For people living with rare and common genetic diseases

Primary Ciliary Dyskinesia (PCD)

PCD is a rare genetic disorder that leads to chronic respiratory infections. Learn more about PCD and ReCode’s work developing a new PCD treatment caused by mutations in the DNAI1 gene, an essential gene for ciliary movement.

Cystic Fibrosis (CF)

ReCode is developing an inhaled mRNA therapy for the 10-13 percent of CF patients who have Class I mutations in the CFTR gene and do not respond to currently approved CFTR modulators.

ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. We are committed to conducting clinical trials to evaluate the safety and efficacy of investigational therapies to obtain the necessary marketing approvals that allow physicians to prescribe them so individuals may have broad access.

In some circumstances, a treating physician may make a request to provide an unapproved therapy to a qualifying individual, which must be approved by the U.S. Food and Drug Administration (FDA) and supported by the pharmaceutical manufacturer. This is called an expanded access program (EAP), sometimes known as “compassionate use,” and refers to a potential pathway for a patient with a serious or life-threatening disease or condition to try an investigational therapy outside of a clinical trial when there is no comparable or satisfactory therapy available.

We understand that some patients may wish to access our unapproved investigational drugs. At this stage of our development, we do not have the internal infrastructure in place nor enough clinical product or evidence of clinical safety and efficacy of our investigational drugs to support an expanded access program. Therefore, ReCode does not currently offer an expanded access program.