Category: Technology Licenses
Created On: 2022-04-28
Record Count: 6
IPSCIO Report Record List
Below you will find the records curated into this collection. This summary includes the complete licensed property description so that you can review and determine if this collection covers the topics, technology or transaction type that is relevant for your needs. The full report will include all relevant deal data such as the royalty base, agreement date, term description, royalty rates and other deal terms. For reference, here is a sample of a full IPSCIO curated royalty rate report: Sample Report
IPSCIO Record ID: 258420
X-linked retinitis pigmentosa (XLRP) is and inherited condition caused by mutations in the RPGR gene. XLRP causes progressive vision loss in boys and young men. The condition begins with night blindness and is followed by progressive constriction of the field of vision. XLRP often results in total blindness and there is no specific treatment for this condition.
AAV-CNGB3 and AAV-CNGA3, gene therapy candidates designed to restore cone function, are delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. AAV-CNGB3 was granted orphan drug designation (ODD), rare pediatric disease and Fast Track designations by the U.S. Food and Drug Administration (FDA), and orphan medicinal product and PRIME designations by the European Medicines Agency (EMA) for the treatment of achromatopsia caused by mutations in the CNGB3 gene. A Phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients is underway.
X-linked retinitis pigmentosa (XLRP) represent some of the most severe forms of RP, resulting in early onset in childhood and rapid progression to blindness by the time patients reach 20 to 30 years old. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. There are currently no approved treatments for XLRP.
AAV-RGPR is designed to treat the most common form of XLRP caused by mutations in the RPGR gene. Both rod and cone photoreceptors require RPGR to function. AAV-RPGR has received Fast Track designation and ODD from the FDA and orphan medicinal product designation from the EMA. A Phase 1/2 clinical trial of AAV-RPGR in adult and pediatric patients is underway.
This strategic collaboration agreement is related to development and commercialization of gene therapies for the treatment of inherited retinal diseases (IRDs). IRDs are a group of rare eye conditions caused by an inherited gene mutation that are often characterized by progressive retinal degeneration which leads to severe vision impairment, loss or blindness.
IPSCIO Record ID: 251005
IPSCIO Record ID: 28734
IPSCIO Record ID: 339531
The First Amendment has been amended to include but not limited to License Product, Regulatory Exclusivity, Sublicensee Royalty Term, Sublicensee Product and Royalty payments.
Licensed Product) is amended by adding (a) Any product, apparatus, kit, composition, or component thereof (i) whose use, sale, offer for sale, or importation of which is covered, in whole or in part, by any valid claim contained in the Patent Rights or (ii) which is made by any method, procedure, process, or step which is covered, in whole or in part, by any valid claim contained in the Patent Rights; or (b) An isolated synthetic nucleic acid molecule comprising a synthetic mini-dystrophin or micro-dystrophin gene encoding a synthetic, non-full-length, dystrophin protein that is able to restore nNOS to the sarcolemma, wherein the non-full-length dystrophin protein comprises, from the N terminus to the C terminus 1. the N-terminal domain of the dystrophin protein or a modified N-terminal domain of the dystrophin protein, 11. at least two repeats of the mid-rod domain of the dystrophin protein, wherein said at least two repeats comprise R16 and Rl 7, 111. at least 2 hinge regions of the dystrophin protein, whereas said at least two hinge regions comprise HI and H4, and 1v. the cysteine-rich domain of the dystrophin protein; wherein the mini- or micro-dystrophin gene is between 5 kb to about 8 kb in length or less than 5 kb in length, respectively.
Duchenne is caused by mutations in the dystrophin gene, which result in the absence or near-absence of dystrophin protein. Dystrophin protein works to strengthen muscle fibers and protect them from daily wear and tear. Without functioning dystrophin and certain associated proteins, muscles suffer excessive damage from normal daily activities and are unable to regenerate, leading to the build-up of fibrotic, or scar, and fat tissue. Efforts are focused on our lead product candidate, SGT-001, a gene transfer candidate under investigation for its ability to drive functional dystrophin protein expression in patientsâ€™ muscles and improve the course of the disease. Based on our preclinical program that included multiple animal species of different phenotypes and genetic variations, as well as preliminary clinical trial biomarker results, we believe that SGT-001, has the potential to slow or even halt the progression of Duchenne, regardless of the type of genetic mutation or stage of the disease.
Sublicensee Product means any Licensed Product commercialized by Sublicensee, its affiliates or permitted sublicensees pursuant to the Collaboration and License Agreement, dated October 22, 2020, by and between Sublicensee and Licensee.
SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (Duchenne). Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solidâ€™s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.
IPSCIO Record ID: 193467
Criglerâ€“Najjar syndrome or CNS is a rare inherited disorder affecting the metabolism of bilirubin, a chemical formed from the breakdown of the heme in red blood cells.
Pompe disease is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially muscles, impairs their ability to function normally.
IPSCIO Record ID: 239657
Oculopharyngeal muscular dystrophy (OPMD) is a rare genetic muscle disorder with onset during adulthood most often between 40 and 60 years of age. OPMD is characterized by slowly progressive muscle disease (myopathy) affecting the muscles of the upper eyelids and the throat.