Helping patients with gene therapy
Bringing life-changing gene therapeutics to patients with diseases that have a high unmet medical need fuels our research and development pipeline. We have active preclinical and clinical programs for neuromuscular, metabolic, cardiovascular and central nervous system conditions, with particular attention on rare diseases.
Lisa can’t get up from a chair on her own, but she is not letting limb-girdle muscular dystrophy type 2I/R9 stop her from being a working mother and loving wife.
“…the first time I was told I had a rare form of muscular dystrophy, it was a profound moment. I wanted to know more, but they said they had no more information than the diagnosis."
Read Lisa’s story
More patient stories »
Embracing patients
AskFirst™ is our collaborative program for advocacy groups, patients, and their families who may benefit from the latest advances in gene therapy research and potential curative therapies. Guided by the voice of patients, we strive to advance therapeutics, technology, regulatory requirements, and public policy in this area.
We “Ask” how we can help patients and their families and welcome your voice to help us erase genetic disease.
AskFirst is driven by our mission to improve patients’ quality of life and our desire to bring better genetic medicines to market at a lower cost.
- Include the patient and family voice in the design of our clinical studies
- Bring together scientists, technicians, and clinical specialists who engage patients during the therapeutic development process
- Create study protocols to maximize patient outcomes to speed the delivery of novel treatments
- Engage patient advocacy liaisons with direct access to research teams to get answers when needed
Ask Jude
“We must ensure gene therapy can be equally accessible, even to those with ultra-rare diseases. Large-scale, efficient manufacturing of AAV product is critical.”
R. Jude Samulski, PhD
Chief Scientific Officer and Co-Founder
Expanded access policy
Expanded access programs are generally limited to patients with a life-threatening condition who either failed to respond to other conventional treatments, are not eligible for a clinical trial, or are ineligible for other treatments. These expanded access programs are regulated by the U.S. FDA and run as a simple study. They require some evidence to support the drug or treatment’s use in patients.
We are committed to making all approved therapies widely available as rapidly as possible. Indeed, this was part of the rationale for our merger with Bayer, a company with global reach. It is too early at this time to consider plans for expanded access. When we have published results from a controlled clinical trial that suggest a therapy may provide benefit for patients with acceptable safety, we will consider options such as expanded access.
Corporate support
RhiaPharmaceutical supports charitable activities by patient advocacy and education organizations. We can also provide educational materials, clinical study data, and SMEs. Visit our Corporate Support page to learn more.
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