Alchemab Therapeutics Ltd. has entered into a licensing agreement with Eli Lilly and Co. for ATLX-1282, Alchemab’s first-in-class IND-ready program targeting a novel receptor and mechanism for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.
Abnormal aggregation of α-synuclein is thought to contribute to the pathology of Parkinson’s disease, the second most frequent neurodegenerative disorder. Therefore, developing drugs that prevent such aggregation could be effective for slowing or even preventing the disease.
Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.
Multiple sclerosis (MS) is characterized by persistent inflammation, primarily driven by CNS-resident immune cells like microglia, particularly in the progressive stages. The purinergic P2X7 receptor (P2X7R) is widely expressed on immune cells and is elevated on reactive astrocytes and microglia of MS lesions.
Following a stroke, multiple immune cell types contribute to the neuroinflammatory response, with T cells being among the most consistently associated with both the initial and secondary phases of brain injury.
Blackfinbio Ltd. has obtained IND clearance from the FDA for its novel AAV gene therapy, BFB-101, for hereditary spastic paraplegia type 47 (SPG47), which is caused by changes in the AP4B1 gene. A phase I/II trial will be conducted in the U.S. at Boston Children’s Hospital.