Disease or Syndrome – Page 3

Intellia Pauses Two CRISPR Trials After Severe Liver Toxicity Hospitalizes Patient

1 week ago talkbio0Tagged Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, Hospitalization, Intellia, Liver, Pauses, Phase 3 Clinical Trials, Safety, Senile cardiac amyloidosis, Toxicity aspects

Intellia Therapeutics; CRISPR; transthyretin amyloidosis; Phase III trials; gene editing; liver toxicity; patient hospitalization; safety pause

BridgeBio Shifts Filing Plans After Phase 3 Success in Rare Disease Drug BBP-418

1 week ago talkbio0Tagged Acoramidis, Approved, BBP-418, Biological Markers, BridgeBio, commercial, disease drug, File (record), Infrequent, Limb-girdle muscular dystrophy type 2A, NDA, New Drug Application, Phase 3 success, Ribitol, United States Food and Drug Administration

BridgeBio; BBP-418; Phase 3 success; limb-girdle muscular dystrophy type 2I/R9; FDA filing; rare disease drug; NDA (New Drug Application); ribitol; full approval; biomarker; acoramidis; commercial strategy

OpenAI Collaborates with Thermo Fisher and Lundbeck to Enhance Pharma Research

1 week ago talkbio0Tagged DNA Integration, Lundbeck, OpenAI, Pharma, Thermo Fisher

OpenAI; Thermo Fisher; Lundbeck; Pharma; AI Integration

Unlocking Japan’s Potential in Rare Disease Drug Development: Recent Progress and Challenges (2025)

1 week ago talkbio0Tagged Clinical Trials, drug lag, Drugs, Orphan, innovation, Japan, pharmaceutical partnerships, Rare Diseases, regulatory approval

rare disease; Japan; orphan drug; drug lag; innovation; regulatory approval; pharmaceutical partnerships; clinical trials

Novartis CEO on $12B Avidity Deal: ‘Could Have Been Twice as Big’

1 week ago talkbio0Tagged Acquisition, Avidity Biosciences, financial outlook, growth strategy, Merger, Neuromuscular Diseases, Novartis, RNA, SpinCo ', Vas Narasimhan

Novartis; Avidity Biosciences; Vas Narasimhan; merger; acquisition; biotech; RNA therapeutics; $12 billion deal; neuromuscular diseases; SpinCo; financial outlook; growth strategy

Novartis to Acquire RNA Drugmaker Avidity Biosciences in $12 Billion Deal

1 week ago talkbio0Tagged 2026, Acquisition, AOC, Avidity Biosciences, Closed, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Facioscapulohumeral, MYOTONIC DYSTROPHY 1, Neuromuscular Diseases, Novartis, Platform, RNA therapeutics, SpinCo ', xRNA

Novartis; Avidity Biosciences; acquisition; RNA therapeutics; neuromuscular diseases; Duchenne muscular dystrophy; myotonic dystrophy type 1; facioscapulohumeral muscular dystrophy; AOC platform; SpinCo; biotech; xRNA strategy; 2026 closing

MapLight Therapeutics Announces Pricing of $17/share IPO; to Raise Over $250 Million, Begins Trading on Nasdaq

1 week ago talkbio0Tagged Alzheimer 's disease, biotech, CNS disorder, IPO, MapLight Therapeutics, MPLT, Nasdaq, Phase 2 Clinical Trials, Psychotic Disorders, Public Offering, Schizophrenia

MapLight Therapeutics; IPO; Initial Public Offering; Nasdaq; MPLT; Schizophrenia; Alzheimer’s disease psychosis; CNS disorders; Phase 2 clinical trials; Biotech