Genomics

Tenaya Therapeutics has secured RMAT designation for TN-401, its AAV9-based gene therapy for PKP2-associated ARVC, backed by Phase 1b/2 interim data.

The US ad watchdog ruled that Nucleus IVF+ marketing overstated accuracy for disease risk, height, IQ and eye colour; Nucleus will appeal.

CAMP4 Therapeutics has secured MHRA authorisation to include UK sites in its global Phase 1/2 study of CMP-002, an ASO targeting SYNGAP1-related

MolDX has issued a proposed LCD that could establish Medicare coverage for optical genome mapping in hematologic malignancy workups.

All five Cohort 1 participants showed clinically meaningful visual improvement, and the FDA has aligned on a potential pivotal endpoint for the rare retinal disease

A landmark DNA study of Western North Atlantic white sharks has identified three full sibling pairs and seven likely half-sibling pairs, challenging the species'

The USPTO notice covers LSD1 inhibitor combinations for myeloid cancers, with protection expected to run to 2042 pending formal grant.

Tarsus gains worldwide rights to Phase 3 oral candidate ALK-001 for Stargardt disease, a blinding inherited retinal condition with no approved therapy.

Kala Bio will be the sole US distributor of Virotek's ophthalmology genomic testing programme under a one-year deal effective 4 September 2026.

Four-year open-label extension data showed durable seizure reductions and cognitive gains in Dravet syndrome; Phase 3 readout is expected in Q3 2027.

Ultragenyx's apazunersen missed both its primary and key secondary endpoints in the Phase 3 Aspire study, leaving Angelman syndrome without an approved treatment.

The Alameda-based CRISPR company began a Phase 1 trial of its epigenetic LDL-C therapy and secured $25m in non-dilutive CIRM grants alongside

Kala Bio will market Virotek's CLIA-registered ophthalmology genetic screening programme across U.S. eye care practices, targeting a definitive deal within 30 days.

Medicus Pharma has received UAE DOH authorisation to begin PRECISION-E2, a Phase 2a genomics-integrated study of Teverelix in 84 women with endometriosis.

BioNexus Gene Lab Corp. is targeting 2026 deployment of its VitaGuard liquid-biopsy platform in Malaysia and Singapore after a technical visit to its Chinese

DiamiR Biosciences and AlzLabs will combine protein and microRNA blood biomarkers to improve early detection of Alzheimer's disease.

The Barcelona epigenetics biotech priced 4.4 million new shares at €2.70 each and secured a €25m anchor commitment from Spain's Social Impact

A pooled analysis of 8,478 patients shows Myriad's Prolaris Biopsy Test adds prognostic value beyond standard clinical risk tools in localised prostate cancer.

Roche's Krascendo 1 study showed divarasib improved both progression-free and overall survival versus sotorasib or adagrasib in previously treated KRAS G12C lung cancer

The peer-reviewed Oxford study of 409 men over six years underpins the GPS-ProtecT randomised controlled trial of mdxhealth's 17-gene prostate assay.