Quickfire megarounds tee Passage Bio up for gene therapy trials

FierceBiotech | September 05, 2019

Passage Bio has raised its second megaround of 2019. Months after breaking cover with a $115.5 million series A, the James Wilson-founded gene therapy startup has reeled in another $110 million to take several assets into the clinic while hustling other programs through preclinical studies. The series B, which comes seven months after the series A, was led by Access Biotechnology, the life science investment arm of Len Blavatnik’s Access Industries. OrbiMed, Frazier Healthcare Partners, Versant Ventures and other top-tier investors that powered Passage Bio to the series A round also returned for the latest fundraising. Passage Bio plans to use the money to move two gene therapies into the clinic in the first half of next year, hewing closely to the strategy it sketched out in February. Those programs target rare pediatric disease GM1 gangliosidosis and the neurodegenerative disorder frontotemporal dementia.

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SoftExpert Excellence Suite (SE Suite) lowers the cost of regulatory compliance and helps companies maximize success, increase productivity, reduce risk and adhere to various regulations.The solution enables pharmaceutical and biotechnology organizations to map, analyze and continually improve the efficiency of operations by integrating in a single platform of different management frameworks, including product development management, regulations, strategy, processes, performance, customers, documents and records, quality, risks and assets.

Spotlight

SoftExpert Excellence Suite (SE Suite) lowers the cost of regulatory compliance and helps companies maximize success, increase productivity, reduce risk and adhere to various regulations.The solution enables pharmaceutical and biotechnology organizations to map, analyze and continually improve the efficiency of operations by integrating in a single platform of different management frameworks, including product development management, regulations, strategy, processes, performance, customers, documents and records, quality, risks and assets.

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RESEARCH

Synthace Unveils First Life Sciences R&D Cloud Addressing Complexity, Speed & Reproducibility for Scientists

Synthace | August 03, 2021

Synthace, a leading life sciences software company, today announced the first life sciences R&D cloud that includes a no-code software platform addressing the largest barriers to innovation that R&D life scientists currently face. With the release of this new platform, Synthace is also the first vendor to enable scientists to automate experimentation and insight sharing in a unified, global R&D environment. Scientists can now focus on asking the most impactful questions and unlock the true potential of biology. To solve humanity’s hardest problems, such as delivering breakthrough therapies or alternative food sources, the life sciences industry is under tremendous pressure to simultaneously overcome biology’s complexity, accelerate speed to scientific insight, and ensure the reproducibility of experiments. Synthace alleviates these challenges by empowering scientists to improve and accelerate decision-making with more statistically powerful, automated experiments that can minimize human error. Furthermore, Synthace’s cloud platform leverages intelligent, dynamic automation to produce the highest quality data sets that are primed for machine learning (ML) and other advanced analyses to lead to better insights. With Synthace, the life sciences can now benefit from a quantum leap in experimentation capabilities, accelerating development timelines that would have previously been impossible. Customers Accelerate R&D with Synthace Ipsen has been using Synthace to automate the design and construction of therapeutic candidates. With Synthace, Ipsen produced approximately 90 constructs five times faster than previous methods, substantially increasing the number of molecules entering the screening cascade. The platform also achieved a 10-fold reduction in costs associated with DNA synthesis. Karen Bunting, Director of Protein Sciences at Ipsen commented, “Synthace sits very well at the beginning of our drug discovery process. It allows us to explore larger drug design space by simplifying planning and production of multiple molecule variants with combinatorial construct assembly. These throughput improvements help us deliver well-tolerated and effective therapeutic solutions more rapidly to our patients.” Microsoft Research also uses Synthace to automatically generate biological data at a volume that allows its ML algorithms to rapidly improve. As part of reporting on advancements in programming biological systems, a member of its Biocomputation Group noted: “Synthace really comes into its own when we’re performing experiments with complex layouts like combinatorial construct assembly and design of experiments. When we’re building 12 constructs at a time, Synthace automates all the planning that would go into setting up such an experiment and allows it to become routine.” Synthace Life Sciences R&D Cloud The platform provides end-to-end management of the experimental lifecycle, from design through execution to data visualization and knowledge transfer. Synthace adheres to FAIR principles to support interoperability with other major lab informatics platforms to ensure streamlined data management for all of its customers. Only the Synthace Life Sciences R&D Cloud delivers: Complete experimental design, planning and automation, requiring no coding expertise. Scientists can define more informative and impactful experiments that would otherwise be impossible to run and easily implement Quality by Design (QbD) and Design of Experiments (DOE). Seamless, cloud-based data capture, processing, and visualization. R&D teams can deliver deeper and faster insights from fully contextualized, machine learning-ready data sets that are automatically generated from the laboratory. Minimal deployment and onboarding. Customers experience rapid time-to-insight through Synthace’s out-of-the-box platform features and pre-validated protocols for common applications such as ELISA and high-throughput purification, helping them shorten R&D cycles and study more candidates per program. About Synthace Synthace is a life sciences software company enabling life science the way it should be done. Delivering a life sciences R&D cloud to scientists who want to innovate faster, the Synthace platform seamlessly automates experimentation and insight sharing so that scientists can focus on asking the most impactful questions to unlock the true potential of biology. Top global pharmaceuticals, high-growth biotech companies, leading CDMOs, and innovators in artificial intelligence all turn to Synthace to discover solutions to humanity’s hardest problems.

