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Intravitreal Gene Therapy
Intravitreal gene therapy represents an approach to treating retinal diseases by delivering therapeutic genes directly into the vitreous humor of the eye. This method uses a viral vector, often an adeno-associated virus (AAV), to carry genetic material into retinal cells. Once inside, the therapeutic genes are expressed to address genetic deficiencies or modify biological pathways, offering a long-term or potentially permanent treatment ("biofactory approach") for conditions like wet age-related macular degeneration (AMD), diabetic macular edema, and inherited retinal dystrophies. Unlike traditional therapies requiring frequent injections, intravitreal gene therapy aims to reduce the treatment burden while improving efficacy potentially providing lifelong benefit. Advantages and challenges One of the key advantages of intravitreal administration is its minimally invasive nature compared to subretinal delivery. Intravitreal injections are already commonly used for administering ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Retinal Diseases
The retina (; or retinas) is the innermost, photosensitivity, light-sensitive layer of tissue (biology), tissue of the eye of most vertebrates and some Mollusca, molluscs. The optics of the eye create a focus (optics), focused two-dimensional image of the visual world on the retina, which then processes that image within the retina and sends nerve impulses along the optic nerve to the visual cortex to create visual perception. The retina serves a function which is in many ways analogous to that of the photographic film, film or image sensor in a camera. The neural retina consists of several layers of neurons interconnected by Chemical synapse, synapses and is supported by an outer layer of pigmented epithelial cells. The primary light-sensing cells in the retina are the photoreceptor cells, which are of two types: rod cell, rods and cone cell, cones. Rods function mainly in dim light and provide monochromatic vision. Cones function in well-lit conditions and are responsible fo ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Adverum Biotechnologies
Adverum Biotechnologies, Inc., formerly known as Avalanche Biotechnologies, is a publicly traded clinical stage gene therapy company located in Redwood City, California. The company is targeting unmet medical needs for serious ocular and rare diseases, including wet age-related macular degeneration (wet AMD). History Avalanche Biotechnologies was founded in 2006 by Tom Chalberg, Mark Blumenkranz, Mitchell Finer, and Steven Schwartz. Avalanche went public through an IPO in 2014, raising $102 million. In 2015, co-founder Tom Chalberg stepped down as CEO following Phase IIa trial results that were labeled as "iffy". In May 2016, Avalanche acquired Annapurna Therapeutics and changed its name to Adverum Biotechnologies. It also changed its ticker symbol on the Nasdaq exchange from AAVL to ADVM. In October 2016, former Annapurna Therapeutics CEO Amber Salzman became CEO of Adverum. In May 2018, CEO Amber Salzman left Adverum, and Chief Medical Officer Athena Countouriotis resigned ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Immunogenicity
Immunogenicity is the ability of a foreign substance, such as an antigen, to provoke an immune response in the body of a human or other animal. It may be wanted or unwanted: * Wanted immunogenicity typically relates to vaccines, where the injection of an antigen (the vaccine) provokes an immune response against the pathogen, protecting the organism from future exposure. Immunogenicity is a central aspect of vaccine development. * Unwanted immunogenicity is an immune response by an organism against a therapeutic antigen. This reaction leads to production of anti-drug-antibodies (ADAs), inactivating the therapeutic effects of the treatment and potentially inducing adverse effects. A challenge in biotherapy is predicting the immunogenic potential of novel protein therapeutics. For example, immunogenicity data from high-income countries are not always transferable to low-income and middle-income countries. Another challenge is considering how the immunogenicity of vaccines changes wi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Retinoschisis
Retinoschisis is an eye disease characterized by the abnormal splitting of the retina's neurosensory layers, usually in the outer plexiform layer. Retinoschisis can be divided into degenerative forms which are very common and almost exclusively involve the peripheral retina and hereditary forms which are rare and involve the central retina and sometimes the peripheral retina. The degenerative forms are asymptomatic and involve the peripheral retina only and do not affect the visual acuity. Some rarer forms result in a loss of vision in the corresponding visual field.Cassin, B. and Solomon, S. ''Dictionary of Eye Terminology''. Gainesville, Florida: Triad Publishing Company, 1990. Almost all cases are X-linked recessive and caused by a mutation in the retinoschisin gene (''RS1''). Classification *Hereditary **X linked juvenile retinoschisis **Familial foveal retinoschisis *Tractional *Exudative **Secondary to optic disc pit *Degenerative **Typical **Reticular Degenerative reti ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Botaretigene Sparoparvovec
Botaretigene sparoparvovec (AAV5-RPGR) is an experimental gene therapy for X-linked retinitis pigmentosa developed by Janssen Pharmaceuticals Johnson & Johnson Innovative Medicine (formerly Janssen Pharmaceuticals) is a Belgian pharmaceutical company headquartered in Beerse, Belgium, and wholly owned by Johnson & Johnson. It was founded in 1953 by Paul Janssen. In 1961, Janssen Ph .... References {{reflist Experimental gene therapies Janssen Pharmaceutica ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Dry AMD
