article thumbnail

Unfolding The Folds Of Transthyretin

Delveinsight

Transthyretin (TTR) is a protein that the liver produces to facilitate the transfer of thyroid hormone and vitamin A in the blood. They help in preventing confrontational changes and dissociation of protein, and not halt the production of the protein. Gene-silencing Therapies. Liver Transplantation.

article thumbnail

2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

Biogen and Denali will co-market the lead LRRK2 product in the US and China, and Biogen will commercialise in all other markets. NT-814 is a non-hormonal drug that works on the neurokinin (NK) 1,3 receptor antagonist, thus treating the vasomotor symptoms associated with the menopause. Rare disease progress.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran

pharmaphorum

The passport means that LentiGlobin will be reviewed by the Medicines and Healthcare products Regulatory Agency (MHRA) via the new innovative licensing and access pathway (ILAP) introduced in 2020, which reduces the review time for a new medicine to 150 days.

article thumbnail

Alnylam finds belly fat gene and drug target in UK Biobank study

pharmaphorum

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease.