Aegle Therapeutics Receives Rare Pediatric Disease Designation from the FDA for AGLE-102 for Patients with Dystrophic Epidermolysis Bullosa

Aegle Therapeutics Corporation, a first-in-class biotechnology company committed to delivering cell-free therapy to patients affected by severe dermatological conditions, today announced that the FDA has granted Rare Pediatric Disease (RPD) Designation to AGLE-102™ for the treatment of dystrophic epidermolysis bullosa (DEB), a skin blistering disorder.  AGLE-102 is a composite of mesenchymal stem cell-derived extracellular vesicles that deliver proteins, genetic material and regenerative healing factors to diseased and damaged tissue. "The FDA's grant of Rare Pediatric Disease designation following its earlier grant of Fast Track Designation to AGLE-102 for DEB underscores the significant unmet medical need of children and adults living with this debilitating disease. AGLE-102 has the potential to be the first multifaceted approach to treat this rare patient population," said Shelley Hartman, CEO of Aegle Therapeutics. The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children ages 18 years or younger and fewer than 200,000 persons in the U.S. If Aegle's new drug application ("NDA") for DEB is approved, Aegle may receive a priority review voucher from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be sold to another company for their programs.

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Capsida Biotherapeutics Enters Strategic Collaboration With Kate Therapeutics to Manufacture KateTx's Next-Generation Gene Therapies

PR Newswire | October 05, 2023

Capsida Biotherapeutics Inc. and Kate Therapeutics announced a strategic partnership to leverage Capsida's expertise and adeno-associated virus (AAV) manufacturing capabilities to enable KateTx's initial internal portfolio of muscle and heart disease programs. Under the terms of the agreement, Capsida will provide GMP manufacturing of KateTx's gene therapies as the medicines advance through preclinical and clinical development. In return, Capsida will receive undisclosed funding for the term of the agreement. KateTx's publicly disclosed internal programs include myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy, which are two of the leading causes of adult-onset muscular dystrophy. "Capsida possesses end-to-end expertise – which includes AAV capsid engineering, preclinical, clinical, and state-of-the-art manufacturing," said Peter Anastasiou, chief executive officer (CEO) of Capsida Biotherapeutics. "We are thrilled to be able to leverage our established capabilities in AAV manufacturing and support a peer company to reach patients in a complementary space, while continuing to advance our own internal and partnered pipeline in CNS and ophthalmology." "KateTx is leading the way in developing capsid and cargo combinations to address muscle and heart diseases in ways that have not been previously possible," said Kevin Forrest, Ph.D., president and CEO of Kate Therapeutics. "Capsida's exceptional AAV manufacturing capabilities allow us to build our pipeline, de-risk our programs, and advance them into the clinic in the most rapid manner possible, so we can bring much-needed therapies to patients quickly." "This collaboration between Capsida and KateTx leverages complementary expertise and overlapping needs across both innovative companies to advance programs in a capital-efficient manner. Capsida has established first-in-class engineered AAV manufacturing capabilities applicable across broad therapeutic areas, and this partnership with KateTx aims to bring these next-generation gene therapies to patients sooner than with traditional approaches," said Beth Seidenberg, M.D., co-founding managing director at Westlake Village BioPartners, member of the board of Capsida, and chair of the board at KateTx. About Capsida Biotherapeutics Capsida Biotherapeutics is an integrated gene therapy company developing treatments for rare and common diseases across all ages. Capsida's new class of engineered adeno associated virus (AAV)-based delivery technologies enables a broad range of therapeutic modalities, including DNA, RNA, gene editors, vectorized antibodies and other genetic cargoes, to be systemically delivered to specific organ systems while simultaneously limiting exposure to non-targeted organs. Capsida's proprietary capsid engineering and broad IP estate allows for identification and development of capsids with tropism for any tissue or cell type. The company's initial focus has been delivering therapeutic cargoes intravenously to the central nervous system (CNS) with high transduction levels to the desired tissues and cells. The company is developing a pipeline of both wholly owned programs in CNS and partnered programs with AbbVie (CNS and broad range of ophthalmic disorders), Lilly (CNS), and CRISPR Therapeutics (CNS). Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. About Kate Therapeutics Kate Therapeutics (KateTx) is a patient-focused biotechnology company developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined muscle and heart diseases. The company is applying novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation. These breakthroughs have the potential to improve the efficacy and safety of gene therapies and enable the pursuit of a broader set of targets that are otherwise difficult to drug with current technologies.

