SEEING NEW WAYS FORWARD

Revolutionizing the treatment of blinding eye diseases

We are a leading clinical-stage gene therapy company focused on developing and commercializing novel, one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness.

Our mission is to maintain and restore sight in patients with inherited retinal diseases.

Our patients are at the forefront of everything we do. They inspire us to work hard every day in an effort to bring sight-saving therapies to people with blinding eye diseases.

LEARN MORE ABOUT US

OUR CLINICAL PROGRAMS

CHOROIDEREMIA

We are evaluating NSR-REP1, a gene therapy for the treatment of choroideremia (CHM), a rare, degenerative, genetic retinal disorder that presents itself in childhood and generally leads to vision loss in early adulthood and total blindness thereafter.

LEARN ABOUT OUR CHM PROGRAM

X-LINKED RETINITIS PIGMENTOSA

Our NSR-RPGR gene therapy is being evaluated for the treatment of X-linked retinitis pigmentosa (XLRP), an inherited X-linked recessive retinal disease that causes night blindness during childhood, followed by a narrowing of peripheral vision and progressive loss of central vision and eventually legal or total blindness.

LEARN ABOUT OUR XLRP PROGRAM

OUR CLINICAL TRIALS

If you are a patient or caregiver interested in one of our trials, we want to hear from you. Please visit the link below to request additional information.

CLINICAL INFORMATION REQUEST

PATIENT STORIES

Toby Stroh

I start my story with some defining comments that were made to me in my 20’s after I’d been misdiagnosed with retinitis pigmentosa.

FULL STORY

Jonathan Wyatt

I am lucky. My choroideremia condition did not develop to disadvantage me until I was well into middle age.

FULL STORY

Make a Difference with Us

Our team is committed to bringing life-changing medicines for the treatment of people with inherited retinal diseases to the market as quickly as possible. We are looking for driven and inspired people to make a difference with us and help us advance the development of our therapies for inherited retinal diseases.

Open Positions