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 syndrome 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 syndrome 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 syndrome 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

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. 

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.

RPE65-associated retinal dystrophy is an ultra-rare IRD which impairs vision from birth and results in the degeneration of the entire retina over time. RPE65-associated retinal dystrophy is caused by variants in the RPE65 gene.

Most RPE65-deficient patients experience poor vision in low-light conditions from a young age and suffer from central vision loss that progresses to complete blindness by early adulthood.

One in 125,000 people in the U.S. are expected to be living with RPE65-associated retinal dystrophy.* There are estimated to be approximately 6,000 RPE65-deficient patients in the U.S., Japan and EU5, with almost 30% of those patients under the age of 30 years old. Approximately 50 new cases are diagnosed annually.

RPE65-associated retinal dystrophy is often characterized as a specific subtype of Leber congenital amaurosis (LCA) caused by disease-causing variants in the RPE65 gene (LCA2), or a specific subtype of retinitis pigmentosa caused by disease-causing variants in the RPE65 gene (RP20).

*Based on an estimated prevalence of approximately one in 500,000 people in the U.S. with LCA related to disease-causing variants in the RPE65 gene, and approximately one in 70,000 people in the U.S. with RP due to disease-causing variants in the RPE65 gene.

Achromatopsia (ACHM) is an IRD that specifically prevents cone photoreceptors from functioning properly. As a result, those with ACHM are legally blind from birth and usually suffer from severely reduced visual acuity, a disabling sensitivity to light (also known as photophobia), total color blindness, and involuntary back and forth eye movements (also known as nystagmus).

ACHM occurs in approximately one in 30,000 people in the U.S. and is estimated to impact approximately 24,000 patients in the U.S., EU5, and Japan. To date, mutations in six different genes have been identified as causing ACHM, however the CNGB3 and CNGA3 genes are the two most common of these genes, together accounting for up to 90% of ACHM cases.¹

¹ Koma´romy, A., Alexander, J., Rowlan, J., Garcia, M., Chiodo, V., Tanaka, J., Acland, G., Hauswirth, W., & Aguirre, G. (2010). Gene therapy rescues cone function in congenital achromatopsia. Human Molecular Genetics, 19(13), 2581–2593. doi: 10.1093/hmg/ddq136

Achromatopsia (ACHM) is an IRD that specifically prevents cone photoreceptors from functioning properly. As a result, those with ACHM are legally blind from birth and usually suffer from severely reduced visual acuity, a disabling sensitivity to light (also known as photophobia), total color blindness, and involuntary back and forth eye movements (also known as nystagmus).

ACHM occurs in approximately one in 30,000 people in the U.S. and is estimated to impact approximately 24,000 patients in the U.S., EU5, and Japan. To date, mutations in six different genes have been identified as causing ACHM, however the CNGB3 and CNGA3 genes are the two most common of these genes, together accounting for up to 90% of ACHM cases.¹

¹ Koma´romy, A., Alexander, J., Rowlan, J., Garcia, M., Chiodo, V., Tanaka, J., Acland, G., Hauswirth, W., & Aguirre, G. (2010). Gene therapy rescues cone function in congenital achromatopsia. Human Molecular Genetics, 19(13), 2581–2593. doi: 10.1093/hmg/ddq136

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 for compassionate use by MeiraGTx under a Specials license in the UK and has received rare pediatric disease designation (RPDD) from FDA.

RDH12
RDH12-associated retinal dystrophy, often characterized as a specific subtype of Leber congenital amaurosis or retinitis pigmentosa, is a severe IRD caused by variants in the RDH12 gene. Mutations in the RDH12 gene cause widespread retinal degeneration, resulting in marked central visual loss by 20 years of age. MeiraGTx is investigating delivery of a functional copy of the RDH12 gene to the retina of RDH12-deficient patients. AAV5-RDH12 received rare pediatric disease designation (RPDD) from FDA.

BBS10
Bardet-Biedl Syndrome (BBS) is a rare genetic disease affecting approximately 1 in 250,000 people around the world.  One of the primary symptoms of BBS is visual impairment secondary to retinal degeneration.  More than 20 different genes are associated with the development of BBS, with BBS10 accounting for approximately 25% of cases. AAV8-RK-BBS10 received rare pediatric disease designations (RPDD) from FDA.

ABCA4
ABCA4-associated Stargardt disease, is a type of macular degeneration caused by mutations in the ABCA4 gene. Stargardt disease results in marked central visual loss by first or second decade of life and it is one of the commonest IRDs. MeiraGTx is investigating novel approaches for the delivery of full-length functional ABCA4 to the retina of ABCA4-deficient patients.

KCNV2
KCNV2-associated retinal dystrophy is a form of cone dystrophy which is characterized by progressive cone-rod loss. Onset of symptoms in early childhood with total blindness by 2nd to 4th decade of life.