Disease or Syndrome – Page 13

FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome

1 month ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Accelerated, Approved, Clinical Trials, Elamipretide, Forzinity, Infrequent, Mitochondrial Diseases, Pediatric Disorder, Stealth BioTherapeutics, United States Food and Drug Administration

FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial

Sanofi’s MS Drug Tolebrutinib Faces FDA Delay Amid Extended Review

1 month ago talkbio0Tagged Breakthrough Therapy, Bruton 's tyrosine kinase inhibitor, Deferred, GEMINI trial, HERCULES trial, Multiple Sclerosis, NDA, nrSPMS, PERSEUS trial, regulatory review, sanofi, Tolebrutinib, United States Food and Drug Administration

Sanofi; tolebrutinib; FDA delay; multiple sclerosis; nrSPMS; Bruton’s tyrosine kinase inhibitor; breakthrough therapy; HERCULES trial; GEMINI trial; PERSEUS trial; NDA; regulatory review

ACIP Vote Restricts Combined MMRV Vaccine for Children Under 4, Raising Access Concerns

2 months ago talkbio0Tagged ACIP, CDC, CDC vaccine recommendation, Chickenpox, Child, Childhood, Immunization, measles, MMRV vaccine, Mumps, Rubella, vaccine access, Vaccines

ACIP; MMRV vaccine; measles; mumps; rubella; varicella; childhood immunization; Vaccines for Children (VFC) program; CDC vaccine recommendation; vaccine access

Ono Pharmaceutical and Jorna Therapeutics Announce AI-Driven Research Pact to Develop Novel RNA Editing Medications

2 months ago talkbio0Tagged Artificial Intelligence, Dosage Forms, Japan, Jorna, RNA Editing, SkyEngine

Ono Pharmaceutical; Jorna Therapeutics; RNA editing; AI drug discovery; SkyEngine; biotech partnership; Japan

Novo Nordisk Awaits FDA Decision on Oral Wegovy Pill for Obesity

2 months ago talkbio0Tagged Approved, Cardiovascular Risk, Contraceptives, Oral, Glucagon-Like Peptide 1, Nordisk, Obesity, oral semaglutide, United States Food and Drug Administration, Wegovy, Weight Loss

Novo Nordisk; Wegovy; oral semaglutide; FDA approval; obesity treatment; GLP-1 pill; weight loss; cardiovascular risk

bluebird bio Rebrands as Genetix Biotherapeutics Following Private Equity Buyout

2 months ago talkbio0Tagged Anemia, Sickle Cell, Bluebird Bio, Capital Partners, Carlyle, Cerebral Adrenoleukodystrophy, commercial, David Meek, DKK1 gene, execution, FDA-approved therapies, gene therapy, Genetix Biotherapeutics, Investments, Manufacturing, Private Equity, rebranding, Thalassemia

bluebird bio; Genetix Biotherapeutics; private equity buyout; Carlyle; SK Capital Partners; gene therapy; rebranding; FDA-approved therapies; David Meek; sickle cell disease; β-thalassemia; cerebral adrenoleukodystrophy; commercial execution; manufacturing investment

VectorY Strikes $1.2B Option and License Pact with Shape Therapeutics for Brain-Penetrant Gene Therapy Delivery

2 months ago talkbio0Tagged Alzheimer 's disease, Antibodies, biobucks, brain-penetrant delivery, Capsid, Dependovirus, gene therapy, Huntington Disease, milestone payments, Neurodegenerative Disorders, pipeline, SHP-DB1, vectorized, VectorY

VectorY Therapeutics; Shape Therapeutics; SHP-DB1; adeno-associated virus (AAV) capsid; brain-penetrant delivery; gene therapy; neurodegenerative diseases; Huntington’s disease; Alzheimer’s disease; biobucks; pipeline; milestone payments; vectorized antibodies

Lilly’s Mounjaro Shows Strong Results in Youth Type 2 Diabetes, Paving Way for Expansion

2 months ago talkbio0Tagged Adolescent (age group), Body mass index procedure, Child, Clinical Trials, Diabetes Mellitus, Non-Insulin-Dependent, eli lilly, Hemoglobin A1c measurement, Mounjaro, SURPASS-PEDS

Mounjaro; tirzepatide; type 2 diabetes; children; adolescents; SURPASS-PEDS; A1C reduction; BMI reduction; clinical trial; Eli Lilly

Roche Acquires 89bio with $2.4 Billion Buyout to Enter FGF21 MASH Therapeutics Space

2 months ago talkbio0Tagged Acquisition, cardiometabolic diseases, Conditional, fibroblast growth factor 21, Liver diseases, MASH, metabolic dysfunction-associated steatohepatitis, pegozafermin, Roche (company)

Roche; 89bio; acquisition; FGF21; pegozafermin; MASH; metabolic dysfunction-associated steatohepatitis; liver disease; biotech; contingent value rights; cardiometabolic diseases

A Concerning Pattern: Pharma Mulls UK Divestments as Government Action Lags in Life Sciences

2 months ago talkbio0Tagged AstraZeneca, Biological Science Disciplines, drug price, drug spending, eli lilly, Government, Investments, Merck, Nance-Horan syndrome, pharmaceutical divestment, Policy, R&D, sanofi, UK

UK life sciences; pharmaceutical divestment; Merck; AstraZeneca; Sanofi; Eli Lilly; R&D investment; drug pricing; government policy; NHS drug spending