UNC13A

PASADENA, Calif., Sept. 3, 2024 /PRNewswire/ -- AcuraStem, a patient-based biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, announced today that it has successfully secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM). Continue Reading This latest funding will specifically support AcuraStem's continued efforts to advance its targeted therapeutic program for the UNC13A gene, using its iNeuroRx® technology platform. iNeuroRx® has been instrumental in the company's development of targeted therapeutics focused on the UNC13A gene, which plays a crucial role in the pathophysiology of ALS and FTD. "This funding will enable us to leverage our advances to help revolutionize the treatment landscape for ALS and FTD." Post this An overwhelming majority of ALS cases, and roughly half of FTD cases, are characterized by TDP-43 pathology. TDP-43, an RNA-binding protein typically found in the nucleus, is mislocalized to the cytoplasm in affected neurons, leading to RNA processing errors. These errors result in the incorporation of a cryptic exon into the UNC13A messenger RNA, causing a loss of UNC13A protein. This loss is further exacerbated by a risk variant in the UNC13A gene, closely linked to the TDP-43 binding site and associated with reduced survival in ALS and FTD patients. AcuraStem's iNeuroRx® platform has successfully replicated UNC13A pathology in patient neurons, enabling the rapid development and testing of antisense oligonucleotides (ASOs). These ASOs effectively suppress the cryptic exon and restore normal UNC13A function, offering a promising therapeutic approach for TDP-43 proteinopathies and a deeper understanding of UNC13A's role in disease progression. "The support from CIRM is a testament to the potential of our research and its capacity to address critical challenges in neurodegenerative disease treatment," said Sam Alworth, MS, MBA, co-founder and CEO of AcuraStem. "This funding will facilitate the development of our UNC13A program toward clinical trials, enabling us to leverage our advances to potentially revolutionize the treatment landscape for ALS and FTD." Funding from CIRM was awarded following a highly competitive and rigorous peer-review process, reflecting the strength of AcuraStem's research and potential impact on the treatment of ALS and FTD. For additional information, please visit acurastem.com and follow on LinkedIn, Facebook, and X. About AcuraStem AcuraStem is a patient-based biotechnology company pioneering treatments for neurodegenerative diseases including sporadic ALS and FTD. AcuraStem's best-in-class disease modeling platform, iNeuroRx®, enables the discovery of innovative, effective, and broadly acting treatments. The team's strong expertise in ASO technology provides for the rapid advancement of treatments to the clinic. AcuraStem's research is funded in part by support from the Alzheimer's Drug Discovery Foundation, Harrington Discovery Institute, Alzheimer's Association, Rainwater Charitable Foundation, Department of Defense and the National Institute of Neurological Disorders and Stroke. About the California Institute for Regenerative Medicine (CIRM) At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality. Contacts: Kissy Black Director of Communications, AcuraStem [email protected] 615.310.1894 Roxan Olivas Lotos Nile [email protected] 520-954-1634 SOURCE AcuraStem - Patient-Based Therapeutics

HONG KONG, June 12, 2024 /PRNewswire/ -- Even with the ever-changing situation, technological innovation is still the most critical component for biopharmaceutical companies' long-term development. This continuous innovation keeps companies up to date and promotes the evolution of R&D and the success of commercialization. Recently, Viva Biotech's portfolio companies have new updates. Keep reading for more details. VivaVision Forges Strategic Alliance with Renhe Shanliang, and Intitated Their Domestic Phase III Trial for VVN001 On June 6th, World Eye Health Day, a themed event was held in Shanghai. At this grand occasion, guests witnessed the signing of a joint venture agreement between VivaVision, invested and incubated by Viva BioInnovator (VBI), and Jiangxi Shanliang Pharmaceutical Co., Ltd. The two parties will join forces to build a renowned Chinese brand for contact lens care products. Back on March 16th, VivaVision had announced that the initiation meeting for the domestic Phase III clinical trial of VVN001 eye drops - their independently developed dry eye treatment, a LFA-1 antagonist - was successfully convened in Shanghai, marking the official setting sail of the