CAMBRIDGE, Mass.--(BUSINESS WIRE)--BioAxone BioSciences, Inc., a clinical-stage biotechnology company focused on developing innovative drugs to restore neurological function, today announced that Lisa McKerracher, Ph.D., Founder and CEO, will be a panelist on orphan diseases at the 12th Annual Neurotech Investing and Partnering Conference on Thursday, June 1.
“It’s an honor to be invited to speak on the challenges and opportunities of addressing orphan diseases,” said Dr. McKerracher. “BioAxone is now focused on developing BA-1049, a first-in-class ROCK2 inhibitor targeting cerebral cavernous malformation, also known as angioma, a genetic rare disease allowing blood to leak into the brain. We have brought our first drug candidate into clinical trials in partnership with Vertex and look forward to building on that success.”
Dr. McKerracher is a renowned scientist in regenerative medicine and translational neuroscience with extensive experience in preclinical research and clinical development. Dr. McKerracher earned a BSc in Biology and Genetics from McGill University and Ph.D. in Cell Biology from York University.
She invented Cethrin™, since renamed VX-210, a protein therapeutic promoting regeneration of cut axons and remodeling of damaged circuits for acute spinal cord injury (SCI), an orphan indication. VX-210 is currently in Phase 2b/3 clinical trials in partnership with Vertex.
Dr. McKerracher will be speaking at the conference on Thursday, June 1 at 3:45 p.m. PT on targeting cerebral cavernous malformation and orphan diseases. For more information about the panel and the Neurotech Investing and Partnering Conference, please visit http://www.neurotechpartnering.com.
BA-1049 is a first-in-class Rho kinase 2 (ROCK2) inhibitor targeting the protein kinase that causes cerebral cavernous malformation (CCM). BA-1049 normalizes ROCK2 signaling in brain endothelial cells thus restoring the blood-brain barrier function.
About Cerebral Cavernous Malformation (CCM)
Cerebral cavernous malformation (CCM) is a serious genetic disease where patient have a lifetime risk of brain hemorrhage from vascular malformations in the brain. In patients with CCM, endothelial cells form single or multiple cystic brain lesions that leak and may cause seizure, hemorrhagic stroke and neurological deficits. Inherited cases of CCM are caused by loss of function in one of the 3 CCM genes (CCM1, CCM2 and CCM3) and the numbers of lesions rise with age, increasing risk of a hemorrhagic event. Sporadic cases result from mutations in the same genes.
About BioAxone BioSciences
BioAxone BioSciences is a clinical-stage biotechnology company developing innovative drugs to restore neurological function for patients with Spinal Cord Injuries (SCI) and vascular malformations in the central nervous system with unmet medical need. Led by a team of scientists renowned for their work on axon regeneration and neuronal signaling pathways, BioAxone has a pioneering SCI drug currently in a Phase 2b/3 clinical trial with Vertex, and is positioned to move other candidates into clinical trials. For more information, visit www.bioaxonebio.com