Programs & Pipeline

Xerostomia, a chronic and debilitating disorder of the salivary glands in which saliva production is impaired. Xerostomia has a number of different potential causes, including radiation therapy for head and neck cancer. Radiation as a treatment for head and neck cancer can cause irreversible damage to non-diseased tissues located near oral tumors, such as the salivary glands.

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Sjogren’s disease is an autoimmune disease in which the immune system may target the salivary glands, causing chronic inflammation and long-term damage of the glands.

Sjogren’s disease is one of the most prevalent autoimmune diseases, believed to affect nearly four million Americans.¹ Nine out of ten diagnosed patients are women, however Sjogren’s can be diagnosed in all ages, races and genders.¹

Symptoms of Sjogren’s disease vary from person to person, however the two most common symptoms are dry mouth and dry eye.² Other symptoms may include, dental decay, dry or peeling lips, dry or burning throat, or difficulty swallowing, chewing, or speaking.¹

¹ https://www.sjogrens.org/understanding-sjogrens

² Sjogrens foundation Harris poll 2016 https://www.sjogrens.org/understanding-sjogrens/resources/patient-survey-results

Parkinson’s disease is a progressive neurodegenerative disorder affecting nearly one million Americans and approximately 10 million people worldwide.

Parkinson’s is caused by degeneration of dopaminergic neurons which affect motor control and function. In addition to cardinal symptoms such as shaking or tremors, bradykinesia (slow movement), and rigidity, dopamine loss also commonly leads to additional non-motor symptoms, including cognitive impairment, mood, and behavioral changes.

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Amyotrophic lateral sclerosis (ALS) is a progressive, neurodegenerative disease leading to the loss of motor neurons, which are critical to a person’s ability to move, speak, swallow and ultimately to breathe. Over time, loss of motor neurons gradually leads to paralysis and invariably, death.

Mutations in over 20 genes have been identified that cause the inherited ALS cases.¹ While 10 percent of ALS cases are caused by inherited genetic mutations, most ALS occurs sporadically, with no known genetic cause. Rather than targeting a specific genetic defect that defines a small subset of ALS patients, we aim to use gene therapy to target the underlying cell biology driving motor neuron death, potentially enabling us to treat both sporadic and inherited forms of the disease.

¹Taylor, J., Brown Jr., R., & Cleveland, D. (2016). Decoding ALS: from genes to mechanism. Nature, 539, (197–206). doi:10.1038/nature20413

X-linked retinitis pigmentosa (XLRP) is a severe form of retinitis pigmentosa, a group of IRDs that that cause serious vision impairment and blindness. Retinitis pigmentosa affects about 1 in 3,500 people, with XLRP accounting for approximately 15% of all retinitis pigmentosa cases.

Visual impairment in XLRP initially manifests with loss of night vision during adolescence, followed by progressive constriction of the visual field in adulthood, and eventual advancement to central vision loss and legal blindness by 30 to 40 years of age. Due to its inheritance pattern, XLRP affects men and women differently, with men more likely to experience severe symptoms. One in 40,000 people in the U.S. are expected to be living with XLRP, and it’s estimated that approximately 20,000 XLRP patients are among the U.S., Japan and EU5.

XLRP is most commonly caused by a defect in the RPGR gene, which encodes a protein called X-linked retinitis pigmentosa GTPase regulator (RPGR) that plays a vital role in the development of the cells that make up the retina, a thin layer of tissue found on the back wall of the eye. Absence of this functional protein results in poorly functioning rod and cone photoreceptors, parts of the eye which are responsible for vision at low and high light levels, as well as seeing color.

AIPL1-associated Leber congenital amaurosis 4 (LCA4) is an ultra-rare and severe inherited retinal disease resulting from mutations in the aryl hydrocarbon receptor interacting protein-like 1 gene (AIPL1). Children with AIPL1-LCA4 are blind from birth and by age four, retinal degeneration is irreversible. 

AAV-AIPL1 is manufactured and released by MeiraGTx under a Specials license in the UK and has received rare pediatric disease designation (RPDD) from FDA.

Mutations in the melanocortin-4 receptor (MC4R) gene are among the most common monogenic causes of obesity, affecting approximately 150,000 patients in the US. Patients with mutations in MC4R experience increased appetite and impaired satiety, resulting in severe obesity that starts within the first few years of life.