ElevateBio LLC

Plus, news about Astellas, Clover, ElevateBio, Monopar, Idorsia and Neurocrine Biosciences: Vera Therapeutics plans $300M offering: The California biotech is selling shares after presenting Phase 2b data for an experimental drug called atacicept over the weekend. The company shared 96-week data for patients with immunoglobulin A nephropathy. — Kyle LaHucik Abeona Therapeutics resubmits rare skin disease therapy: Back in April, the FDA rejected Abeona’s cell therapy, called pz-cel, requesting more information about manufacturing. Abeona is developing pz-cel for recessive dystrophic epidermolysis bullosa, a rare genetic skin disease. — Lei Lei Wu Astellas yanks Izervay’s submission in Europe: The company said it withdrew its marketing authorization application for Izervay, a C5 protein inhibitor for geographic atrophy secondary to age-related macular degeneration. Astellas did not detail feedback from the Committee for Medicinal Products for Human Use, but said it is still “confident” in Izervay’s risk-benefit profile and will continue to engage with regulators in Europe and elsewhere. There are currently no treatments approved for GA secondary to AMD outside of the US, where Izervay was greenlit in August of last year. — Ayisha Sharma Clover releases early RSV vaccine data: The Shanghai-based biotech said its bivalent vaccine candidate, SCB-1019, produced “comparable” levels of RSV neutralizing antibodies to GSK’s Arexvy at 28 days in a Phase 1 test. Based on these data, Clover plans to advance the asset into studies focused on re-vaccination next year. — Ayisha Sharma ElevateBio lays off 4%: The genetic medicines company, which employs about 500 people, is ending its preclinical cellular engineering work, a spokesperson told Endpoints News on Monday. The Waltham, MA-based biotech laid off about 13% of its staff in October 2023. It’s one of the most well-funded private biopharma startups after having raised about $1.25 billion, including its $401 million Series D last year. Partnerships with companies like Novo Nordisk, Moderna, Kyverna and Affini-T are unaffected, the spokesperson confirmed. — Kyle LaHucik Monopar Therapeutics prices $19.2M offering: The Chicago-area biotech sold shares to Janus Henderson, RA Capital and others. A few days ago, Monopar licensed a Wilson disease drug candidate from AstraZeneca’s Alexion. — Kyle LaHucik Idorsia ends work with Neurocrine Biosciences: The companies were developing Idorsia’s T-type calcium channel blocker for patients with a rare form of pediatric epilepsy. The treatment failed a Phase 2 study in 2022, and further analysis led to the decision to stop work. — Jaimy Lee

- Proprietary LNP platform delivered Life Edit gene editing system in murine studies, leading to high in vivo editing with low immunogenicity profile - Non-viral delivery expands the Company’s capabilities, enhancing flexibility in therapeutic design and applications for partners and internal programs WALTHAM, Mass., Oct. 16, 2024 (GLOBE NEWSWIRE) -- ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering the creation of life-transforming genetic medicines, today announced the successful in vivo delivery of Life Edit gene editing systems with its proprietary LNP platform to enable nuclease editing in the liver in murine models. Data on the LNP platform, including additional data demonstrating durable activity in non-human primates (NHPs), will be presented by Life Edit, ElevateBio’s gene editing and R&D technology business, at the 6th Annual Formulation & Delivery US Congress in San Diego, California at 5:15 p.m. PST today. The proprietary LNP delivery system provides a non-viral delivery option designed to optimize the precision and activity of gene editing therapies. In murine models, the platform demonstrated the ability to deliver gene editing systems precisely to the liver while maintaining a low immunogenicity profile. It also has the potential for repeat dosing with minimal immune response for other therapeutic applications. Importantly, the platform demonstrates potential for liver de-targeting, allowing it to bypass the liver and selectively target other tissues, expanding its flexibility to address a range of genetic conditions. “This is an important milestone for Life Edit as we advance our proprietary delivery platforms alongside our base and reverse transcriptase editing capabilities to develop and deliver safe and efficacious gene editing therapeutics for a broad range of genetic diseases,” said Tedd Elich, Chief Scientific Officer at Life Edit. “As part of ElevateBio’s broad collection of cutting-edge technologies spanning cell, RNA, and gene editing, this platform reinforces our mission to offer comprehensive solutions to innovators across the industry that are developing genetic medicines.” ElevateBio’s Life Edit gene editing platform is available to make a broad spectrum of edits – including base editing and reverse transcriptase editing – for the development of ex vivo and in vivo therapies to treat and potentially cure disease. The non-viral LNP platform complements the Company’s viral delivery systems for precise DNA targeting in cells and tissues. The flexibility of viral and non-viral delivery systems allows tailored solutions for a broad spectrum of genetic diseases, from single-gene disorders to complex multi-gene conditions. About ElevateBio:ElevateBio is a technology-driven company built to power transformative genetic medicines today and for many decades to come. The Company commercializes its enabling technologies, manufacturing capabilities, and industry-leading expertise through partnerships to accelerate development across a breadth of therapeutic approaches and modalities. The ElevateBio ecosystem combines multiple R&D technology platforms – including Life Edit, a next-generation, full-spectrum gene editing platform; comprehensive cell engineering technologies; and an expanded viral and non-viral therapeutic delivery platform – with BaseCamp®, its end-to-end genetic medicine cGMP manufacturing and process development business, to accelerate the discovery and development of advanced therapeutics. ElevateBio aims to be the dominant engine inside the world’s greatest scientific advancements harnessing human cells and genes to alter disease. For more, visit www.elevate.bio or follow ElevateBio on LinkedIn or X. About Life Edit Therapeutics Inc.Life Edit, ElevateBio’s next-generation gene editing business, offers one of the world’s largest and most diverse libraries of RNA-guided nucleases (RGNs), base editors, and reverse transcriptase (RT) editors that provide flexible editing and unprecedented access to the genome. The platform allows Life Edit to target any genomic sequence and potentially develop novel human therapeutics for the most challenging genetic diseases by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, Life Edit will continue to strengthen its platform of genome-editing enzymes, provide gene-editing expertise to strategic partners, and form other third-party partnerships to discover and develop new therapies. For more, visit www.lifeeditinc.com or follow Life Edit on LinkedIn or X. Investor contact:Catherine Huchu@elevate.bio Media contact:DJ Websterdwebster@elevate.bio

