Royalty Report: Drugs, Genome, Disease – Collection: 260420

$150.00

Curated Royalty Rate Report
Category: Technology Licenses, Created On: 2022-04-28, Record Count: 20

Description

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: 20

Primary Industries

  • Drugs
  • Genome
  • Disease
  • Therapeutic
  • cardiac
  • Delivery
  • Cancer
  • Drug Discovery
  • Biotechnology
  • DNA
  • Proteins
  • Diabetes Treatment
  • cell therapy
  • Biological Materials

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: 260420

License Grant
Licensor grants to the Licensee of the Netherlands a non-exclusive, non-sublicensable license under the Licensed Patent Rights in each country of the world where there are Valid Claims of Licensed Patent Rights, to make, have made, develop, use, sell, offer to sell and import Licensed Products.
License Property
Licensor has patent rights related to an AAVl Vector gene delivery system.  AAV1 Vector means the adeno-associated virus serotype 1 vector technology which includes without limitation the AAV serotype 1 rep, cap, and ITR sequences and proteins whether utilized in whole or in part to deliver therapeutic genes into cells.
Field of Use
Field means treatment of Lipoprotein Lipase, or LPL, deficiency type 1 and LPL deficiency type 5 by in vivo gene therapy utilizing an AAV 1 Vector encoding the LPL gene.

AMT intends to use the vector for developing therapeutics for treating type I and type V lipoprotein lipase (LPL) deficiency. In particular, AMT will use the vector in the development of its lead gene product, AMT-011, which is in Phase II clinical trials.

Lipoprotein lipase deficiency is a genetic disorder in which a person has a defective gene for lipoprotein lipase, which leads to very high triglycerides, which in turn causes stomach pain and deposits of fat under the skin, and which can lead to problems with the pancreas and liver, which in turn can lead to diabetes.

IPSCIO Record ID: 262744

License Grant
In the original agreement Licensor granted to the Licensee, of the Netherlands, a non-exclusive sublicense under the Licensed Patent Rights pursuant to the License Agreement, which does not permit further downstream sublicensing.

This amendment adds sublicensing rights.  Licensor grants Licensee of the Neterlands a non-exclusive license under the Licensed Patent Rights in each country of the world where there are Valid Claims of Licensed Patent Rights, to make, develop, use, sell offer to sell, and import Licensed Products, with Restricted Sublicensing Rights.   Restricted Sublicensing Rights means a non-exclusive sublicense under the Licensed Patent Rights from Licensee to sublicensees of Licensee, including Chiesi, pursuant to the sublicensing conditions contained in this Agreement.

License Property
Licensor has exclusively licensed the Licensed Patent Rights relating to an AAVI Vector gene delivery system from a University.

AAV1 Vector means the adeno-associated virus serotype 1 vector technology which includes without limitation the AAV serotype 1 rep, cap, and ITR sequences and proteins whether utilized in whole or in part to deliver therapeutic genes into cells.

Field of Use
Licensee has developed and is commercializing an AAVI product to treat LPL type 1 and LPL type 5 deficiencies.

The original agreement field means treatment of Lipoprotein Lipase, or LPL, deficiency type 1 and LPL deficiency type 5 by in vivo gene therapy utilizing an AAV 1 Vector encoding the LPL gene.

Lipoprotein lipase deficiency is a genetic disorder in which a person has a defective gene for lipoprotein lipase, which leads to very high triglycerides, which in turn causes stomach pain and deposits of fat under the skin, and which can lead to problems with the pancreas and liver, which in turn can lead to diabetes..

IPSCIO Record ID: 245877

License Grant
The Parties,  both of the Netherlands,  agree that, with effect from the Effective Date, all of the Original Agreements shall terminate.

Licensor transfers and assigns its right, title or interest in the Existing Patent Rights to Licensee.

License Property
Licensee is a leading medical centre in the Netherlands which, amongst other activities, conducts research into the therapy, prophylaxis and diagnosis of various diseases and other conditions.

AAV Vector is an adeno-associated virus vector composed of a transgene cassette with limited length,  approximately 4.5 kb, flanked by an ITR DNA sequence.

FG Products are LPL Products, IL-10 Products and Nash Products.

IL-10 Product is a gene therapy product for the treatment of diseases and conditions caused by the deficiency or mutation of the IL-10 gene.

LPL Product is a gene therapy product for the treatment of diseases and conditions caused by lipoprotein lipase deficiency or mutation.

Nash Product is a gene therapy product for the treatment of non-alcoholic steatotic hepatitis.

