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The NIH and FDA’s newly launched 27-member public–private partnership will spend US$76 million addressing the hurdles of AAV-based gene therapies for ultra-rare diseases.
Biogen and Ionis’s SOD1-antisense oligonucleotide tofersen failed a first phase III trial, raising questions about the next steps for this drug and for future ALS trials.
Cancer, hepatotoxicity, haematological and neurotoxicity concerns cause experts to call for more transparency and better manufacturing standards for AAV-based candidates.
AlphaFold and RoseTTAFold have delivered a revolutionary advance for protein structure predictions, but the implications for drug discovery are more incremental. For now.
Despite a regulatory setback for Provention Bio’s diabetes prevention candidate teplizumab, hopes remain high for T cell-regulating therapies in autoimmune indications including multiple sclerosis, systemic lupus erythematosus and coeliac disease.
As small activating RNAs gear up to enter phase II testing, other noncoding RNAs that upregulate gene expression are also progressing towards the clinic. But it’s early days for a complex field.
Clinical failures of antisense candidates from two companies highlight the challenges for huntingtin-lowering approaches, but a diverse pipeline could yet provide a disease-modifying therapy.
Thirty-five years on from the FDA’s approval of a first monoclonal antibody, these biologics account for nearly a fifth of the agency’s new drug approvals each year.
As researchers work to understand the biology and epidemiology of post-acute COVID-19, a pioneering platform trial is now testing treatments to try to address the long-term complications of infection in previously hospitalized individuals.