The BioWorld Neurological Diseases Index (BNDI) finished 2025 up 6.33%, continuing the gains seen through November (closing up 2.64%), after reaching a low in July when it was down 22.48%. By comparison, the Nasdaq Biotechnology Index (NBI) ended the year up 32.4%, while the Dow Jones Industrial Average (DJIA) rose 12.97%. In the first two months of 2026, the BNDI slipped 1.24%, compared with gains of 5.85% for the NBI and 1.9% for the DJIA.
Glucocerebrosidase (GCase), encoded by the gene GBA1, is a ubiquitous lysosomal enzyme that breaks down lipid substrates, glucosylceramide (GL-1) and glucosylsphingosine (Lyso-GL1), into glucose and ceramide. Loss-of-function mutations in GBA1 reduce GCase activity, resulting in lipid accumulation within lysosomes and subsequent lysosomal dysfunction.
Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma, retinoblastoma and Group 3 medulloblastoma – have been linked to their shared origin during pineal gland development. Scientists at St. Jude Children’s Research Hospital have identified the molecular signatures that drive these tumors from pinealocyte progenitor cells that conserve a common differentiation program, providing a shared therapeutic target for these three cancer types.
Keros Therapeutics Inc. has presented data regarding their activin receptor ligand trap, RKER-065, for the inhibition of the activin/myostatin signaling axis.
Casma Therapeutics Inc. has been awarded approximately $7.6 million in funding across two competitive grant programs from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support Casma’s lead program, CSM-101.
Acurastem Inc. has received a two-year research grant from Target ALS to advance therapeutics targeting SYF2, a recently identified regulator of TDP-43 function. TDP-43 dysfunction is a central biological hallmark of amyotrophic lateral sclerosis (ALS).
Friedreich’s ataxia (FA) is an inherited neurodegenerative disorder caused by GAA repeat expansions in the FXN gene, which produces a mitochondrial protein vital for iron-sulfur cluster assembly and energy metabolism. Researchers at Solid Biosciences Inc. presented preclinical data supporting the first-in-human trial on SGT-212 gene therapy in FA models.
Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease.
Rapport Therapeutics Inc. added $20 million to its cash runway for its lead phase III oral seizure drug, RAP-219, through a potential $328 million license deal signed with Tenacia Biotechnology Co. Ltd.
Medtronic plc has agreed to buy Scientia Vascular Inc. for $550 million, as it makes good on its promise to embark on more strategic acquisitions this year. The acquisition will bolster its neurovascular business as it adds a portfolio of guidewires and catheters, which uses Scientia’s microfabrication technology to simplify complex neurovascular procedures.
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