The existing patent rights are for LPL variant therapeutics, and, IL-10 gene transfer to peripheral mononuclear cells, and, New method of treatment for non alcoholic steatotic hepatitis (NASH).

Field of Use
The field of use is as a gene therapy product for the treatment of non-alcoholic steatotic hepatitis.

IPSCIO Record ID: 260419

License Grant
The Licensor of France grants to the Licensee of the Netherlands an exclusive license right under the Patents to develop, use, make, have made, sell or offer to sell Product in the Field of Use and in the Territory.
License Property
The patents are relating to the preparation and use of recombinant viruses in gene therapy.

The Product shall mean a gene therapy product containing a recombinant virus with a nucleic acid coding for a lipoprotein lipase (LPL) developed, used and/or commercialized by or for Licensee or its affiliates where the activity in relation to such product occurs in one of the countries of the Territory and is covered in whole or in part by a Valid Claim within the Patents in that country.

Field of Use
The Field of Use shall mean indications of Lipoprotein Lipase (LPL) deficiency also referred to as Type I or V hyperlipoproteinernia.

Lipoprotein lipase deficiency (LPLD) is a rare monogenic disorder of triglyceride metabolism. Deficiency of the enzyme lipoprotein lipase results in pronounced accumulation of triglycerides in the plasma. Specifically, the clearance of chylomicrons is impaired. Chylomicrons, fatty droplets that transport fat in the form of triglyceride, appear in the bloodstream shortly after the ingestion of dietary fat. Chylomicrons are normally cleared from the body after a period of fasting. Lipoprotein lipase recognizes a protein in chylomicrons called apolipoprotein C2, and is then activated to metabolize and clear out triglycerides. However, deficiency or lack of lipoprotein lipase results in the accumulation of chylomicrons and triglycerides in the body. High triglyceride levels are present from infancy and childhood, and various associated symptoms may occur. Some patients remain undiagnosed until adulthood. LPLD is caused by mutations in the LPL gene and is inherited in an autosomal recessive manner.

IPSCIO Record ID: 261354

License Grant
This amendment between the French Licensor and the Licensee of the Netherlands will modify the financial terms associated with royalty payments and create reporting obligations regarding pricing and reimbursement of Licensees gene therapy product Glybera® and its development milestones, and add a Right of First Negotiation for Licensor.  The original agreement was exclusive.
License Property
The original agreement identifies the patents are relating to the preparation and use of recombinant viruses in gene therapy.

The Product shall mean a gene therapy product containing a recombinant virus with a nucleic acid coding for a lipoprotein lipase (LPL) developed, used and/or commercialized by or for Licensee or its affiliates where the activity in relation to such product occurs in one of the countries of the Territory and is covered in whole or in part by a Valid Claim within the Patents in that country.

Field of Use
The Field of Use shall mean indications of Lipoprotein Lipase (LPL) deficiency also referred to as Type I or V hyperlipoproteinernia.

Lipoprotein lipase deficiency (LPLD) is a rare monogenic disorder of triglyceride metabolism. Deficiency of the enzyme lipoprotein lipase results in pronounced accumulation of triglycerides in the plasma. Specifically, the clearance of chylomicrons is impaired. Chylomicrons, fatty droplets that transport fat in the form of triglyceride, appear in the bloodstream shortly after the ingestion of dietary fat. Chylomicrons are normally cleared from the body after a period of fasting. Lipoprotein lipase recognizes a protein in chylomicrons called apolipoprotein C2, and is then activated to metabolize and clear out triglycerides. However, deficiency or lack of lipoprotein lipase results in the accumulation of chylomicrons and triglycerides in the body. High triglyceride levels are present from infancy and childhood, and various associated symptoms may occur. Some patients remain undiagnosed until adulthood. LPLD is caused by mutations in the LPL gene and is inherited in an autosomal recessive manner.

Glybera is used to treat adults with lipoprotein lipase deficiency who have severe or multiple attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet.  This is a type of medicine that works by delivering genes into the body.

IPSCIO Record ID: 248329

License Grant
The Canadian Licensor hereby grants to Licensee of Netherlands within the Field of Use, and Licensee accepts, the exclusive worldwide right, sublicense and privilege under the Licensor Licensed Rights to use the Licensor Licensed Technology and to use, manufacture, distribute and sell Licensed Products.  Licensee shall sponsor the research Project as defined in the Collaborative Research Agreement between Licensor and University and, for that purpose, Licensee and Licensor entered into Heads of Agreement effective August 1, 2000.
License Property
Project means the project description entitled “LPL Gene Therapy for LPL Deficiency”, which is attached to the Collaborative Research Agreement, including any amendments thereto which the Parties and University may mutually agree to, from time to time, of the Collaborative Research Agreement.