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Zenfold Leverages Ginkgo Enzyme Services to Enable Veterinary Active Ingredient Development with Sustainable Biology

PR Newswire | October 17, 2023

Ginkgo Bioworks which is building the leading platform for cell programming and biosecurity, and Zenfold Sustainable Technologies, a company focused on developing and manufacturing specialty ingredients using sustainable technologies and precision fermentation, today announced a collaboration to leverage Ginkgo Enzyme Services in its effort to discover an enzyme critical to the manufacturing of veterinary products. This partnership aims to replace traditional chemical processes in veterinary active ingredient production with a sustainable biological method. "This collaboration marks a significant step forward in Zenfold's mission to bring sustainable manufacturing technology to the veterinary medicine industry," said Dr. BSV Prasad, CEO and Managing Director of Zenfold Sustainable Technologies. "By utilizing Ginkgo's expertise and innovation, we are well-positioned to develop a sustainable solution that will have a lasting impact on the Indian market and beyond." "We are excited to partner with Zenfold, which brings a powerful vision of sustainable biotechnology to veterinary active ingredient development. This collaboration will enable us to apply our state-of-the-art enzyme discovery module to enable more innovation in the veterinary medicine space," said Jennifer Wipf, SVP, Head of Commercial, Cell Engineering at Ginkgo. "The project involves creating a broad library of cDNA candidates from a metagenomic collection of billions of enzyme sequences. That's the kind of scale visionary projects like this need, and Ginkgo is committed to scaling up this solution for the betterment of animal health and the environment." About Ginkgo Bioworks Ginkgo Bioworks is the leading horizontal platform for cell programming, providing flexible, end-to-end services that solve challenges for organizations across diverse markets, from food and agriculture to pharmaceuticals to industrial and specialty chemicals. Ginkgo's biosecurity and public health unit, Concentric by Ginkgo, is building global infrastructure for biosecurity to empower governments, communities, and public health leaders to prevent, detect and respond to a wide variety of biological threats. About Zenfold Zenfold Sustainable Technologies is a pioneer in the field of sustainable biology, with a focus on veterinary actives and enzyme supply. This collaboration with Ginkgo Bioworks marks Zenfold's expansion into the global veterinary market and further emphasizes their commitment to environmental responsibility and innovative biological solutions.

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Repligen Announces Agreement to Acquire Fluid Management Innovator Metenova

GlobeNewswire | September 28, 2023

Repligen Corporation a life sciences company focused on bioprocessing technology leadership, announced that it has entered into a definitive agreement to acquire privately-held Metenova AB (Metenova) of Mölndal, Sweden. Metenova is projected to generate revenues of $24 million to $25 million for fiscal year 2023, led by the success of its magnetic mixing and drive train technologies that are widely used by global biopharmaceutical companies and contract development and manufacturing organizations (CDMOs). The company recently entered the single-use mixing market with the launch of its MixOne platform, leveraging the success of its existing stainless steel (repeat-use) product line. Metenova is expected to contribute approximately $5 million in revenue to Repligen in the fourth quarter of 2023, and to contribute $25 million to $27 million in revenue in 2024, with 20%-25% revenue growth in 2025 forward. The acquisition is anticipated to be accretive to Repligen’s adjusted gross and operating margins in 2024, and to be accretive to adjusted earnings per share in 2025. Over the past decade, Metenova has established market leadership through design innovation in magnetic mixing, with a state-of-the-art suite of both stainless steel and single-use impellers (mixer heads) and drive trains – key components of mixing vessels. The company offers a broad range of products, including high power solutions for efficient media and buffer preparation, and low shear solutions for gentle mixing of sensitive proteins. Metenova’s high performance aseptic mixing technologies are designed to minimize product damage and improve product yield and product consistency, contributing to improved ROI for customers. The company’s solutions are applicable to a wide range of small molecule and large molecule therapeutics and vaccines, including monoclonal antibodies, recombinant proteins, and cell and gene therapies. Tony J. Hunt, President and Chief Executive Officer at Repligen said, “The addition of Metenova further strengthens our Fluid Management portfolio, with a product line that expands on the market success of our systems and fluid management assemblies and complements our recently acquired single-use bag business from FlexBiosys. We are excited to welcome the Metenova team to Repligen and we look forward to further developing and integrating their differentiated magnetic mixing technologies into our portfolio.” Johan Westman, Chief Executive Officer at Metenova said, “We have made tremendous progress over the last ten years, building a market leading mixing portfolio. We are now at a stage in our development where we can benefit from more rapid expansion into the single-use market where many of our customers are scaling today. Repligen is the ideal partner for us to take this next step of growth and we look forward to working with our colleagues at Repligen to drive additional global demand for our single-use mixing products, while continuing to advance innovative single-use solutions for the industry.” About Metenova AB Metenova is a leading innovator and manufacturer of magnetic mixers for pharmaceutical and biotechnology use. We provide aseptic cutting-edge mixing technology for critical applications. Sales are mainly performed by distributors in over 30 countries with approximately one-third of sales in North America, one-third of sales in Europe and one-third of sales in Asia/ROW. Metenova recently entered the single-use mixing market with the launch of its MixOne platform, leveraging the success of its existing repeat-use (stainless steel) product line. Metenova is headquartered in Mölndal in the Gothenburg area and has approximately 50 employees. About Repligen Corporation Repligen Corporation is a global life sciences company that develops and commercializes highly innovative bioprocessing technologies and systems that enable efficiencies in the process of manufacturing biological drugs. We are “inspiring advances in bioprocessing” for the customers we serve; primarily biopharmaceutical drug developers and contract development and manufacturing organizations (CDMOs) worldwide. Our focus areas are Filtration and Fluid Management, Chromatography, Process Analytics and Proteins. Our corporate headquarters are in Waltham, Massachusetts, and the majority of our manufacturing sites are in the U.S., with additional key sites in Estonia, France, Germany, Ireland, the Netherlands and Sweden.

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