Translational Science Highlight Scientists in NCATS' Assay Development and Screening Technology program are developing new methods for screening small molecules that could help researchers develop new treatments for many diseases. Collabo...

[Read More...]

PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for an adaptive Phase 2/3 trial with t...

[Read More...]

Scientific founders who originated the miRNA mediated “silence and replace” approach take on Alpha-1 and ALS

US patent awarded for its proprietary THRIVE™ platform

Appoints new Chief Technology Officer with rAAV gene therapy cGMP manufacturing and virology expertise

 

Apic Bio, Inc. an innovative gene therapy company developing best-in-class treatment options for patients with rare genetic diseases, today announced the completion of a $40 million Series A financing led by Morningside Venture. Existing investors, The Alpha-1 Project (TAP) and A1ATD Investors, LLC also participated along with new investor, the ALS Investment Fund.

[Read More...]

Based on the most recent data from the ADVANCE clinical trial, the Company is reviewing the learnings from the study, notably on the AAVrh.10 capsid in order to inform further development of gene therapy candidates for the treatment of rare diseases. The Company plans to conduct additional preclinical studies to determine the best gene therapy candidate to advance. The Company plans to provide an update on the rare disease programs in the first half of 2019 and will not submit an IND application for ADVM-053 for the treatment of hereditary angioedema (HAE) in the fourth quarter of 2018.

[Read More...]

London, UK and Redwood City, California, USA, 5 December 2018 – Mereo BioPharma Group plc (AIM: MPH) (“Mereo”, the “Company” or the “Group”), the clinical stage UK based biopharmaceutical company focused on rare diseases, and OncoMed Pharmaceuticals, Inc. (NASDAQ: OMED) (“OncoMed”), a NASDAQ listed oncology-focused clinical stage biopharmaceutical business, today announce the proposed combination of Mereo and OncoMed (the “Transaction”). The Transaction has been unanimously approved by the Board of Directors of each company.

[Read More...]