The scientific collaboration is in the area of LPL gene therapy in humans including all research and development required for establishing a successful clinical LPL gene therapy protocol.

Description of Technology
U.S. 5,658,729 – Lipolipase Mutation 291, Implication for Coronary Artery Disease
U.S.  SN 08/737,954 – Recombinant Viruses Preparation and use thereof in Gene Therapy

Field of Use
Field of Use means gene therapy being the treatment of a disease caused by the malfunction of a gene, by transduction of cells of the organism with the normal gene or genetically altered gene.

IPSCIO Record ID: 192627

License Grant
Licensor, government organization, hereby grants and Licensee of the Netherlands accepts a nonexclusive license under the Licensed Patent Rights in the Licensed Territory to make and have made, to use and have used, to sell and have sold, to offer to sell, and to import any Licensed Products or New Products in the Licensed Fields of Use, to practice and have practiced any Licensed Processes in the Licensed Fields of Use, to make, have made, to use and have used but not to sell any Supplied Materials.  As used in this Agreement, “have made” and “have used” means that Licensee shall have the limited right to use a third party contract manufacturer to make and use only (but not to sell) Supplied Materials, Licensed Products or New Products.  Licensee acknowledges and agrees that any such third party contract manufacturer shall be bound to the terms and obligations of this Agreement.
License Property
Licensed Patent Rights shall mean
(a)  Patent applications (including provisional patent applications and PCT patent applications) or patents listed, all divisions and continuations of these applications, all patents issuing from these applications, divisions, and continuations, and any reissues, reexaminations, and extensions of all these patents;
(b)  to the extent that the following contain one or more claims directed to the invention or inventions disclosed in 2.6(a)
     (i)    continuations-in-part of 2.6(a);
     (ii)   all divisions and continuations of these continuations-in-part;
     (iii)  all patents issuing from these continuations-in-part, divisions, and continuations;
     (iv)  priority patent application(s) of 2.6(a); and
     (v)   any reissues, reexaminations, and extensions of all these patents;
(c) to the extent that the following contain one or more claims directed to the invention or inventions disclosed in 2.6(a) all counterpart foreign and U.S. patent applications and patents to 2.6(a) and 2.6(b), including those listed in Appendix A; and
(d) Licensed Patent Rights shall not include 2.6(b) or 2.6(c) to the extent that they contain one or more claims directed to new matter which is not the subject matter disclosed in 2.6(a).

U.S. Patent Application(s) or Patent(s)
(a)  U.S. Patent Application Serial No. 09/986,618, filed November 9, 2001, now abandoned, entitled “Production of Adeno-Associated Virus in Insect Cells” [HHS Ref. No. E-325-2001/0-US-01];
(b)  U.S. Patent No. 6,723,551, issued April 20, 2004, entitled “Production of Adeno-Associated Virus in Insect Cells” [HHS Ref. No. E-325-2001/1-US-01]; and
(c)  U.S. Patent Application Serial No. 10/415,834, filed May 2, 2003, entitled “Production of Adeno-Associated Virus in Insect Cells” [HHS Ref. No. E-325-2001/2-US-02].

Licensed Products means (a) Supplied Materials and (b) tangible materials, which in the course of manufacture, use, sale, or importation, would be within the scope of one or more claims of the Licensed Patent Rights that have not been held unpatentable, invalid or unenforceable by an unappealed or unappealable judgment of a court of competent jurisdiction.

New Product means a product made using a Licensed Process but excluding Licensed Products.

Licensed Processes means processes, which in the course of being practiced, would be within the scope of one or more claims of the Licensed Patent Rights that have not been held unpatentable, invalid or unenforceable by an unappealed or unappealable judgment of a court of competent jurisdiction.

The Disease Lipoprotein Lipase Deficiency
Genetic lipoprotein lipase (LPL) deficiency results in profound hypertriglyceridemia, which is associated with intense chronic abdominal pain, hepatosplenomegaly, eruptive xanthomas, lipemia retinalis, dyspnea, mono- or polyparesthesias, and memory loss. Prolonged elevations in plasma triglycerides (TG) also induce recurrent episodes of often lethal pancreatitis, chronic pancreatic insufficiency, and diabetes mellitus. Currently, no effective treatment for this disease exists. Patients must follow a strict low-fat diet. However, TG levels often remain above the critical threshold. Genetic LPL deficiency type I is a rare, autosomal recessive trait. Prevalence varies between 1 in 1,000,000 in the general population to 1 in 5,000 in French Quebec (a ‘founder effect’).

