Otsuka Pharmaceutical is acquiring Jnana Therapeutics, whose lead drug is a potential treatment for the rare metabolic disorder phenylketonuria (PKU). Jnana’s drug could provide an alternative to two FDA-approved PKU medicines from BioMarin Pharmaceutical.
PTC Therapeutics plans to talk to regulators about regulatory submissions for sepiapterin after the drug met the main goal of a Phase 3 study. The small molecule is a potential treatment for phenylketonuria, a rare enzyme deficiency.
Health care professionals can refer patients to the best options for diagnostic imaging covered by Medicare to give more people the medical assistance they need without extra financial stress.
A collaboration between Nvidia and startup Evozyne was able to produce novel versions of a human protein never before seen in nature but with enhanced function and the same safety as native protein. The research lays the groundwork for potential new therapies for a rare inherited disorder.
A Phase 1/2 study of BioMarin Pharmaceutical’s gene therapy for phenylketonuria has been placed under a clinical hold after interim results from a preclinical study showed that some mice developed liver tumors. Patients did not receive the same high dose that was given to the mice, and no one in the clinical trial has developed cancer.
The company plans to enter the clinic by the close of 2016 with a red blood cell-based therapy that treats phenylketonuria.