Infrequent – Talk Bio

AstraZeneca Amyloidosis Drug Fails Phase 3 Trials but Shows Potential in Subgroup

2 days ago talkbio0Tagged Alexion, Amyloidosis, Anselamimab, AstraZeneca, Clinical trial failure, Drug Development, Infrequent, prespecified, Primary amyloidosis, Subgroup A Nepoviruses

AstraZeneca; amyloidosis; anselamimab; Phase 3 trial; clinical trial failure; prespecified subgroup; rare diseases; Alexion; AL amyloidosis; drug development

AstraZeneca’s Alexion Signs $825M AAV Capsid Deal with JCR Pharma

1 week ago talkbio0Tagged AAVS1 gene, Alexion, AstraZeneca, Capsid, gene therapy, genomic medicines, Infrequent, JCR, JUST-AAV, Licensing Deal

AstraZeneca; Alexion; JCR Pharmaceuticals; JUST-AAV; gene therapy; AAV capsid; rare disease; licensing deal; genomic medicines

Regeneron Leads $66M Series B for Actio Biosciences’ Rare Disease Therapeutics

4 weeks ago talkbio0Tagged ABS-0871, ABS-1230, Actio, Biosciences, Charcot-Marie-Tooth Disease, Clinical Trials, Drugs, Orphan, Epilepsy, Infrequent, Inhibitor, KCNT1 gene, Regeneron, Series B financing, TRPV4 protein, human

Regeneron; Actio Biosciences; rare diseases; Series B financing; KCNT1 inhibitor; TRPV4 inhibitor; ABS-1230; ABS-0871; Charcot-Marie-Tooth disease; epilepsy; orphan drug; clinical trials

Beyond Theory: Real-World AI Wins in Life Science R&D – Faster & Smarter

1 month ago talkbio0Tagged Aortic Valve Insufficiency, Biological Science Disciplines, Clinical Trials, Drug Discovery, Efficiency, Financial cost, Infrequent, R&D, trends qualifier

AI in life sciences; R&D efficiency; drug discovery; clinical trials; pharma innovation; 2025 AI trends; cost reduction; rare diseases

Senate Drops Orphan Cures Act from Trump’s Tax Bill, Dealing Setback to Biopharma

1 month ago talkbio0Tagged Beak, Big Beautiful Bill, Biopharma, Drugs, Orphan, healthcare legislation, Infrequent, Ira (eukaryote), Mediation, Orphan Cures Act, Prices, reconciliation, Senate, Transplant Registry Unified Management Program

Orphan Cures Act; orphan drugs; Trump tax bill; biopharma; Senate; IRA price negotiations; rare diseases; Big Beautiful Bill; healthcare legislation; reconciliation bill

Recursion Pharmaceuticals Lays Off 20% of Staff Amid Pipeline Cutbacks

1 month ago talkbio0Tagged Infrequent, layoffs, Neoplasms, pipeline cutbacks, Recursion, restructuring

Recursion Pharmaceuticals; layoffs; pipeline cutbacks; biotech; workforce reduction; restructuring; rare diseases; oncology

EMA Identifies Very Rare Eye Condition as Side Effect of Semaglutide

1 month ago talkbio0Tagged Adverse effects, Blindness, Infrequent, Multiple Acyl Coenzyme A Dehydrogenase Deficiency, NAION, Ozempic, Rybelsus, semaglutide, Wegovy

EMA; semaglutide; rare side effect; NAION; vision loss; Ozempic; Wegovy; Rybelsus

Sarepta’s rAAVrh74 Viral Vector Receives FDA Platform Technology Designation for Gene Therapy

1 month ago talkbio0Tagged gene therapy, Infrequent, Muscular Dystrophies, Limb-Girdle, platform technology designation, SRP-9003, United States Food and Drug Administration, Viral Vector

Sarepta Therapeutics; FDA; platform technology designation; rAAVrh74 viral vector; gene therapy; SRP-9003; limb-girdle muscular dystrophy; rare diseases

Sanofi to Acquire Blueprint Medicines for Up to $9.5 Billion, Expanding Immunology Focus

2 months ago talkbio0Tagged Acquisition, Ayvakit, Biopharma, Blueprint Medicines, elenestinib, Immunology, Infrequent, Mastocytosis, Systemic, pipeline, sanofi

Sanofi; Blueprint Medicines; acquisition; immunology; systemic mastocytosis; Ayvakit; elenestinib; rare diseases; biopharma; pipeline

FDA Rejects Stealth BioTherapeutics’ Rare Disease Drug but Leaves Door Open for Accelerated Approval

2 months ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Accelerated, Approved, Elamipretide, Infrequent, muscle strength endpoint, Phase 2 trial, regulatory delay, Rejection, Stealth BioTherapeutics, United States Food and Drug Administration

FDA rejection; Stealth BioTherapeutics; elamipretide; Barth syndrome; rare disease; accelerated approval; muscle strength endpoint; phase 2 trial; regulatory delay