Acute intermittent porphyria (AlP) is an autosomal dominant inherited condition caused by mutations in the porphobilinogen deaminase (PBGD) gene. The PBGD gene is located on chromosome 11 q24.1 -24.2 and spread over fifteen exons. The protein encoded by this gene is a rate-limiting enzyme, the PBGD enzyme, in the haem synthetic pathway.

Parkinson’s disease (PD) is a progressive neurodegenerative disease, resulting in tremors, stiffness, slowness of movement, and lack of coordination. Patients are faced with a severely debilitating disease and a serious loss in quality of life. PD is caused by degeneration and death of nerve cells in a specific part of the brain known as the substantia nigra. These cells produce dopamine, a substance necessary for communication between nerve cells involved in the coordination of movement.

Field of Use
This agreement pertains to the drug industry relating to the use of the Licensed Patent Rights for the commercial development of AAV related products within the scope of the Agreement.

Licensee is building gene therapies using adeno-associated viral (AAV)-based vectors.

IPSCIO Record ID: 291138

License Grant
University hereby grants to Licensee and the Covered Affiliates for the term of this Agreement the worldwide right and license, with the right to grant sublicenses, to develop, have developed, make, have made, use, have used, import, have imported, sell, offer for sale and have sold University Licensed Products under the Group 1 Patents in all fields of use. Such right and license shall be non-exclusive to Licensee.

Group 2 License Grant. University hereby grants to Licensee and the Covered Affiliates for the term of this Agreement the worldwide right and license, with the right to grant sublicenses, to develop, have developed, make, have made, use, have used, import, have imported, sell, offer for sale and have sold University Licensed Products under the Group 2 Patents in all fields of use. Such right and license shall be non-exclusive to Licensee in the LSD Field.  Subject to this Agreement, such right and license shall be exclusive to Licensee outside the LSD Field.

University hereby grants to Licensee and the Covered Affiliates for the term of this Agreement the worldwide right and license, with the right to grant sublicenses, to develop, have developed, make, have made, use, have used, import, have imported, sell, offer for sale and have sold University Licensed Products under the Group 3 Patents in in all fields of use.   Subject to this Agreement, such rights and license shall be exclusive to Licensee.

License Property
Group 1 Patents means the following patents
US 6,475,769 – Methods and cell line useful for production of recombinant adeno-associated viruses
US 7,238,526 – Methods and cell line useful for production of recombinant adeno-associated viruses

Group 2 Patents means the following patents
US 6,759,237 – Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US 7,186,552 – Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same

Group 3 Patents means
US 7,056,502 – Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids

University Licensed Product(s) means products that in the absence of this Agreement would, where and when made, used, sold, or imported, infringe at least one issued claim or pending claim of University Patent Rights; and products that are made using a process or machine that in the absence of this Agreement would, where and when used, infringe at least one issued claim or pending claim of University Patent Rights. All University Licensed Products intended to deliver the same Gene shall be considered a single University Licensed Product for purposes of this Agreement.

University Patent Rights means those patents and patent applications listed in this Agreement and all foreign counterparts thereof, as well as continuation, continuation-in-part, provided that such continuation-in-part relates directly to existing patents or patent applications and not to any new matter, divisional and re-issue applications
thereof, together with any and all patents issuing thereupon or upon any foreign counterparts thereof; provided, however, that Licensee acknowledge that the invention relating to non-invasive delivery of transgenes into the bloodstream, which is disclosed in Example 6 of International Patent Application No. PCT/USO1/13000 (M2304PCT), and in a US CIP application No. 09/955,444 (N2605), was made at University following termination of the research under the Sponsored Research Agreement.  Licensee hereby acknowledge that they have no rights in this invention relating to non-invasive delivery of transgenes into the bloodstream, as defined in these applications and divisionals, continuations, re-examinations, re-issues, and any foreign counterparts thereof, and any patents issuing therefrom. Licensee agree that they will not make or pursue any claims that this invention as so defined is or should have been part of the University Patent Rights.

Gene means any nucleotide sequence that is capable of encoding, or of causing or modulating the expression of, a proteiin, protein fragment, or other genetic element.  For the purposes hereof, a Gene having 90% homology to that of another Gene, and which expresses essentially the same protein for at least one biological function, shall be considered the same Gene as such other Gene.

