Functional Concept

Capsida Suspends Gene Therapy Trial After First Patient Death

6 days ago talkbio0Tagged CAP-002, Capsida Biotherapeutics, Clinical Trial Suspension, Encephalopathies, gene therapy, Patient Death, STXBP1 gene, United States Food and Drug Administration

Capsida Biotherapeutics; gene therapy; CAP-002; clinical trial suspension; STXBP1 encephalopathy; patient death; FDA

Avidity Touts Functional Improvements for DMD Therapy, Clearing Way to FDA

7 days ago talkbio0Tagged Approved, Avidity, Biosciences, Blackfoot language, Breakthrough Therapy, Clinical Trials, Functional, Improvements, Muscular Dystrophy, Duchenne, skipping, Submission, United States Food and Drug Administration

Avidity Biosciences; del-zota; DMD; Duchenne muscular dystrophy; FDA approval; functional improvements; exon 44 skipping; Breakthrough Therapy; clinical trial; BLA submission

Daiichi and Merck’s Lung Cancer ADC Achieves 48% Response Rate in Pivotal Trial

1 week ago talkbio0Tagged Accelerated, Antibody-Drug Conjugates, Approved, Breakthrough Therapy, CD276 gene, Co., Daiichi, Deruxtecan, Frequency of Responses, IDeate-Lung01, Identifier, ifinatamab, Overall Survival, Progression-Free Survival, Sankyo, Small cell carcinoma of lung, therapeutic autologous dendritic cells, United States Food and Drug Administration

Daiichi Sankyo; Merck & Co.; ifinatamab deruxtecan; I-DXd; B7-H3; antibody-drug conjugate; ADC; small cell lung cancer; SCLC; IDeate-Lung01; objective response rate; accelerated approval; progression-free survival; overall survival; FDA Breakthrough Therapy Designation

MRM Health Nets $64M and Treeline Gathers $200M: Fierce Biotech Fundraising Tracker 2025

2 weeks ago talkbio0Tagged Biocodex, Biotech Fundraising, Inflammatory Bowel Diseases, LBP, Live Biotherapeutic Product, Microbiome therapeutics, MRM Health, Series B funding, Treeline, Ulcerative Colitis

MRM Health; Series B Funding; Microbiome therapeutics; Ulcerative colitis; Live Biotherapeutic Product (LBP); Biocodex; Treeline; Biotech fundraising; IBD therapy

Sanofi Phase 3 Eczema Win Falls Short of Analyst Expectations

2 weeks ago talkbio0Tagged Amlitelimab, Dermatitis, Atopic, Dupixent, EASI-75, Eczema, Expectations, Market, Reaction, sanofi

Sanofi; amlitelimab; phase 3 trial; eczema; atopic dermatitis; analyst expectations; Dupixent; EASI-75; market reaction

FDA Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER

2 weeks ago talkbio0Tagged Accelerated, Approved, CBER, CDER, gene therapy, Hereditary Diseases, Infrequent, RDEP, regulatory guidance, submission program, United States Food and Drug Administration

CDER; CBER; rare disease; gene therapy; FDA; submission program; accelerated approval; RDEP; regulatory guidance; rare genetic disorders

FDA Announces New Rare Disease Approval Programme

2 weeks ago talkbio0Tagged Accelerated, Approved, Biological Factors, conditional approval pathway, drug review, Endpoints, Infrequent, real-world data, streamlined process, Surrogate, United States Food and Drug Administration

FDA; rare disease; conditional approval pathway; accelerated approval; biologics; drug review; surrogate endpoints; real-world data; streamlined process

Eisai Secures FDA Approval for Leqembi Autoinjector for Maintenance Dosing in Alzheimer’s Patients

2 weeks ago talkbio0Tagged Alzheimer 's disease, Approved, at-home, AutoInjector, Biogen, dosing, Eisai, Lecanemab, Leqembi, Maintenance, Subcutaneous Injections, United States Food and Drug Administration

Eisai; Leqembi; lecanemab; FDA approval; autoinjector; subcutaneous injection; maintenance dosing; Alzheimer’s disease; at-home treatment; Biogen

Teva Launches First Generic GLP-1 Weight-Loss Drug in the US

2 weeks ago talkbio0Tagged Approved, Generic Drugs, Glucagon-Like Peptide 1, liraglutide, Obesity, Saxenda, Teva, United States, United States Food and Drug Administration, Weight Loss

Teva; GLP-1; generic; weight loss; liraglutide; FDA approval; Saxenda; obesity treatment; United States

J&J Halts Phase 2a Rheumatoid Arthritis Program for Imaavy (Nipocalimab) after Lackluster Results

3 weeks ago talkbio0Tagged Autoimmune Diseases, Clinical Research, Clinical trial failure, daisy, Imaavy, Johnson and Johnson, Nipocalimab, Phase 2a Trial, Rheumatoid Arthritis

Johnson & Johnson; Imaavy; nipocalimab; rheumatoid arthritis; Phase 2a trial; DAISY study; clinical trial failure; anti-TNFα therapy; autoimmune diseases