Royalty Report: Drugs, Disease, Therapeutic – Collection: 193467


Curated Royalty Rate Report
Category: Technology Licenses, Created On: 2022-04-28, Record Count: 7


This collection of transactions and supporting information was developed using our AI algorithm to curate similar royalty reports into a cohesive collection to support your licensing, transfer pricing or other transaction scenarios where documented royalty rates and/or deal terms are important.
Category: Technology Licenses
Created On: 2022-04-28
Record Count: 7

Primary Industries

  • Drugs
  • Disease
  • Therapeutic
  • Cellular
  • Enzymes
  • Proteins
  • Cryogenic
  • Diagnostic

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: 193467

License Grant
The Company entered into a services and collaboration agreement with an undisclosed service provider for the treatment of Crigler-Najjar Syndrome related to the Company’s AT342 development program.
License Property
The program is focused on developing and commercializing gene therapy products for patients suffering from serious, life-threatening rare diseases caused by single gene defects.  The gene therapy has powerful potential to treat these diseases through delivery of a functional copy of the affected gene to cells, resulting in production of the normal protein. We have built a compelling portfolio of product candidates, including AT132 for the treatment of X-Linked Myotubular Myopathy, or XLMTM, AT342 for the treatment of Crigler-Najjar Syndrome, or Crigler-Najjar, AT982 for the treatment of Pompe disease and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia, or CASQ2-CPVT.
Field of Use
This agreement pertains to the drug industry relating to the treatment of Crigler-Najjar Syndrome.

Crigler–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: 211012

License Grant
The Israeli Licensor has exclusively licensed PRX-102 to the Italian Licensee for all markets outside of the United States.
License Property
The Licensor will continue to be the manufacturer of PRX-102 for clinical development purposes and commercial purposes after marketing approvals.  PRX-102 is for the treatment of Fabry disease – a rare genetic disorder caused by a defective gene resulting in a deficient quantity of the enzyme alpha-galactosidase A. This enzyme is necessary for the daily breakdown of a lipid in the body called globotriaosylceramide.

Pegunigalsidase alfa, or PRX-102, is the Israeli Licensors chemically modified version of the recombinant protein alpha-Galactosidase-A protein that is currently being evaluated in phase III clinical trials for the treatment of Fabry disease. The Licensor is focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system.

Field of Use
The Italian Licensee will research, develop and market the innovative drug for the treatment of Fabry disease.

Fabry disease is an inherited disorder that results from the buildup of a particular type of fat, called globotriaosylceramide, in the body's cells. Beginning in childhood, this buildup causes signs and symptoms that affect many parts of the body.

IPSCIO Record ID: 263765

License Grant
The Licensors of Spain grant to the Licensee of the Netherlands an exclusive right and license, with the right to grant sublicenses, under Licensors interest, right and title in the Joint Patent Rights to use, develop, make, have made and Commercialize Products within the Territory; and,  a non-exclusive, right and license, with the right to grant sublicenses, under the Licensor Background IP required for the use, development, manufacture and/or Commercialization of the Product within the Territory and in the Gene Therapy Field and only to the extent required for said purpose.
License Property
The licensed properties relate to therapies based on constructs including adeno-associated virus (AAV) vectors.

The joint patent rights are for Porphobilinogen deaminase gene therapy.

Field of Use
With this agreement, the Parties modify their collaboration under aforementioned agreements and to refocus their efforts and resources to the further development and commercialization of a gene therapy treatment for acute intermittent porphyria.

The Field of use is Gene Therapy Field for the Disorder of acute intermittent porphyria.  Acute intermittent porphyria (AIP) is a rare autosomal dominant metabolic disorder affecting the production of heme resulting from a deficiency of the porphobilinogen deaminase. It is the most common of the acute porphyrias.  Porphyria refers to a group of disorders that result from a buildup of natural chemicals that produce porphyrin in your body. Porphyrins are essential for the function of hemoglobin — a protein in your red blood cells that links to porphyrin, binds iron, and carries oxygen to your organs and tissues.

