Sangamo Biosciences Inc., of Richmond, Calif., presented new preclinical data in disease models from its In Vivo Protein Replacement Platform (IVPRP) programs for MPS I (Hurler syndrome) and MPS II (Hunter syndrome). The data demonstrate that the company’s IVPRP approach enabled stable production of therapeutic levels of replacement enzyme from the liver into the circulation and secondary tissues, including the brain, resulting in significant reduction in biomarkers of the disease and, importantly, statistically significant improvements in cognitive function in treated animals. Read More

LONDON – Neuro-Bio Ltd. has delivered preliminary proof for a previously unknown mechanism it says lies at the heart of the neuronal cell death that typifies Alzheimer’s disease. Read More

HONG KONG – South Korea’s Gtreebnt Co. Ltd., a firm that started out researching and developing software, is expanding its drug pipeline, most recently through a deal with a U.S. medical research foundation to develop a product for a lethal form of brain cancer. Read More

A House committee met Wednesday to review the current state of preparedness for the Zika virus, a threat that has the attention of a number of federal agencies. However, the matter of a funding request by the Obama administration dominated the discussions, including whether funding for the federal government’s response to the Ebola virus could be diverted for use in the response to the Zika virus. Read More

More than 100 years since the oncogene SRC first came to the attention of the biomedical research community, researchers have for the first time identified individuals with a germline SRC mutation. Read More

Capricor Therapeutics Inc. designed its Duchenne muscular dystrophy (DMD) trial to fit “exactly what the FDA has now mandated they want” in the troubled space, CEO Linda Marbán told BioWorld Today, and the firm’s CAP-1002 represents “a novel take and a practical approach” to the disease. Read More

In his first Senate budget hearing as FDA commissioner, Robert Califf committed Wednesday to more responsive interaction with lawmakers and a shoring up of the science behind the agency’s actions as he laid out his top priorities concerning drugs and devices. Read More

DUBLIN – Avvinity Therapeutics Ltd., a newly created joint venture between Horizon Discovery Group plc and start-up firm Centauri Therapeutics Ltd., is getting up to £5.3 million (US$7.4 million) in series A funding to apply Centauri’s Alphamer anti-infectives technology to immuno-oncology indications. Read More