Articles tagged with “gene-therapy”

Otarmeni FDA Approval: OTOF Gene Therapy for Hearing Loss

Examine the FDA approval of Otarmeni, the first gene therapy for OTOF-related congenital hearing loss, and its expedited National Priority Voucher review.

35 min read

4/25/2026

otarmeni fda approval gene therapy congenital hearing loss otof mutation national priority voucher genetic deafness dual aav vectors

Top Biotech Startups 2026: An Analysis of Emerging Trends

An evidence-based analysis of 20 biotech startups poised for impact by 2026. Learn about key innovations in gene therapy, diagnostics, AI, and sustainability.

40 min read

12/26/2025

biotechnology biotech startups gene therapy ai in drug discovery biotech funding diagnostics synthetic biology sustainable biotech

Rare Diseases in 2025: Diagnosis, Treatment & Policy

Explore the 2025 rare disease landscape, affecting 300 million people globally. Learn about diagnostic challenges, orphan drug development, and future policy di

135 min read

11/15/2025

rare diseases orphan drugs genetic disorders diagnostic odyssey public health drug development gene therapy

Specialty Pharmacy: An Analysis of Key Players & Trends

An in-depth analysis of the specialty pharmacy industry covering key players (PBMs, distributors), market trends, biosimilar competition, PBM reform, and the economics of high-cost specialty drugs. Updated March 2026.

45 min read

11/3/2025

specialty pharmacy specialty drugs pharmacy benefit manager drug spending pbms gene therapy biosimilars

Most Expensive Drugs in the US: A 2025 Price List

See the 2025 list of the most expensive drugs in the USA, led by multi-million dollar gene therapies like Lenmeldy & Elevidys for rare diseases. Learn why price

10 min read

10/8/2025

drug pricing gene therapy orphan drugs rare diseases lenmeldy elevidys pharmacoeconomics biotechnology