LSD Field means the prevention, treatment, or cure of lysosomal storage diseases whether by in vivo or ex vivo means (together with preparation, research, development, and attempts to do the foregoing).

Cancer Field means the prevention, treatment or cure of malignancies whether by in vivo or ex vivo means together with preparation, research, development, and attempts to do the foregoing).

TNFr/Inflammatory Field means the prevention, treatment, or cure of any disease or diseases in whole or in part through use or delivery of the TNFr Gene or any other Gene acting in the Inflammatory Pathway, whether by in vivo or ex vivo means (together with preparation, research, development, and attempts to do the foregoing).

Field of Use
This agreement pertains to the drug industry relating to Gene Therapy.

IPSCIO Record ID: 127805

License Grant
The English Licensor grants a non-exclusive license to make, use, sell, offer for sale or import Licensed Products under the Licensed Patents, with a right to grant sublicenses.
License Property
The certain patents relate to the Factor IX gene.

The licensed products mean viral vector products, including without limitation adenovirus (AV) and adeno-associated virus (AAV) viral vector products, for in vivo human gene therapy.  Specifically excluded are utilization of non-viral vector products, any ex vivo approach based on the administration of living cells, Factor IX protein produced by or from transgenic animals, and any license rights to make, use, offer for sale, sell, or import Factor IX protein.

Field of Use
This agreement is for the human gene therapy industry.

IPSCIO Record ID: 260409

License Grant
Licensor grants a non-exclusive, worldwide license, including the right to grant sublicenses through multiple tiers of sublicense, under the Patent Rights solely to develop, make, have made, use, offer for sale, sell, have sold, import, have imported, export and have exported Products in the Field of Use.
License Property
The patent relates to genetically-engineered parvovirus capsids and viruses designed to introduce a heterologous gene into a target cell.
Field of Use
The Field of Use shall mean the production and delivery of adeno-associate viral vector (AAV) Serotype 1 to deliver SERCA 2a for the treatment of heart failure and arrhythmias.

IPSCIO Record ID: 294014

License Grant
Licensor, a medical research centers, grants an exclusive license under the Licensed Patent Rights in the Licensed Territory to make and have made, to use and have used, to sell and have sold, to offer to sell, and to import any Licensed Products in the Licensed Fields of Use and to practice and have practiced any Licensed Processes in the Licensed Fields of Use.
License Property
The patents and patent applications relate to Modified Adena-Associated Virus Vector Capable of Expression from a Novel Promoter.

Adeno-associated viruses are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae.

Field of Use
The field of use is the development of compositions and methods utilizing Adeno-Associated Viral Vectors embodied in the Licensed Patent Rights which are useful in the treatment and prophylaxis of human and animal diseases, and does not include compositions and methods for the treatment and prophylaxis of cystic fibrosis.

Cystic fibrosis is a hereditary disease that affects the lungs and digestive system. The body produces thick and sticky mucus that can clog the lungs and obstruct the pancreas. Cystic fibrosis (CF) can be life-threatening, and people with the condition tend to have a shorter-than-normal life span.

IPSCIO Record ID: 352696

License Grant
Licensor is willing to grant to Licensee, a non-exclusive research license to conduct certain research to identify and select Specified Vectors for specified indications and an option to obtain a non-exclusive license to research, develop, and commercialize Licensed Products for specified indications.

For the Research License Grant, including the Retained Rights, during the Research Term, Licensor grants to Licensee a non-exclusive, non-transferable, worldwide license under the Licensed Research Patents to make, have made, and use any and all AAV Materials in the Research Field, including, for the avoidance of doubt, the right to conduct research and pre-clinical development, solely for purposes of identifying and selecting Specified Vector(s) for use in the Commercial Field upon exercise of a Commercial Option.  For the avoidance of doubt, the foregoing license does not include the right to sell, offer for sale, or import any AAV Materials.

For the Commercial License Option. Licensor grants to Licensee the option, exercisable at Licensees sole discretion, to obtain a non-exclusive worldwide license with respect to each of the Disease Indications and a single Specified Vector for such Disease Indication.

For the License Grant Upon Exercise, if Licensee exercises the Commercial Option for a
particular Disease Indication, effective upon both Licensors receipt of the notice and in the case of a Secondary Disease Indication, the fee described for such Secondary Disease Indication, including the Retained Rights. Licensor grants, to Licensee non-exclusive, sublicensable, non-transferable, worldwide license under the applicable Licensed Commercial Patents to make. have made, use, import, sell, and offer for sale Licensed Products using the Specified Vector solely in the Commercial Field for such Disease Indication, including, for the avoidance of doubt, the right to conduct research and development.

