California Gene Therapy Program Expresses Condolences Following Death of Patient in Clinical Trial

CIRM backed the research with $5.8 million

The $12 billion California gene therapy program says it is “deeply saddened” by the death of a patient in a gene therapy trial that it helped to finance and is closely monitoring the situation to “ensure the highest standards of safety and ethics are upheld in this research.”

The death occurred in a phase two clinical trial for Danon disease that was supported by $5.8 million from the California Institute for Regenerative Medicine (CIRM). It is the first death of a patient in the 116 clinical trials that CIRM has helped to finance.

Danon is a rare, fatal genetic affliction. The company involved is Rocket Pharmaceutical, Inc., of New Jersey. The CIRM portion of the trial was conducted at UC San Diego.

Rocket did not release the name, age or gender of the patient or the location of trial site where the fatality occurred.

“We are deeply saddened to learn of the passing of a subject enrolled in Rocket Pharmaceuticals’ trial,” said Jonathan Thomas, CEO of CIRM, in response to a query by the California Stem Cell Report.

“As patient safety remains CIRM’s highest priority, we are closely monitoring the investigation and will continue to work with Rocket to ensure the highest standards of safety and ethics are upheld in this research,” Thomas continued.

“CIRM remains committed to supporting innovative research aimed at treating rare and life-threatening diseases. We will continue to prioritize patient safety and transparency as we move forward.” (The full text of Thomas’ remarks can be found at the end of this article.)

Most of the news coverage last week of the patient's death focused on the medical aspects of the situation. Barron’s, however, also addressed the impact on the gene therapy industry.

“The death poses yet another challenge for the field. Once an area of enormous optimism for biotech investors and pharma companies, gene therapies have had little commercial success thus far,” wrote Josh Nathan-Kazis for Barron’s.