FDA pushes forward on gene-editing therapies with new safety roadmap

Genome editing holds extraordinary promise for treating previously incurable genetic diseases

FDA clears Travere’s FILSPARI for expanded use in rare kidney disease

Alexion to showcase broad neurology pipeline at AAN 2026 with 20 presentations

The programme features headline data from pivotal Phase III trials, including PREVAIL in gMG, KOMET in NF1-PN, and CHAMPION-NMOSD, alongside real-world evidence supporting established therapies

Neurocrine Biosciences to acquire Soleno Therapeutics for $2.9 billion

The acquisition adds VYKAT XR (diazoxide choline), the first FDA-approved therapy for hyperphagia in Prader-Willi syndrome (PWS), a rare genetic disorder, to Neurocrine’s portfolio

HRV Pharma, Shodhana join hands to build next-gen API pipeline for global markets

Multi-year CDMO partnership combines AI-led virtual development, precision chemistry and GMP manufacturing to accelerate five high-science APIs across regulated markets

Sanofi appoints Dr Parikshit Chaudhari as Head of Market Access & Pricing Strategy

He brings extensive cross-functional expertise built through leadership roles

Investigational therapy efzimfotase alfa shows promising Phase III results in rare bone disease

Efzimfotase alfa is an investigational enzyme replacement therapy designed to reduce injection volume

Rocket Pharma gets FDA nod for first pediatric gene therapy targeting severe LAD-I

The approval covers pediatric patients without a matched sibling donor for stem cell transplant

Shionogi launches global Phase 2 trial for late-onset Pompe disease treatment

Esprit is a multicenter, randomized, placebo-controlled, double-blind study assessing the safety, pharmacodynamics, and preliminary efficacy of S-606001 as a substrate reduction therapy

FDA nod to first treatment for rare genetic brain disorder

The approval comes after a systematic review of published literature