Phase III clinical trial. QurAlis Grants Lilly Exclusive Global License for QRL-204 CAMBRIDGE, Mass., June 3rd, 2024–QurAlis Corporation ("QurAlis"), invested and incubated by Viva BioInnovator (VBI), recently announced that it has entered into an exclusive license agreement with Eli Lilly and Company ("Lilly") in which QurAlis is granting Lilly global rights to develop and commercialize QRL-204, a potentially best-in-class splice-switching antisense oligonucleotide (ASO) designed to restore UNC13A function in amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases. Under the terms of the agreement, QurAlis granted Lilly an exclusive, worldwide license to develop and commercialize QRL-204 and other UNC13A-targeting compounds in exchange for an upfront payment of $45 million to QurAlis, plus an additional equity investment. QurAlis is also eligible for future milestone payments of up to $577 million and tiered royalties on net sales. Arthrosi Announces Multiple Presentations at the European League Against Rheumatism (EULAR) Congress 2024 SAN DIEGO, May 30th, 2024 /PRNewswire/ -- Arthrosi Therapeutics, Inc., invested and incubated by Viva BioInnovator (VBI), is a late-stage biotechnology company developing a potentially best-in-class, highly potent and selective next generation URAT1 inhibitor to reduce serum urate levels, flares and Tophi in patients with gout. Recently they announced the acceptance of two presentations, including one poster tour at the European League Against Rheumatism (EULAR) Congress 2024 meeting, being held in Vienna, Austria on June 12-15. Lucy Therapeutics Secures New Funding to Advance New Alzheimer's and Parkinson's Treatments WALTHAM, Mass.--(BUSINESS WIRE)--Lucy Therapeutics, Inc. (LucyTx), incubated and invested by Viva BioInnovator (VBI), announced it has raised $12.5 million in additional funding led by existing investors Engine Ventures and Safar Partners with new participation from Bill Gates, Parkinson's UK, and the Michael J. Fox Foundation, which provided a $2 million non-dilutive grant. The fresh capital brings LucyTx's total funding to date to more than $36 million. The investments will further the company's research programs for Alzheimer's and Parkinson's diseases and continue its development of a novel drug target for Rett syndrome. Technoderma Medicines Initiates Phase 2 Clinical Trial for TDM-180935, a Novel Drug for Atopic Dermatitis/Eczema, and Officially Establishes Presence at Chengdu Tianfu International Bio-Town On April 29th, 2024, Technoderma Medicines Inc., ("Technoderma Medicines"), a clinical-stage biotech company developing innovative drugs that is invested and incubated by Viva BioInnovator (VBI), officially announced the initiation of its Phase 2 clinical trial (NCT 06363461) for TDM-180935, a topical ointment for atopic dermatitis/eczema, with patients beginning treatment. Previously, on April 20th, Thyrologics officially established its presence at the Chengdu Tianfu International Bio-Town. Multipeptide Screening Integrated with Biochips, Unveiling New Strategies of XLEMENT and Healthytide On March 27th, 2024, two leading companies in the biopharmaceutical industry - Hangzhou Healthytide Biotechnology Co., LTD. (hereinafter referred to as "Healthytide") and XLEMENT, which is invested incubated by Viva BioInnovator (VBI), announced a strategic cooperation agreement to jointly advance technological innovation and breakthroughs in the field of peptide drug development. Healthytide brings its single-molecule peptide diversity library to the table, while XLEMENT contributes its innovative and industry-leading biochip technology platform to support the collaboration. About VivaVision Biotech VivaVision is a clinical stage pharmaceutical company focused on best-in-class and first-in-class therapies for ocular diseases. VivaVision's current pipeline includes VVN001 for treating dry eye syndrome, VVN539 for the treatment for Glaucoma or Ocular Hypertension, and VVN461 for the treatment of non-infectious ocular inflammation of Uveitis. VivaVision is also engaged in discovery and development of novel therapies for the treatment of other anterior and posterior eye diseases. For more info, visit . About QurAlis Corporation QurAlis is trailblazing the path to conquering amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets with next-generation precision medicines. QurAlis' proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a deep pipeline of antisense oligonucleotides and small molecule programs including addressing sub-forms of ALS that account for the majority of