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

Therna Biosciences has joined forces with Charles River to advance single-patient RNA medicines for individuals with severe, ultra-rare disorders.

The collaboration initially will focus on developing an mRNA therapy for a type of lung fibrosis and an ASO for Lamb-Shaffer syndrome.

Therna Biosciences, a biotechnology company pioneering programmable RNA therapeutics, today announced a collaboration with Charles River to advance single-patient RNA medicines for individuals with ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

Results show that targeting the underlying cause of Dravet syndrome with zorevunersen may improve outcomes for people with this rare, devastating genetic neurodevelopmental disease- ...

UCL’s Professor Francesco Muntoni has received the 2026 Novo Nordisk Prize for his pioneering research offering hope to children with Duchenne muscular dystrophy.

Antisense oligonucleotides (ASOs), used to treat genetic diseases, can affect how cells repair damage to their DNA. This is ...

Nanocarriers enhance peptide and oligonucleotide therapy by improving stability, bioavailability, and targeted delivery for effective clinical applications.

The oligonucleotides market offers opportunities in therapeutic applications, particularly for untreated diseases using ASOs, siRNA, and aptamers, with leaders like Alnylam driving innovation. Growth ...

Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine and has a lead investigational ...

Advanced analytics and artificial intelligence (AI) will help make mRNA vaccines and therapeutics better by allowing biopharma engineers to fine-tune the enzymes used to manufacture such products.