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-fo ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Janssen Pharmaceuticals
Johnson & Johnson Innovative Medicine (formerly Janssen Pharmaceuticals) is a Belgian pharmaceutical company headquartered in Beerse, Belgium, and wholly owned by Johnson & Johnson. It was founded in 1953 by Paul Janssen. In 1961, Janssen Pharmaceuticals was purchased by New Jersey–based American corporation Johnson & Johnson, and became part of Johnson & Johnson Pharmaceutical Research and Development (J&J PRD), later renamed to Janssen Research and Development, LLC (JRD), which conducts research and development activities related to a wide range of human medical disorders, including mental illness, neurological disorders, anesthesia and analgesia, gastrointestinal disorders, fungal infection, HIV/AIDS, allergies and cancer. Janssen and Ortho-McNeil Pharmaceutical have been placed in the Ortho-McNeil-Janssen group within Johnson & Johnson Company. Subsidiaries * Actelion * Cilag AG * Janssen Biotech (formerly ''Centocor'') * Janssen Vaccines (formerly ''Crucell'') * ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ixoberogene Soroparvovec
Ixoberogene soroparvovec (, also known as ADVM-022), is a gene therapy developed by Adverum Biotechnologies Adverum Biotechnologies, Inc., formerly known as Avalanche Biotechnologies, is a publicly traded clinical stage gene therapy company located in Redwood City, California. The company is targeting unmet medical needs for serious ocular and rare disea ... for wet age-related macular degeneration. It is delivered via the viral vector AAV.7m8. References {{reflist Experimental gene therapies ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ophthalmology
Ophthalmology (, ) is the branch of medicine that deals with the diagnosis, treatment, and surgery of eye diseases and disorders. An ophthalmologist is a physician who undergoes subspecialty training in medical and surgical eye care. Following a medical degree, a doctor specialising in ophthalmology must pursue additional postgraduate residency training specific to that field. In the United States, following graduation from medical school, one must complete a four-year residency in ophthalmology to become an ophthalmologist. Following residency, additional specialty training (or fellowship) may be sought in a particular aspect of eye pathology. Ophthalmologists prescribe medications to treat ailments, such as eye diseases, implement laser therapy, and perform surgery when needed. Ophthalmologists provide both primary and specialty eye care—medical and surgical. Most ophthalmologists participate in academic research on eye diseases at some point in their training and many inc ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Intravitreal Administration
Intravitreal administration is a route of administration of a drug, or other substance, in which the substance is delivered into the vitreous humor of the eye. "Intravitreal" literally means "inside an eye". Intravitreal injections were first introduced in 1911 when Ohm gave an injection of air into the vitreous humor to repair a detached retina. In the mid-1940s, intravitreal injections became a standard way to administer drugs to treat endophthalmitis and cytomegalovirus retinitis. Epidemiology Intravitreal injections were proposed over a century ago however the number performed remained relatively low until the mid 2000s. Until 2001, intravitreal injections were mainly used to treat end-ophthalmitis. The number of intravitreal injections stayed fairly constant, around 4,500 injections per year in the US. The number of injections tripled to 15,000 in 2002 when triamcinolone injections were first used to treat diabetic macular oedema. This use continued to drive an increase to ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Inflammation
Inflammation (from ) is part of the biological response of body tissues to harmful stimuli, such as pathogens, damaged cells, or irritants. The five cardinal signs are heat, pain, redness, swelling, and loss of function (Latin ''calor'', ''dolor'', ''rubor'', ''tumor'', and ''functio laesa''). Inflammation is a generic response, and therefore is considered a mechanism of innate immunity, whereas adaptive immunity is specific to each pathogen. Inflammation is a protective response involving immune cells, blood vessels, and molecular mediators. The function of inflammation is to eliminate the initial cause of cell injury, clear out damaged cells and tissues, and initiate tissue repair. Too little inflammation could lead to progressive tissue destruction by the harmful stimulus (e.g. bacteria) and compromise the survival of the organism. However inflammation can also have negative effects. Too much inflammation, in the form of chronic inflammation, is associated with variou ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Anti-VEGF
Anti–vascular endothelial growth factor therapy, also known as anti-VEGF () therapy or medication, is the use of medications that block vascular endothelial growth factor. This is done in the treatment of certain cancers and in age-related macular degeneration. They can involve monoclonal antibodies such as bevacizumab, antibody derivatives such as ranibizumab (Lucentis), or orally-available small molecules that inhibit the tyrosine kinases stimulated by VEGF: sunitinib, sorafenib, axitinib, and pazopanib (some of these therapies target VEGF receptors rather than the VEGFs). Both antibody-based compounds and the first three orally available compounds are commercialized. The latter two, axitinib and pazopanib, are in clinical trials. Bergers and Hanahan concluded in 2008 that anti-VEGF drugs can show therapeutic efficacy in mouse models of cancer and in an increasing number of human cancers. But, "the benefits are at best transitory and are followed by a restoration of tumour g ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