License Property
Licensor has rights under certain patents pertaining to various recombinant adeno-associated virus vectors.

AAVrh10 means the recombinant adeno-associated virus serotype rh10 vector with the specified sequence set forth in GenBank {protein id AAO88201) and (b) any recombinant
adeno-associated virus derivatives of such serotype rh10 vector that arc covered by the claims of the Licensed Research Patents.

Disease lndications means one or more of the following indications Friedreichs Ataxia that is treated or prevented by administration of the applicable recombinant adeno-associated virus serotype vector directly to the central nervous system, brain and spinal cord, Friedreichs Ataxia CNS, Friedreichs Ataxia that is treated or prevented by administration of the applicable recombinant adeno-associated virus serotype vector by any route except administration directly to the central nervous system, brain and spinal cord, Friedrcichs Ataxia Systemic, Huntingtons Disease, and  Amyotrophic Lateral Sclerosis.

Licensed Product means any product using the applicable Specified Vector capsid protein that is made, made for, used, sold, offered for sale, or imported by Licensee, its Affiliates, and any of its or their Sublicensees, the manufacture, use, sale. offer for sale, or import of which product, in the absence of the license granted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim of the Licensed Commercial Patents in the country of manufacture, use, sale, offer for sale, or import; or any service sold by Licensee.

Field of Use
The license agreement for use of Licensor’s proprietary NAV® vectors for the development and commercialization of gene therapies to treat Amyotrophic Lateral Sclerosis (ALS), Friedreich’s ataxia (FA) and Huntington’s disease (HD).

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive, fatal neurodegenerative disease that leads to muscle weakness, loss of mobility, impaired speech, and difficulty breathing and swallowing.

Friedreich's ataxia (FA) is the most common hereditary ataxia, with approximately 8,000 patients living with the disease in the United States and Europe.  FA patients have a genetic mutation in the FXN gene, which limits the production of the protein frataxin, causing a variety of debilitating symptoms and complications, loss of coordination and balance, muscle weakness, impaired vision, hearing and speech, scoliosis, diabetes, and cardiomyopathy.

Huntington’s disease (HD) is an inherited neurodegenerative disorder where symptoms typically become noticeable between 30 and 50 years of age.  HD is caused by a genetic mutation in the huntingtin gene, which leads to the production of a mutated huntingtin protein, resulting in symptoms such as chorea, rigidity, abnormal posturing, cognitive impairment and psychiatric symptoms, and difficulty with speech and swallowing.

IPSCIO Record ID: 352695

License Grant
University grants an exclusive, worldwide license in the Patent Rights to make, have made, use, offer for sale, sell, have sold and import Licensed Products in the Field.
License Property
The patents include
–  Allele-specific RNA Interference;
–  In Vivo Production of Small Interfering RN As that Mediate Gene Silencing;
–  RNA Interference for the Treatment of Gain-of-Function Disorders;
–  Novel AAVs and Uses Thereof;
–  CNS Targeting AAV Vectors and Methods of Use Thereof;
–  Methods and Compositions for Controlling Efficacy of RNA Silencing;
–  Methods and Compositions for Enhancing the Efficacy and Specificity of Single and Double Blunt-Ended siRNA; and,
–  Methods and Compositions for Enhancing the Efficacy and Specificity of RNAi.
Field of Use
The Field of use is the treatment of human diseases using gene therapy applications.  Any commercial sale of research reagents covered by the Patent Rights is specifically excluded
from the Field.

The gene therapy approach uses AAV (adeno-associated virus) vectors, which are modified, non-replicating versions of AAV, and which we believe are ideal vectors for CNS gene therapy.

The Licensee expects to utilize established and novel techniques for dosing and delivery of our AAV gene therapies to the CNS.

IPSCIO Record ID: 203266

License Grant
Licensor grants, during the Term, an exclusive license, under the Technology to Licensee Products in the Field.
License Property
This technology includes, without limitation, MYDICAR® (AAV1/SERCA2a).

Mydicar is a genetically targeted enzyme replacement therapy being studied for use in patients with severe heart failure.  It is designed to increase the level of SERCA2a, a sarcoplasmic endoplasmic reticulum calcium (Ca2+) ATPase found in the membrane of the sarcoplasmic reticulum (SR). The SERCA2a gene is delivered to the heart via an adeno-associated viral vector.