IPSCIO Record ID: 258420

License Grant
The parties entered into a collaboration to develop and commercialize gene therapy treatments for Inherited Retinal diseases (IRDs).  The Licensee will receive worldwide exclusive rights to commercialize product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, X-linked retinitis pigmentosa (XLRP) and options to additional IRD programs.
License Property
Under the agreement, the two companies will collaborate in the clinical development of Licensors leading IRD pipeline, including product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3 and X-linked retinitis pigmentosa (XLRP). In addition, the parties are entering into a research collaboration covering Licensor’s pipeline of pre-clinical programs for IRDs. The two companies are also entering into a research collaboration to further develop AAV manufacturing technology as well as clinical and commercial manufacturing supply agreements for the clinical and research programs.

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.

Field of Use
Achromatopsia (ACHM) is an inherited retinal disease that prevents cone photoreceptors from functioning. Patients are legally blind from birth and usually suffer from severely reduced visual acuity of 20/200 or worse; a disabling sensitivity to light, or photophobia; total color blindness; and involuntary back and forth eye movements, or nystagmus. There are currently no approved treatments for achromatopsia.

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: 239657

License Grant
License grants the Licensee  exclusive, worldwide rights to AXO-AAV-OPMD utilizing proprietary Silence-and-Replace technology, which suppresses mutant protein production while restoring expression of functional protein.   License also grants rights to five additional investigational gene therapy products for neurological conditions.
License Property
An investigational Silence-and-Replace gene therapy program for the treatment of oculopharyngeal muscular dystrophy.  The Silence-and-Replace gene therapy technology is designed to deliver a combination of DNA-directed RNA interference (silence) along with a functional copy of the gene (replace) in a single vector construct.
Field of Use
The AXO-AAV-OPMD Program is an investigational gene therapy being developed as a one-time treatment for oculopharyngeal muscular dystrophy.

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.

IPSCIO Record ID: 67284

License Grant
The company entered into a global collaboration agreement focused on the discovery and worldwide development and commercialization of potentiator and corrector molecules in a potential triple combination therapy for the treatment of CF (Cystic Fibrosis).
License Property
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects approximately 80,000 patients worldwide and approximately 30,000 patients in the United States.  CF is a chronic disease that affects the lungs and digestive system.  CF patients, with significantly impaired quality of life, have an average lifespan approximately 50% shorter than the population average, with the median age of death at 40.  There currently is no cure for CF.  CF patients require lifelong treatment with multiple daily medications, frequent hospitalizations and ultimately lung transplant, which is life-extending but not curative.  CF is caused by a mutation in the gene for the CFTR protein, which results in abnormal transport of chloride across cell membranes.  Transport of chloride is required for effective hydration of epithelial surfaces in many organs of the body.  Normal CFTR channel moves chloride ions to outside of the cell.  Mutant CFTR channel does not move chloride ions, causing sticky mucous to build up on the outside of the cell.  CFTR dysfunction results in dehydration of dependent epithelial surfaces, leading to damage of the affected tissues and subsequent disease, such as lung disease, malabsorption in the intestinal tract and pancreatic insufficiency.
Field of Use
This agreement pertains to the drug industry for the treatment of cystic fibrosis.

IPSCIO Record ID: 203251

License Grant
The University Licensor grants an exclusive, worldwide right and license and the right to sublicense third parties, to make, have made, use, and sell Product under the Licensed Patents; and to use the Technical Information provided by Licensor.
License Property
The product relates to certain clinical and preclinical data, information and patent rights relating to the production and use of recombinant x-galactosidase A.
Field of Use
The license is for the treatment of Fabry Disease.

Fabry disease is a rare genetic lysosomal storage disease, inherited in an X-linked manner.  Fabry disease is an inherited disorder that results from the buildup of a particular type of fat, called globotriaosylceramide, in the body's cells.

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