Latest Genetic Diseases News

PYC Therapeutics reports positive safety outcomes from the initial phase of its clinical trial for PYC-003, a promising treatment for Polycystic Kidney Disease. The drug was well tolerated in healthy volunteers, paving the way for patient dosing and further trials.

PYC Therapeutics has reported significant progress across its four RNA therapeutic programs targeting genetic diseases, alongside a strong cash runway supporting ongoing development.

PYC Therapeutics has kicked off a global Phase 1b repeat dose study of its investigational drug PYC-001 targeting Autosomal Dominant Optic Atrophy, a rare genetic eye disease with no current treatments. Initial dosing has begun, with key data expected in the second half of 2026.

PYC Therapeutics has reported encouraging pre-clinical results for its RNA therapy candidate PYC-002 targeting Phelan-McDermid Syndrome, supporting plans to enter human trials in 2026. The data demonstrate safety, brain distribution, and gene expression restoration in key disease regions.

PYC Therapeutics has reported positive safety outcomes from its Phase 1 trial of PYC-001 for Autosomal Dominant Optic Atrophy, paving the way for a global Phase 1/2 study later this year.

PYC Therapeutics reported a 14.5% rise in income to AUD 26.17 million for FY2025 alongside a 33.3% increase in net loss, while increasing its stake in Vision Pharma to 97.1%. The auditor flagged material uncertainty over the company’s ability to continue as a going concern.

PYC Therapeutics has escalated dosing to the highest level in its Phase 1a trial of PYC-003 and begun dosing patients with Polycystic Kidney Disease, marking key progress toward regulatory milestones.

PYC Therapeutics reports solid progress across its four RNA therapeutic programs targeting genetic diseases, backed by a strong cash position extending runway beyond 2027.

PYC Therapeutics has secured Safety Review Committee approval to escalate dosing in its PYC-003 clinical trial and will soon begin dosing patients with Polycystic Kidney Disease, marking a pivotal step forward.

PYC Therapeutics has gained FDA alignment on the design of its pivotal Phase 2/3 trial for VP-001, a promising treatment for the rare childhood blindness RP11. The regulator accepted the trial’s sham control arm and primary endpoints, setting the stage for a critical next step toward drug approval.

PYC Therapeutics has secured Safety Review Committee approval to increase dosing in its clinical trial for a novel treatment targeting Autosomal Dominant Optic Atrophy, a rare blinding genetic disease. This milestone marks a significant step forward in the development of PYC-001, a first-in-class RNA therapy.

PYC Therapeutics has secured approval to escalate dosing in its Phase 1a trial of PYC-003 for Polycystic Kidney Disease, moving closer to patient dosing later this year.