TAF1

Award from MGH CCXDP advances effort to identify new treatments for X-linked dystonia Parkinsonism (XDP) BOSTON, March 29, 2023 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), a therapeutics company that leverages a machine-learning model of human health to advance drugs for complex diseases, today announced receipt of grant funding from the Collaborative Center for X-linked Dystonia Parkinsonism (CCXDP) at Massachusetts General Hospital (MGH) to enable rapid target discovery for XDP. The MGH award will allow for a significant expansion of research effort that follows on the successful completion of work funded by an earlier grant that supported development of whole-organism models of XDP through TAF1 gene CRISPR editing and drug candidate screening. In the earlier project funded by MGH CCXDP, Unravel successfully demonstrated XDP model creation in under 3 weeks using Xenopus tadpoles engineered with Unravel's proprietary "SquishyWare™" disease model generation process. Additionally, Unravel's BioNAV™ AI prediction platform identified a novel therapeutic mechanism and candidate compound that led to broad rescue of the tadpole XDP phenotype. The current award will advance target discovery and validation to enable new drug development tailored to the needs to XDP patients. "We are pleased with the opportunity to continue this collaboration with CCXDP," said Frederic Vigneault, Ph.D., Unravel Co-Founder and CSO. "The expansion of our collaboration with MGH is a great validation of the power of our scientific platform and its potential to expedite the identification of promising therapeutic compounds to take into the clinic. Within a year, our computational and biology teams have homed in on specific and novel therapeutic mechanisms. Our ambition by the end of this grant is to nominate a candidate that can move quickly to clinical trials." XDP is a severe adult-onset neurodegenerative disease that causes progressive dystonia and Parkinsonian symptoms. Patients experience worsening motor control and a significantly shorter lifespan. The disease is linked to a DNA repeat expansion within an intron in the TAF1 gene that disrupts TAF1 splicing and reduces levels of the full-length transcript. XDP currently lacks an approved treatment. About Unravel Biosciences Unravel Biosciences is a personalized therapeutics company that redefines diseases through RNA network analysis to seamlessly bridge target discovery with clinical efficacy of new drugs. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases impacting the whole body. Unravel's platform discovered RVL001, a proprietary formulation targeting Rett Syndrome that will enter the clinic this year, and RVL002, a new small molecule with applications in CNS and metabolic diseases. . SOURCE Unravel Biosciences, Inc.

Award from MGH CCXDP advances effort to identify new treatments for X-linked dystonia Parkinsonism (XDP) BOSTON, March 29, 2023 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), a therapeutics company that leverages a machine-learning model of human health to advance drugs for complex diseases, today announced receipt of grant funding from the Collaborative Center for X-linked Dystonia Parkinsonism (CCXDP) at Massachusetts General Hospital (MGH) to enable rapid target discovery for XDP. The MGH award will allow for a significant expansion of research effort that follows on the successful completion of work funded by an earlier grant that supported development of whole-organism models of XDP through TAF1 gene CRISPR editing and drug candidate screening. In the earlier project funded by MGH CCXDP, Unravel successfully demonstrated XDP model creation in under 3 weeks using Xenopus tadpoles engineered with Unravel's proprietary "SquishyWare™" disease model generation process. Additionally, Unravel's BioNAV™ AI prediction platform identified a novel therapeutic mechanism and candidate compound that led to broad rescue of the tadpole XDP phenotype. The current award will advance target discovery and validation to enable new drug development tailored to the needs to XDP patients. "We are pleased with the opportunity to continue this collaboration with CCXDP," said Frederic Vigneault, Ph.D., Unravel Co-Founder and CSO. "The expansion of our collaboration with MGH is a great validation of the power of our scientific platform and its potential to expedite the identification of promising therapeutic compounds to take into the clinic. Within a year, our computational and biology teams have homed in on specific and novel therapeutic mechanisms. Our ambition by the end of this grant is to nominate a candidate that can move quickly to clinical trials." XDP is a severe adult-onset neurodegenerative disease that causes progressive dystonia and Parkinsonian symptoms. Patients experience worsening motor control and a significantly shorter lifespan. The disease is linked to a DNA repeat expansion within an intron in the TAF1 gene that disrupts TAF1 splicing and reduces levels of the full-length transcript. XDP currently lacks an approved treatment. About Unravel Biosciences Unravel Biosciences is a personalized therapeutics company that redefines diseases through RNA network analysis to seamlessly bridge target discovery with clinical efficacy of new drugs. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases impacting the whole body. Unravel's platform discovered RVL001, a proprietary formulation targeting Rett Syndrome that will enter the clinic this year, and RVL002, a new small molecule with applications in CNS and metabolic diseases. . View original content to download multimedia: SOURCE Unravel Biosciences, Inc.