This week in drug discovery (14-18 July)
News round-up for 14-18 July by DDW Senior Digital Content Editor Diana Turner.
This week’s news highlights reflect the incredible levels of innovation and investment, but also the hurdles to overcome, in developing gene therapies – breakthrough treatments in many genetic diseases that can bring their own unique challenges in terms of manufacturing, delivery and safety risks.
The top stories:
Atsena expedites gene therapy for X-linked retinoschisis
The US Food and Drug Administration (FDA) has agreed to the expansion of Atsena Therapeutics’ ongoing Phase I/II LIGHTHOUSE study of ATSN-201 into a continuous Phase I/II/III trial in the treatment of X-linked retinoschisis (XLRS).
JCR and Alexion deal boosts AAV-based gene therapy
JCR Pharmaceuticals and Alexion, AstraZeneca Rare Disease, have entered into a license agreement for JCR’s JUST-AAV capsids to develop genomic medicines.
Voyager adds fourth Alzheimer’s disease programme to pipeline
Voyager Therapeutics has added a new programme that modulates the expression of apolipoprotein E (APOE), the strongest genetic risk factor for Alzheimer’s disease (AD).
FDA raises manufacturing concerns for Ultragenyx gene therapy
The US Food and Drug Administration (FDA) has decided not to recommend Ultragenyx Pharmaceutical’s UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA).
Sarepta Therapeutics focuses pipeline and cuts 36% of workforce
Sarepta Therapeutics has announced a strategic restructuring plan designed to prioritise high-value, high impact programmes, and support its long-term financial viability.