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ALK-001
Gildeuretinol is an investigational new drug being developed bAlkeus Pharmaceuticals, Inc.for the treatment of retinal diseases, particularly Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD). Stargardt disease is caused by a defect in the ABCA4 gene that clears toxic byproducts resulting from the dimerization of vitamin A. Gildeuretinol is new molecular entity designed to reduce the dimerization of vitamin A in the eye without affecting the visual cycle. Gildeuretinol has received breakthrough therapy, orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ... and Pediatric Rare Disease designations from the U.S. Food and Drug Administration. References {{pharma-stub Vitamin A Deuterated compounds ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Stargardt Disease
Stargardt disease is the most common inherited single-gene retinal disease. In terms of the first description of the disease, it follows an autosomal recessive inheritance pattern, which has been later linked to bi-allelic ABCA4 gene variants (STGD1). However, there are Stargardt-like diseases with mimicking phenotypes that are referred to as STGD3 and STGD4, and have a autosomal dominant inheritance due to defects with ELOVL4 or PROM1 genes, respectively. It is characterized by macular degeneration that begins in childhood, adolescence or adulthood, resulting in progressive loss of vision. Signs and symptoms The presentation usually occurs in childhood or adolescence, though there is no upper age limit for presentation and late-onset is possible. The main symptom is loss of visual acuity, uncorrectable with glasses. This manifests as the lack of the ability to see fine details when reading or viewing distant objects. Symptoms typically develop before age 20 (median age of ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Investigational New Drug
The United States Food and Drug Administration's Investigational New Drug (IND) program is the means by which a pharmaceutical industry, pharmaceutical company obtains permission to start human clinical trials and to ship an experimental drug interstate commerce, across state lines (usually to clinical investigators) before a marketing application for the drug has been approved. Regulations are primarily at . Similar procedures are followed in the European Union, Japan, and Canada due to regulatory harmonization efforts by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, International Council for Harmonisation. Types * Research or investigator INDs are non-commercial INDs filed by researchers to study an unapproved drug or to study an approved drug for a new indication or in a new patient population. * Expanded access, Emergency Use INDs, also called compassionate use or single-patient INDs, are filed for emergency use of a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Geographic Atrophy
Geographic atrophy (GA), also known as atrophic age-related macular degeneration (AMD) or advanced dry AMD, is an advanced form of age-related macular degeneration that can result in the progressive and irreversible loss of retinal tissue (photoreceptor cell, photoreceptors, retinal pigment epithelium, choriocapillaris) which can lead to a loss of central vision over time. It is estimated that GA affects over 5 million people worldwide and approximately 1 million patients in the US, which is similar to the prevalence of neovascular (wet) AMD, the other advanced form of the disease. The incidence of advanced AMD, both geographic atrophy and neovascular AMD, increases exponentially with age. Most current clinical trials aim to reduce the progression of GA lesion enlargement. Presentation Geographic atrophy is a chronic disease, which leads to visual function loss centrally. This often results in difficulties performing daily tasks such as reading, recognizing faces, and driving, and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Age-related Macular Degeneration
Macular degeneration, also known as age-related macular degeneration (AMD or ARMD), is a medical condition which may result in blurred or no vision in the center of the visual field. Early on there are often no symptoms. Some people experience a gradual worsening of vision that may affect one or both eyes. While it does not result in complete blindness, loss of central vision can make it hard to recognize faces, drive, read, or perform other activities of daily life. Visual hallucinations may also occur. Macular degeneration typically occurs in older people, and is caused by damage to the macula of the retina. Genetic factors and smoking may play a role. The condition is diagnosed through a complete eye exam. Severity is divided into early, intermediate, and late types. The late type is additionally divided into "dry" and "wet" forms, with the dry form making up 90% of cases. The difference between the two forms is categorized by the change in the macula. Those with dry- ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
ABCA4
ATP-binding cassette, sub-family A (ABC1), member 4, also known as ABCA4 or ABCR, is a protein which in humans is encoded by the ''ABCA4'' gene. ABCA4 is a member of the ATP-binding cassette transporter gene sub-family A (ABC1) found exclusively in multicellular eukaryotes. The gene was first cloned and characterized in 1997 as a gene that causes Stargardt disease, an autosomal recessive disease that causes macular degeneration. The ''ABCA4'' gene transcribes a large retina-specific protein with two transmembrane domains (TMD), two glycosylated extracellular domains (ECD), and two nucleotide-binding domains (NBD). The ABCA4 protein is almost exclusively expressed in retina localizing in outer segment disk edges of rod photoreceptors. Structure Previously known as the photoreceptor rim protein RmP or ABCR, the recently proposed ABCA4 structure consists of two transmembrane domains (TMDs), two large glycosylated extracytosolic domains (ECD), and two internal nucleotide binding d ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Vitamin A
Vitamin A is a fat-soluble vitamin that is an essential nutrient. The term "vitamin A" encompasses a group of chemically related organic compounds that includes retinol, retinyl esters, and several provitamin (precursor) carotenoids, most notably Β-Carotene, β-carotene (''beta''-carotene). Vitamin A has multiple functions: growth during embryo development, maintaining the immune system, and healthy vision. For aiding vision specifically, it combines with the protein opsin to form rhodopsin, the light-absorbing molecule necessary for both low-light (scotopic vision) and color vision. Vitamin A occurs as two principal forms in foods: A) retinoids, found in Animal source foods, animal-sourced foods, either as retinol or bound to a fatty acid to become a retinyl ester, and B) the carotenoids Α-Carotene, α-carotene (''alpha''-carotene), β-carotene, Γ-Carotene, γ-carotene (''gamma''-carotene), and the xanthophyll beta-cryptoxanthin (all of which contain β-ionone rings) that ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Breakthrough Therapy
Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "breakthrough therapy" designation is not intended to imply that a drug is actually a "breakthrough" or that there is high-quality evidence of treatment efficacy for a particular condition; rather, it allows the FDA to grant priority review to drug candidates if preliminary clinical trials indicate that the therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases. The FDA has other mechanisms for expediting the review and approval process for promising drugs, including fast track designation, accelerated approval, and priority review. Requirements A breakthrough therapy designation can be assigned to a drug if "it is a drug which is intended alone or in combination wit ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drug
An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug medical research, research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to en ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Vitamin A
Vitamin A is a fat-soluble vitamin that is an essential nutrient. The term "vitamin A" encompasses a group of chemically related organic compounds that includes retinol, retinyl esters, and several provitamin (precursor) carotenoids, most notably Β-Carotene, β-carotene (''beta''-carotene). Vitamin A has multiple functions: growth during embryo development, maintaining the immune system, and healthy vision. For aiding vision specifically, it combines with the protein opsin to form rhodopsin, the light-absorbing molecule necessary for both low-light (scotopic vision) and color vision. Vitamin A occurs as two principal forms in foods: A) retinoids, found in Animal source foods, animal-sourced foods, either as retinol or bound to a fatty acid to become a retinyl ester, and B) the carotenoids Α-Carotene, α-carotene (''alpha''-carotene), β-carotene, Γ-Carotene, γ-carotene (''gamma''-carotene), and the xanthophyll beta-cryptoxanthin (all of which contain β-ionone rings) that ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
