Treatments that target a fragment of the mutant protein that causes Huntington's disease might be more effective than treatments—now in clinical trials—that target the whole protein but leave this ...

Peptide conjugation techniques are advancing CNS delivery of oligonucleotides, addressing blood-brain barrier challenges and ...

Acurastem received a grant to develop ALS therapies targeting SYF2, a regulator of TDP-43, in collaboration with academic research teams.

In this interview, Professor Anne Willis discusses her research on posttranscriptional control of gene expression and shares insights into the challenges faced by women in STEMM and highlights her ...

Researchers found that targeting a protein fragment, huntingtin 1a, restored baseline gene activity and reduced aggregate ...

Luxna Biotech & Inabata ink license agreement for manufacture and sale of modified nucleic acid GuNA amidite: Osaka, Japan Monday, March 16, 2026, 09:00 Hrs [IST] Luxna Biotech Co ...

Camp4 Therapeutics FY25 GAAP EPS missed expectations, but ended the year with a robust cash position. Check out why CAMP ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

AcuraStem, a patient-based biotechnology company developing disease-modifying therapies for amyotrophic lateral sclerosis (ALS), today announced it has received a two-year research grant from Target ...

The commentary titled 'Establishing a Commercial Solution for Extremely Rare Genetic Diseases' was published in Nature Biotechnology Commentary coincides with draft guidance from the FDA outlining a ...