Royalty Report: Drugs, Disease, Therapeutic – Collection: 190271


Curated Royalty Rate Report
Category: Technology Licenses, Created On: 2022-04-28, Record Count: 4


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: 4

Primary Industries

  • Drugs
  • Disease
  • Therapeutic

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: 190271

License Grant
Licensor granted an exclusive worldwide option and collaboration agreement under which both companies will develop and commercialize the antisense investigational drug candidate, SPINRAZA, for the treatment of spinal muscular atrophy or SMA.  Licensee exercised the option to develop and commercialize SPINRAZA.
License Property
SPINRAZA is an antisense oligonucleotide. Antisense drugs are small snippets of synthetic genetic material that bind to ribonucleic acid (RNA), so they can be used to fix splicing errors in genes such as SMN2.

SPINRAZA is approved by the U.S. Food and Drug Administration for the treatment of SMA in pediatric and adult patients in the U.S.

Spinal muscular atrophy (SMA) is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement.

Field of Use
SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.

Licensee is a biopharmaceutical company focused on discovering, developing, manufacturing and delivering therapies to people living with serious neurological and neurodegenerative diseases.

IPSCIO Record ID: 295927

License Grant
Licensor granted Licensee worldwide exclusive licenses, with the right to grant sublicenses, to our patent rights and know-how with respect to such compounds and products. Licensee is responsible for pursuing worldwide clinical development of compounds from the research program and has the exclusive right to develop and commercialize compounds from the collaboration  to further develop and commercialize compounds identified under our Foundation sponsored research program with the Foundation and to research other small molecule compounds with potential for therapeutic use in patients with Foundation.
License Property
Licensor developed several high-throughput drug discovery technology platforms that enable us to identify small molecule modifiers of pre-mRNA splicing. These technologies rely on sensitive quantification of pre-mRNA isoforms directly in human cells or tissue samples. Using this technology, we have successfully identified orally bioavailable small molecules that correct splicing of SMN2 mRNA. One of these molecules, risdiplam, is a potential treatment for the genetic disorder SMA.
Field of Use
The small molecule compounds with the potential for therapeutic use in patients with Spinal Muscular Atrophy (SMA).

Spinal muscular atrophy is a genetic disorder characterized by weakness and wasting (atrophy?) in muscles used for movement (skeletal muscles). It is caused by a loss of specialized nerve cells, called motor neurons that control muscle movement. The weakness tends to be more severe in the muscles that are close to the center of the body (proximal) compared to muscles away from the body's center (distal). The muscle weakness usually worsens with age.

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: 198450

License Grant
The Netherland Licensor parties granted the Licensee a royalty-bearing, worldwide license under patent rights and know-how with respect to the  Duchenne muscular dystrophy program, which are potentially necessary or useful for the treatment of DMD,  in all fields of use and for all purposes, including to develop and commercialize antisense oligonucleotide products that target one or more exons of the dystrophin gene to induce exon skipping, including eteplirsen.
License Property
IP includes the disease-modifying pipeline of exon-skipping drug candidates targeting Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness.
Field of Use
The Licensee is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases.
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