Field of Use
The field is human therapeutics and prophylactics.

IPSCIO Record ID: 211877

License Grant
Buyer grants to Seller a non-exclusive, perpetual, irrevocable, worldwide license under the Transferred Manufacturing Technology, Seller Manufacturing Patent Rights, Johnson Patent Rights, Transferred Platform Technology, Seller Platform Patent Rights and Exclusively Licensed Materials to develop, make, have made, use, sell offer for sale and import AAV Vectors, and products containing AAV Vectors, solely within the field.

In the asset purchase, the Seller agrees to sell, convey, transfer and assign to Buyer, and Buyer agrees to purchase from Seller, free and clear of any Encumbrances, all of Seller’s right, title and interest in and to the following properties, assets and rights
(a) the Transferred Technology;
(b) the Assigned Patent Rights;
(c) the Transferred Equipment;
(d) all of Seller’s rights under that certain Exclusive Sublicense Agreement; and
(e) all rights of Seller to Actions and rights of Seller to sue at law or in equity.

License Property
AAV Vector shall mean a recombinant adeno-associated virus gene vector composed of a viral capsid and a genome.

Excluded Technology shall mean (i) any composition that is specific to a particular AAV Vector serotype or pseudotype and any data and other Technology that is specific to such a composition; any composition, method, process or other Technology that is specific to an AAV Vector comprising a particular gene; any composition, method, process or other Technology that is specific to a particular gene; any final formulation buffer for clinical delivery of an AAV Vector comprising a particular gene and any data or other Technology that is specific to such buffer; and any analytical method specific to product performance of an AAV Vector comprising a particular gene.

Field of Use
This agreement is for solely within the following four fields
(iii) delivery of tgAAC94 as identified in Seller's regulatory filings as of the Closing Date which comprises the DNA sequence encoding the p75 TNFr receptor lg fusion protein, and
(iv) the delivery of RNAi using AA V Vectors for the revention or treatment of Hepatitis C.

IPSCIO Record ID: 260408

License Grant
Licensor, an individual, hereby grants an exclusive, even as to Licensor, worldwide license, with the right to sublicense through multiple tiers of sublicense, under Licensors interest in the Licensed Patents for any purpose in the Field, including, without limitation, to develop, make, have made, use, sell, offer for sale, have sold and import Products in the Field.
License Property
The patent relates to methods for transferring genes into the heart and blood vessels.
Field of Use
The Field shall mean the treatment or prevention of heart failure using products that act by the delivery of genes encoding proteins whose role is to regulate calcium uptake or release in the sarcoplasmic reticulum.

IPSCIO Record ID: 263767

License Grant
Licensor grants to Licensee of the Netherlands a worldwide, exclusive, even as to Licensor, license, with the right to grant Sublicenses, under the Licensed Patent Rights to Exploit GDNF Products in the Licensed Field, and a worldwide, exclusive, even as to Licensor, license, with the right to Sublicenses, under the Licensed Know-How to Exploit GDNF Products in the Licensed Field.
License Property
The licensed property is glial-cell derived neurotrophic factor or GDNF.  The GDNF gene contains the information to produce a protein necessary for the development and survival of nerve cells.

GDNF Product means any Gene Therapy product capable of delivering GDNF or the gene encoding GDNF, or in either case any fragment of GDNF that has Functional Activity.

Field of Use
The Licensed Field means all uses in Gene Therapy.   This agreement specifically mentions the treatment of neurological diseases and other diseases.  The Licensee also plans to combine the GDNF gene with its proprietary adeno-associated virus (AAV) technology to develop gene therapies for a range of CNS applications, such as Huntingtons disease and amyotrophic lateral sclerosis (ALS), with an aim to protect and enhance the function of the affected nerve cells.

Based on the promising early results of GDNF gene therapy product in Parkinson’s disease models, Licensee believe there is an opportunity for a similar approach in other debilitating CNS disorders.

IPSCIO Record ID: 293665

License Grant
University grants to Licensee, to the extent it may lawfully do so, the right and exclusive license in the Territory to make, have made, use and sell the Licensed Technology in the Field and to practice the Patent Rights in the Field for the Term.

University grants to Licensee, to the extent it may lawfully do so, the right and nonexclusive license in the Territory to practice the Know-How in the Field for the Term.

License Property
The patents are for Virally-mediated endogenous neogenesis of beta cells yields autoimmune- resistant beta cells, and, Endoegnous Neogenesis of beta cells.

