RNA binding protein

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

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.

Degron Therapeutics emerged in 2021 from the lab of ShanghaiTech University professor and company co-founder Yong Cang, Ph.D. Takeda has taken a trip all the way to Shanghai to find just the right kind of glue to stick together a $1.2 billion biobucks deal for new candidates in oncology, neuroscience and inflammation. Molecular glue biotech Degron Therapeutics is the target of the deal, which includes an undisclosed upfront payment, equity investment and milestones. Degron will also be eligible for royalties and a cut of sales down the road. The two companies will work on molecular glue degraders for multiple targets in the three disease areas. The collaboration will utilize Degron’s GlueXplorer platform to find the new glues for therapeutics targets selected by Takeda. Degron will guide this early work before Takeda takes over. Degron has already developed an in-house pipeline of molecular glue drugs, including five in oncology. The furthest along is an undisclosed RNA binding protein in preclinical development for multiple cancer indications, according to the company’s pipeline. The rest of the programs remain in lead optimization. Elsewhere, the biotech has discovery programs in inflammation and metabolic disease as well as rare disease. “This collaboration with Degron not only adds an innovative new platform to our drug discovery toolbox, it is also an example of cutting-edge innovation emerging in the exciting China biotech sector,” said Takeda’s Chris Arendt, Ph.D., chief scientific officer and head of research. Degron emerged in 2021 from the lab of ShanghaiTech University professor and company co-founder Yong Cang, Ph.D. The biotech has raised $34.5 million in capital so far. Takeda is the latest pharma to throw down big bucks—at least in the form of massive milestone potential—to secure a crack at molecular glues. Novo Nordisk signed a deal with Neomorph in February worth a potential $1.46 billion to develop molecular glue degraders. And, in September 2023, Genentech plunked down $47 million upfront plus $2 billion in milestones for Orionis Biosciences to use its Allo-Glue platform to discover and optimize molecular glues for targets.