Charles Moore,  —

The Ensuring Access to Clinical Trials Act of 2015 (EACT) has been passed by the U.S. House of Representatives, and having already been passed the Senate in July, the bill will now go to President Obama to be signed into law. The EACT bill authorizes individuals to receive up to…

Dallas-based gene therapy platform company AveXis Inc., a leader in developing treatments for rare and life-threatening neurological genetic diseases, has announced completion of a $65 million Class D common stock financing. The proceeds will be used to advance the company’s ongoing Spinal Muscular Atrophy (SMA) research and clinical programs…

Biogen, a Cambridge, Massachusetts based developer, manufacturer and marketer of innovative therapies for patients living with serious neurological, autoimmune and hematologic disorders, in collaboration with Isis Pharmaceuticals — a leader in discovery and and development of drugs based on antisense and RNAi technology and focused on RNA…

Dallas, TX based AveXis has announced that company co-founder John Carbona has resigned from his position as CEO. Mr. Carbona will remain a Director of AveXis. This announcement stems from a succession plan, the development of which has been in process as AveXis advances clinical development of its…

Cure SMA, an organization dedicated to the treatment and cure of Spinal Muscular Atrophy (SMA) — a severe and devastating neuromuscular disease that is one of the most common fatal genetic disorders, occurring in 1 in every 6,000 to 10,000 live births and the number-one genetic cause of infant death…

Carlsbad, California-based Isis Pharmaceuticals, Inc. has received a $7 million milestone payment from Biogen Idec, to advance an ongoing open-label extension clinical study of the drug ISIS-SMNRx in children with spinal muscular atrophy (SMA). The study of ISIS-SMNRx is open to children with SMA who have completed…

South San Francisco based Cytokinetics, Incorporated and Astellas Pharma Inc. of Tokyo, Japan, have announced amendments expanding their research collaboration alliance for development and commercialization of skeletal muscle activators. The two companies have been cooperating on research and development efforts, the objective being to advance novel treatment therapies…

Spinal Muscular Atrophy (SMA) is a genetic disorder that causes progressive muscle wasting and robs the afflicted of physical strength and leads to mobility impairment due to its effect on motor nerve cells in the spinal cord. SMA eventually results in loss of the ability to walk, eat,…