf biosensor portfolio to 14 products for the North America market. Gator Bio also offers on demand custom biosensors for specific application needs. GatorPlus is a bench-top instrument for real-time label-free analysis of molecular interactions and quantitation in 96-well or 384-well microplates. The instrument offers longer walk-away time and more automation compared to GatorPrime, the first Gator instrument launched in March 2019.
Based on Gator Bio's proprietary technology, the Gator™ Flex SA Kit is the industry's first ever reactivable streptavidin biosensor for BLI. It can be reactivated and reused more than 20 times without performance degradation, enabling significant cost saving for Gator system customers. The Gator™ AAVX probe quantitates many serotypes of AAVs automatically in minutes, greatly simplifying AAV developers' workflow. The results obtained from the Gator™ AAVX probes correlate tightly to the gold standard method.
Gator Bio was founded by Dr. Hong Tan and Mr. Robert Zuk, the pioneers of biolayer interferometry. The company has been focused on development and commercialization of the next generation BLI that greatly enhances the performance and cost effectiveness of this simple yet powerful analytical technology. In addition to the newly introduced Gator™ Flex SA Kit and Gator™ AAVX probes, Gator Bio offers a wide range of products, including the highly sensitive SMAP biosensor and the regenerable mouse Fc capture biosensor. The Gator systems and associated biosensors provide convenient kinetic analysis and concentration determination for the development of antibody and protein therapeutics.
"We saw problems and shortcomings with the original BLI technology, so we decided to come back to close the gaps with new ways to realize BLI. Our objective is to enable the most effective and efficient BLI systems for scientists".
- Dr. Hong Tan, CEO of Gator Bio.
About Gator Bio,Inc.
Gator Bio is a life sciences company providing bioanalytical systems to accelerate the development of therapeutics and diagnostics. Gator Bio along with its sister company ET Healthcare are part of Access Medical Systems. The Gator instruments and biosensors enable real-time analysis of biomolecular interactions providing information on affinity, kinetics, concentration and epitope binning, etc. Most importantly, Gator Bio's analytical capabilities enable better and faster characterization of drug candidates, thus providing greater value in drug development applications where existing methods have limitations in throughput, performance, and cost. The company is headquartered in Palo Alto, California with facilities in Shanghai and Suzhou, China.