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CELL AND GENE THERAPY

Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle

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. About Astellas 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.

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MEDICAL

Absci Opens Door to New Biotechnology Campus

Absci | October 23, 2021

Absci Corporation the drug and target discovery company harnessing deep learning AI and synthetic biology to expand the therapeutic potential of proteins, today announced the opening of its new campus located in Vancouver, Washington, a few miles east of the downtown location that housed the company for the last four years. “Absci has been an incredibly positive force in the Vancouver community since establishing its headquarters here in 2016 I have followed Absci’s progress as it expanded within and has now outgrown that initial Vancouver facility. Absci is not only attracting investment and top talent to Washington, it is continuously innovating, creating exciting new possibilities for the future of medicine. I look forward to cutting the ribbon on Absci’s new campus early next month, and to continuing to support the development of our biotechnology ecosystem here in Washington. Washington Governor Jay Inslee The new Absci facility was concepted and constructed in a close collaboration between the company, Portland design firm Bora Architecture & Interiors, and local contractor Bremik Construction. Covering over 85,000 square feet of laboratory, office, and meeting spaces, it features regionally sourced, renewable, and natural building materials, custom finishes, and a vibrant aesthetic. The scalable laboratory space was designed to maximize flow, increase capacity, and readily reconfigure as needed. In addition to tripling capacity for program execution, the incorporation of in-house tissue culture capabilities, high throughput analytical instrumentation, advanced liquid handling and automation, and a dedicated clean air fermentation facility, in conjunction with a state-of-the-art offsite computational cluster, facilitate the expanded scope of Absci’s research and development activities. Additionally, integrated technologies for high volume data capture across Absci’s workflow generate the input for ongoing training of Absci’s Denovium EngineTM deep learning AI models for drug discovery and cell line design. “It’s inspiring to come to work each day in this exceptional building,” said Sean McClain, founder and CEO of Absci. “We designed the light-filled space around collaboration and the pursuit of scientific excellence, and we are thrilled with the pride our Unlimiters feel inhabiting this new campus. We strive every day to bring new insights to challenging problems, and this collaborative setting really encourages all of us to step beyond the impossible to create new possibilities for patients.” “When I visited Absci’s campus, I was impressed by the quality and productivity that imbues the space,” commented Bharat Dixit of ClearB Therapeutics. “The facility is designed with a modern look, yet with practical aspects in mind -- the futuristic gowning rooms, the bright, open lab spaces with lots of natural light and intuitive flow, as well as state-of-the-art robotic equipment, to name a few. And the friendly town hall full of people sharing ideas seems to be a sign of big success ahead.” About Absci Absci is the drug and target discovery company harnessing deep learning AI and synthetic biology to expand the therapeutic potential of proteins. We built our Integrated Drug Creation™ Platform to identify novel drug targets, discover optimal biotherapeutic candidates, and generate the cell lines to manufacture them in a single efficient process. Biotech and pharma innovators partner with us to create the next generation of protein-based drugs, including Bionic Proteins™ containing nonstandard amino acids, and other novel drug designs that may be impossible to make with other technologies. Our goal is to enable the development of better medicines by Translating Ideas into Drugs™. For more information visit www.absci.com. Availability of Other Information about Absci Investors and others should note that we routinely communicate with investors and the public using our website (www.absci.com) and our investor relations website (investors.absci.com), including without limitation, through the posting of investor presentations, SEC filings, press releases, public conference calls and webcasts on these websites. The information that we post on these websites could be deemed to be material information. As a result, investors, the media, and others interested in Absci are encouraged to review this information on a regular basis. The contents of our website, or any other website that may be accessed from our website, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Absci Forward-Looking Statements Certain statements in this press release that are not historical facts are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including statements containing the words “will,” “may,” "pursues," “anticipates,” “plans,” “believes,” “forecast,” “estimates,” “expects,” and “intends,” or similar expressions. We intend these forward-looking statements, including statements regarding drug development efforts, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act, and we make this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies, and prospects, which are based on the information currently available to us and on assumptions we have made. We can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved, and, furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control, including, without limitation, risks and uncertainties relating to drug development efforts; along with those risks set forth in our Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission on September 7, 2021, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events, or otherwise.

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