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Rare & Genetic Diseases
| 5 min read
Emerging optogenetic, gene, and cell therapies are moving beyond single-gene treatments, offering hope for a broader range of patients with retinitis pigmentosa.
| 2 min read
The milestone marks meaningful progress toward developing new therapeutic options for Alport syndrome, with first-patient dosing expected in early 2026.
| 3 min read
The partnership leverages real-world patient data and advanced analytics to support faster, more patient-centered drug discovery.
| 4 min read
Cross-industry collaborations, coupled with technology innovations, could help bring CRISPR therapies to millions of patients with genetic diseases.
| 6 min read
New AI tools promise to speed up and simplify CRISPR experiments, but their real-world impact is still being tested.
| 5 min read
A new study finds that non-core and non-essential procedures may add significant burden to participants and research sites.
| 4 min read
Greater focus on non-animal testing methods in the UK, a new CRISPR treatment to lower cholesterol, and positive results for a BTK inhibitor in MS led the news this week.
| 4 min read
Radiprodil is in development as the first therapy designed to directly address the underlying biology of GRIN-related neurodevelopmental disorder.
| 6 min read
With the first wave of FDA-approved siRNA drugs under their belt, companies are racing to expand into new tissues and indications.
| 5 min read
After a decade of promise, AAV therapies are now confronting economic, clinical, and regulatory hurdles.
| 3 min read
People with erythropoietic protoporphyria (EPP) must go to great lengths to avoid sun exposure. There have been no approved treatments that address the disease mechanism, but a new drug could change that.
| 4 min read
The HDAC inhibitor givinostat has received regulatory approval in the US, EU, and UK for patients six years and older.
