Latest Genetic Diseases News

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.

PYC Therapeutics has launched a A$145.8 million pro-rata accelerated non-renounceable entitlement offer to fund late-stage clinical trials across its pipeline targeting rare diseases with high unmet needs. The capital raise aims to extend the company’s cash runway beyond FY27, supporting four drug candidates through critical human safety and efficacy milestones.

PYC Therapeutics is set to raise up to $146 million through a pro-rata entitlement offer, aiming to accelerate its RNA-based drug candidates targeting major unmet medical needs. Major shareholder support and underwriting commitments underpin the capital raise.

PYC Therapeutics has secured regulatory approval to initiate human trials of PYC-003, a novel drug candidate targeting the root cause of Polycystic Kidney Disease (PKD). Initial safety and efficacy data are expected within 2025, marking a critical step in addressing a disease affecting millions worldwide.

PYC Therapeutics has secured Safety Review Committee approval to escalate dosing in its clinical trial for Autosomal Dominant Optic Atrophy, marking a pivotal step in developing a treatment for this currently untreatable blinding disease.