Global Blood Therapeutic Inc.'s homegrown lead candidate is an oral, once-daily drug aimed at sickle cell disease, a genetic condition that causes red blood cells to become deformed or “sickled." The goal with GBT440 is to correct the disease, so that patients would actually have no evidence of it following treatment.

Derived from Strategic Transactions, Informa’s premium source for tracking life sciences deal activity, the Dealmaking column is a survey of recent health care transactions listed by relevant industry segment – In Vitro Diagnostics, Medical Devices, and Pharmaceuticals – and then categorized by type – Acquisition, Alliance, or Financing. This month’s column covers deals announced June-August 2014.

Britain moves ahead with a drug-repurposing research program that brings together big pharma and academic researchers in novel collaborations.

Disease advocacy organizations are expanding their “honest broker” function in drug development into the early stages of the regulatory discussions. They are engaging in the patient-focused meetings established by PDUFA V and finding more ways to interact with FDA review staff on general development issues in their specific areas. That may become the model for building on the patient input process in the next user fee agreement.