Gene or Genome – Page 3

Rethinking Clinical Trial Data in the Era of Decentralization: Key Trends and Challenges (2025)

2 months ago talkbio0Tagged Aortic Valve Insufficiency, Clinical Trials, Data Integration, Data Quality, DCT gene, decentralized, Diversity, Preventive monitoring, regulatory guidance, Remote, risk-based oversight, trials

decentralized clinical trials; DCT; data quality; patient diversity; remote monitoring; AI in trials; data integration; risk-based oversight; clinical trial data; regulatory guidance

Avalyn Pharma Raises $100M to Advance Inhaled Lung Disease Drugs

2 months ago talkbio0Tagged AP01, AP02, aspects of adverse effects, Avalyn, Clinical Trials, drug safety, Holdings, inhaled lung disease, nintedanib, Pharma, pirfenidone, Pulmonary Fibrosis, Series D, SNCG gene, Suvretta Capital Management

Avalyn Pharma; $100 million Series D; inhaled lung disease medications; pulmonary fibrosis; AP01; AP02; pirfenidone; nintedanib; clinical trials; drug safety; side effects; Suvretta Capital Management; SR One; Novo Holdings

Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy

2 months ago talkbio0Tagged AAVS1 gene, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophies, Limb-Girdle, Patient Death, Safety, Study on Hold, United States Food and Drug Administration, vector

Sarepta Therapeutics; limb-girdle muscular dystrophy; SRP-9004; gene therapy; patient death; acute liver failure; FDA clinical hold; AAV vector; Elevidys; safety concerns

Kowa Secures Expanded Rights to Nicox’s Glaucoma Drug NCX 470 in Major Global Deal

2 months ago talkbio0Tagged Clinical Trials, Denali, Glaucoma, Kowa, licensing agreement, milestone payments, naproxen-n-butyl nitrate, Ophthalmology specialty, royalties, TLX2 gene

Kowa; Nicox; NCX 470; glaucoma; exclusive rights; licensing agreement; Denali clinical trial; ophthalmology; royalties; milestone payments

RFK Jr. Removes Top HHS Aides in Major Leadership Shakeup

2 months ago talkbio0Tagged Buckham, chief of, Hannah Anderson, Health, Heather Flick Melanson, Leadership, Policy, reshuffle, RFK Jr., Shakeup, Social Welfare, Staff, TMEM79 gene, Trump loyalists

RFK Jr.; Health and Human Services; leadership reshuffle; chief of staff; Matt Buckham; Heather Flick Melanson; Hannah Anderson; Trump loyalists; policy shakeup

RFK Jr. Fires Top HHS Leadership in Major Department Shakeup

2 months ago talkbio0Tagged Buckham, CHARGE Syndrome, Hannah Anderson, Health Policy, Heather Flick Melanson, Leadership, RFK Jr., Shakeup, Staff, TMEM79 gene, White House liaison

RFK Jr.; HHS; leadership shakeup; chief of staff replaced; Heather Flick Melanson; Hannah Anderson; Matt Buckham; health policy; White House liaison

RFK Jr. Reshuffles Top HHS Leadership, Replacing Chief of Staff and Deputy Chief

2 months ago talkbio0Tagged Buckham, Changing, CHARGE Syndrome, chief of, federal, Hannah Anderson, Health Policy, Heather Flick Melanson, Leadership, RFK Jr., Shakeup, Staff, TMEM79 gene

RFK Jr.; HHS; leadership shakeup; chief of staff; Heather Flick Melanson; Hannah Anderson; Matt Buckham; staff changes; federal health policy

RFK Jr. Fires Top Deputies, Reshuffles Leadership at HHS

2 months ago talkbio0Tagged Buckham, Changing, chief of, Hannah Anderson, Health, Heather Flick Melanson, Leadership, Research Personnel, reshuffle, Robert F. Kennedy Jr., Social Welfare, Staff, TMEM79 gene, Trump loyalists

Robert F. Kennedy Jr.; Health and Human Services; leadership reshuffle; chief of staff; personnel changes; Matt Buckham; Trump loyalists; Heather Flick Melanson; Hannah Anderson