SPLICEBIO POLICY ON EXPANDED ACCESS TO INVESTIGATIONAL THERAPIES
At SpliceBio, we are extremely sympathetic to patients and families seeking care for loved ones diagnosed with devastating genetic diseases. Our mission is to develop and deliver next-generation gene therapies for individuals living with rare genetic disorders, such as Stargardt’s disease. Our team is working tirelessly to overcome the limitations of genetic medicine to efficiently deliver large genes. Our unique Protein Splicing platform is unlocking the potential of gene therapy to bring life-changing treatments that directly target the root cause of genetic diseases.
OUR COMMITMENT TO CLINICAL RESEARCH
At SpliceBio, we are committed to working collaboratively with researchers, healthcare professionals, clinical trial sites, patients, and families to enroll, conduct, and analyze our clinical studies. If successful, data from these trials may support submissions to regulatory authorities such as the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for approval – with the ultimate goal of making our therapies available to as many eligible patients as safely and efficiently as possible.
EXPANDED ACCESS / COMPASSIONATE USE POLICY
At this time, SB-007 is an investigational gene therapy that is only available through participation in ongoing clinical trials. More information on the ongoing ASTRA study can be found here.
There is currently no “Expanded Access Program”. Expanded access is sometimes also referred to as compassionate use or pre-approval access, which is sometimes offered by companies, typically at a later stage of drug development when there are more data available on the investigational product.
If you have questions regarding our Expanded Access Policy, please ask your doctor to contact us through our form.
We will continue to evaluate the possibility of expanded access as we advance development of our investigational medicines. SpliceBio may revise this Expanded Access Policy at any time in keeping with the 21st Century Cures Act.