WALNUT CREEK, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Dark Horse Consulting Group (“DHC”), the leading global consultancy to the cell and gene therapy (“CGT”) industry, today unveils the Initiative for Certification of Manufacturing Capabilities (“ICMC™”), a groundbreaking offering designed to close a critical gap in the CGT CDMO market. ICMC was conceptualized as a way to meet the growing demand for transparency and consistency in the rapidly expanding CGT CDMO landscape. Currently, new service providers are entering a market where their claims of providing compliant and scalable manufacturing services are not proactively verified by the relevant regulatory bodies. As such, the CDMO market is experiencing fragmentation and the perception of a diminished outsourcing value. With an industry-leading track record in supporting both CGT therapeutic developers and manufacturing organizations, DHC is uniquely positioned to deliver credible and unbiased evaluations in order to help drug developers make informed decisions in selecting their manufacturing partner. DHC maintains a global database of CGT CDMOs and in the past 4 years has performed more than 50 CGT CDMO selection projects, more than 40 audits, and over 60 remediation projects. The ICMC program offers the opportunity for CDMOs to opt-in to independent, third-party evaluations across nine capability systems following published criteria, ensuring a fair, objective, and comprehensive certification into the Certified Member Directory. This Directory is publicly available, providing developers with a trusted resource for initiating the selection of credible manufacturing partners. “With ICMC, we are setting a new benchmark for transparency and trust in the CDMO marketplace,” said Anthony Davies, DHC Founder and CEO. “Sponsors often struggle to find credible, unbiased information when choosing a CDMO. ICMC is designed to help by providing a list of vendors with verified capabilities as a starting point for sponsors’ CDMO selection process.” Key benefits of the ICMC program include: Credibility: ICMC Certification builds trust between therapeutic developers and prospective CDMO partnersDifferentiation: ICMC Certification highlights the extent of capabilities that exist within the member CDMOsVisibility: The ICMC Certified Member Directory enables identification of credible manufacturing partners Several early adopter partners—including ElevateBio, AGC Biologics, and Andelyn Biosciences—have already joined the program and the certification process has commenced. Discussions are ongoing with a number of additional prospective members. About Dark Horse Consulting Group Dark Horse Consulting Group, a global company with offices in the U.S., U.K., and Singapore, was founded in 2014 with the purpose of accelerating cell and gene therapies through unmatched consulting expertise. DHC’s collective knowledge spans product and process development, analytical development, device development, facilities, manufacturing & compliance, quality assurance, project & program management, regulatory affairs, nonclinical development, clinical regulatory, quantitative modeling, market expertise, and diligence & business strategy. DHC offers an unmatched understanding of the challenges faced by cell and gene therapy developers and applies best practices from this and other industries to address a diverse range of client needs. A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/c1977410-6ac8-402c-a8a5-0f68549fe5b9