ALS patients. For more information, please visit or follow us on Twitter @QurAlisCo. About Arthrosi Arthrosi Therapeutics, Inc. was founded in San Diego, CA, in 2018 with a mission to create a revolutionary treatment option to target uric acid levels and reduce joint damage for people living with gout. With its vast therapeutic and treatment knowledge, Arthrosi has accumulated a comprehensive and robust intellectual property portfolio and impressive Phase 1 and Phase 2b data showing industry-leading efficacy rates and superior safety profiles. About Lucy Therapeutics Lucy Therapeutics is a private biotechnology company in Waltham, MA, founded by Amy Ripka, Ph.D., in 2017, dedicated to unraveling the complex and interrelated biological processes driving mitochondrial dysfunction and addressing them to slow and stop the diseases they cause. Sitting at the center of genetics and metabolism, Lucy Therapeutics is leading the change in how we think about mitochondria and the intersecting pathways that play critical roles in human health and disease, particularly those of the central nervous system, including Parkinson's disease and Rett syndrome. For more information, visit lucytherapeutics.com. About Technoderma Medicines Technoderma Medicines, Inc. is a privately held clinical stage biopharmaceutical company. The Company was originally located in Jiaxing Xiuzhou Biomedicine Guoqian Park, China, and recently relocated to Chengdu Biomed Town, Chengdu, Sichuan Province, China. Its current core programs focus on development of innovative therapies for Androgenetic Alopecia, Atopic Dermatitis, Psoriasis and Lupus Erythematosus. Its "first-in-class" small molecule thyromimetic drug candidate TDM-105795 for Androgenetic Alopecia is currently in Phase 2a clinical testing. Technoderma's novel JAK1/TYK2 inhibitor TDM-180935 for Atopic Dermatitis is now in Phase 1 clinical testing. The pipeline targets dermatologic indications. About XLEMENT As an Innovator in the field of biopharmaceutical R&D and health diagnostics, Liangzhun has independently developed and launched the pioneering next-generation ultra-surface plasmon resonance detection "MetaSPR Biochip Technology". SOURCE Viva Biotech Holdings

Lilly will work with QurAlis to identify and develop additional therapies targeting UNC13A Eli Lilly is splicing another asset onto its neurodegenerative disease pipeline, paying QurAlis $45 million for rights to a preclinical treatment for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The deal gives Lilly global rights to QRL-204, a splice-switching antisense oligonucleotide (ASO) designed to restore UNC13A function. Researchers linked a variant in UNC13A to ALS and FTD years ago, but it took longer to find a causal link between the gene and diseases. A 2022 paper showed how UNC13A variants exacerbate reduced function of TDP-43, the RNA-binding protein implicated in many ALS and FTD cases.  QurAlis designed QRL-204 to modulate UNC13A splicing and restore normal synaptic activities in ALS and FTD. Having shared preclinical evidence that it is on to something in March, the biotech has persuaded Lilly to pay $45 million upfront and up to $577 million in milestones to add the ASO to its pipeline.  “Genetic precision medicines like QRL-204 that target specific causal components of disease pathology hold great promise for delivering meaningful advances against a range of neurodegenerative diseases like ALS and FTD,” Andrew Adams, Ph.D., senior vice president, neurodegeneration research at Lilly, said in a statement. While hustling QRL-204 toward the clinic, Lilly will work with QurAlis to identify and develop additional therapies targeting UNC13A. The partners will use a QurAlis platform designed to create splice-switching ASOs with improved potency and increased therapeutic index. UNC13A is implicated in roughly two-thirds of ALS cases and one-third of FTD cases, numbers that have caught the attention of a small band of biotechs. AcuraStem has an ASO against UNC13A in development for ALS and FTD. Maze Therapeutics also discovered an UNC13A ASO but has handed the rights to a new biotech that is yet to exit stealth, according to its pipeline. While Lilly is a new challenger for the UNC13A niche, it is a familiar presence in the broader ALS and FTD spaces. The drugmaker expanded in ALS through its $135 million takeover of Disarm Therapeutics in 2020 and a smaller partnership with Verge Genomics the following year. Lilly added a FTD prospect to its pipeline when it bought Prevail Therapeutics for $880 million and has a stake in Arkuda Therapeutics.