The know-how is Viral infusion related information, including AAV serotypes solution and equipment.

Licensed Technology shall mean any product or component or part thereof which is Covered in whole or in part by a Valid Claim in the Patent Rights.

The diabetes gene therapy works by reprogramming beta cells in the pancreas to restore their function, thereby replenishing levels of insulin. The novel infusion process uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The proteins these genes express transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system.

61/983,295 VIRALLY-MEDIATED ENDOGENOUS NEOGENESIS OF BETA CELLS YIELDS AUTOIMMUNE-RESISTANT BETA CELLS

Field of Use
The Field shall mean diabetes therapy.

IPSCIO Record ID: 244658

License Grant
The Parties have an acquisition agreement.  Upon consummation of the Acquisition, the Parties desire to establish a new cooperative development program intended to discover, develop, and certify for use in humans one or more new pharmaceutical products.

Licensor grants an exclusive, worldwide license under the Ad Interferon-beta Product Licensor Patents and the Ad Interferon-beta Product Technology to develop, research, make, have made, use, have used, sell, offer for sale, import and have sold the Ad Interferon-beta Product in the Cancer Field and the Beta Interferon Field .

For the Manufacturing License,  Licensor will provide a transferable, limited license or subiicense as the case may be under the Licensor Technology and the Licensor Patents to manufacture such Clinical Vectors and Commercial Vectors to the extent necessary to enable Licensee to manufacture quantities of the Clinical Vectors and Commercial Vectors that otherwise would have been supplied by Licensor to Licensee under this Agreement, solely for use and application by Licensee for the uses and applications in the Territory that would be permitted under this Agreement with respect to such Clinical Vectors and Commercial Vectors purchased from Licensor.

Licensor grants a license, with right to grant sublicenses, under the Licensor Technology and the Licensor Patents to research, develop, manufacture, have manufactured, use, import, export, offer to sell or sell Products in the Territory.

Licensor grants  a nonexclusive, worldwide license, with right to sublicense to Contractors permitted under this Agreement,  to the Licensed Technology, other than the Applied Licensor Technology, as designated in the applicable Project Plan and Budget, and under any Licensor Patents, other than the Exhausted Licensed Patents and the Exempted Licensed Patents, as designated in the applicable Project Plan and Budget, which would, but for this license grant, be infringed by Licensees performance of its Development Tasks solely as necessary for performance of Licensees Development Tasks during and in the course of any approved Development Project.

In addition to the other rights granted under this Agreement, Licensor hereby grants an option to acquire a license, with the right to grant sublicenses, to Licensor Technology, that is not otherwise designated as Applied Licensor Technology in the applicable Project Plan and Budget, and under any and all Targeted Patents, that are not designated as Exhausted Licensor Patents or as Exempted Licensor Patents in the applicable Project Plan and Budget.

License Property
The patents include Adenovirus Vectors for Gene Therapy, and, .Method of Reducing an Immune Response to a Recombinant Adenovirus.

AAV Interferon-beta Product means a Product incorporating the Beta Interferon Gene and any AAV Vector as the delivery platform.

AAV Vector means an adeno-associated viral vector useful or potentially useful for the delivery of Genes to human cells.

Ad Interferon-beta Product means the Product incorporating the Beta Interferon Gene and an Ad Vector that is the subject of the Ad Interferon-beta Project.

Ad Vector means an adenoviral vector useful or potentially useful for the delivery of Genes to human cells.

Field of Use
Beta Interferon Field means the treatment or prevention in humans of one or more diseases through the use or application of one or more gene therapy vectors to deliver Genes; such Genes being defined herein as the Beta Interferon Genes.

Cancer Field means the treatment or prevention in humans of one or more cancers through the use or application of AAV Vectors or Ad Vectors.

IPSCIO Record ID: 204300

License Grant
The Licensor, a university foundation, granted the Licensee  a license to a U.S. patent with claims covering the use of HSV helpers to produce AAV vectors in gene therapy. The patent is expected to expire in 2025.  The Licensee has the right to sublicense our rights under this agreement.
License Property
AAV vectors can be used to introduce functional genes into many different cell types by a variety of delivery methods and can carry genes of up to 4,000 base pairs in length, a payload capacity sufficient to accommodate more than 90% of the individual genes in the human genome.
Field of Use
The Licensee has developed a proprietary manufacturing process on a commercial scale. Our gene therapy platform therefore has the potential to provide treatments for many other diseases outside of our current focus.
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