Royalty Report: Drugs, Disease, Therapeutic – Collection: 339531

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Curated Royalty Rate Report
Category: Technology Licenses, Created On: 2022-04-28, Record Count: 11

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: 11

Primary Industries

  • Drugs
  • Disease
  • Therapeutic
  • Cancer
  • Genome
  • Drug Discovery

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: 339531

License Grant
The parties amend certain terms of the original License Agreement in light of the sublicensee agreements.

The First Amendment has been amended to include but not limited to License Product, Regulatory Exclusivity, Sublicensee Royalty Term, Sublicensee Product and Royalty payments.

License Property
Patent applications relates to a novel synthetic microdystrophin gene to make, sell and distribute products for use in the treatment of Duchene and related disease indications resulting from a lack of functional dystrophin.

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 patients’ 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 Solid’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.

Field of Use
Field of use is for the cure Duchenne muscular dystrophy (“Duchenne”), a genetic muscle-wasting disease predominantly affecting boys, with symptoms that usually manifest between three and five years of age. Duchenne is a progressive, irreversible and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births.  SGT-001 is our lead gene transfer candidate and the focus of our research and development efforts. Gene transfer, a type of gene therapy, is designed to address diseases caused by mutated genes through the delivery of functional versions of those genes, called transgenes. The transgenes are then utilized by the body to produce proteins that are absent or not functional prior to treatment, potentially offering long-lasting beneficial clinical effects.

IPSCIO Record ID: 212182

License Grant
The Netherlands Licensor agreed to provide the Licensee with an exclusive license to certain intellectual property with an option to convert the exclusive license into a co-exclusive license and the Settlement Parties agreed to stop most existing efforts to continue with ongoing litigation and opposition and other administrative proceedings concerning Licensee’s intellectual property.

Th parties entered into an agreement pursuant to which all legal actions in the U.S. and certain legal actions in Europe would be stopped or withdrawn as between the Settlement Parties. Under the terms of the License Agreement and the Settlement Agreement, the company agreed to make up-front payments.  Additionally, the company may be liable for regulatory and sales milestones for eteplirsen as well as exon 45 and exon 53 skipping product candidates.  The Parties will also be eligible to receive royalty payments for exon 51 skipping products, exon 45 skipping products and exon 53 skipping products.

License Property
EXONDYS 51, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. EXONDYS 51 is studied in clinical trials under the name of eteplirsen and is marketed in the U.S. under the trademarked name of EXONDYS 51® (eteplirsen) Injection.
Field of Use
Disease-modifying pipeline of exon-skipping drug candidates targeting DMD.

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact. DMD is one of four conditions known as dystrophinopathies.

EXONDYS 51 uses Licensee's proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51 has not been established.

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.

IPSCIO Record ID: 289156

License Grant
The Netherland Licensor hereby grants to English Licensee, and Licensee hereby accepts and shall have with effect from the Effective Date, an exclusive (even as to Licensor and its Affiliates), worldwide sublicenseable license in the Territory under all of Licensor’s and its Affiliates’ rights, title and interest in and to the Exclusively Licensed IP pertaining to the Exon 51 Program and/or to any Compound(s) therein to make, have made, use, sell, offer for sale and import Compounds under the Exon 51 Program as and into Licensee Products in the Field.
License Property
Compound means any of (i) each of PRO051 and PRO044 and (ii) compounds comprising an antisense oligonucleotide (AON) that is directed to exon skipping by a mechanism intended to directly induce single exon skipping in the dystrophin gene for the treatment of DMD for the relevant exons of the Exon 51 Program, the Exon 44 Program, the DMD Program 3, or the DMD Program 4, as applicable, and meeting the criteria set by the JSC  (Joint Steering Committee for the relevant Licensor Collaboration Program, unless otherwise mutually agreed by the JSC, and, in the case of (i) or (ii), all derivatives and improvements of such compound, (a) that are existing as of the Effective Date or (b) that are Researched and/or Developed by Licensor under a Licensor Collaboration Program or (c) as identified, further modified, optimized or otherwise Researched or Developed by Licensee under a Licensee Development Program.

PRO044 means a 2’-O-methyl-phosphoro-thioate-oligoribonucleotide with sequence 5’-UCA GCU UCU GUU AGC CAC UG-3’.

“PRO051 means a 2’-O-methyl-phosphoro-thioate-oligoribonucleotide with sequence 5’-UCA AGG AAG AUG GCA UUU CU-3’.

Exon 51 Program means a program of Research and Development activities for Licensor’s proprietary Compounds targeted to specific exons of the human dystrophin gene existing as of the Effective Date or arising under this Agreement that are intended to treat DMD by a mechanism of single exon-skipping intended to induce cells to specifically skip translation of exon 51 of the dystrophin gene, including the Lead Compound “PRO051” and any other Compounds that are Researched or Developed by or on behalf of Licensor or its Affiliate qualifying under this definition.

DMD means Duchenne Muscular Dystrophy.

Exclusively Licensed IP means (i) with respect to each Compound in the Exon 51 Program, and (ii) with respect to each Compound in a Licensor Collaboration Program for which Licensee exercises the Option and receives rights upon exercise pursuant to or by operation of the applicable provisions, in the case of either (i) or (ii) above shall include both (a) and (b) as follows (a) any and all Licensor Know-How and Joint Know-How, in each case that describes the composition of matter of or is necessary or useful for the making, use (including method of use) or sale of such Compound, and (b) any and all Licensor Patent Rights and Joint Patent Rights, in each case that claims or covers the composition of matter of or the making, use (including method of use) or sale of any such Compound.

Licensee Product means a Product Developed and commercialized by Licensee or its Affiliate or Sublicensee under or resulting from a Licensee Development Program.

Licensee Development Program means each of (i) the Exon 51 Program, and (ii) any Licensor Collaboration Program for which Licensee has exercised its Option and, for both (i) and (ii), where such Program has not been terminated by Licensee or terminated by Licensor (i.e for a termination by Licensor in the case of an uncured material breach by Licensee of its diligence or other obligations with respect to such Program).

Exon 51 Program means a program of Research and Development activities for Licensor’s proprietary Compounds targeted to specific exons of the human dystrophin gene existing as of the Effective Date or arising under this Agreement that are intended to treat DMD by a mechanism of single exon-skipping intended to induce cells to specifically skip translation of exon 51 of the dystrophin gene, including the Lead Compound “PRO051” and any other Compounds that are Researched or Developed by or on behalf of Licensor or its Affiliate qualifying under this definition.

Exon 44 Program means a program of Research and Development activities for Licensor’s proprietary Compounds targeted to specific exons of the human dystrophin gene existing as of the Effective Date or arising under this Agreement that are intended to treat DMD by a mechanism of single exon-skipping intended to induce cells to specifically skip translation of exon 44 of the dystrophin gene, including the Lead Compound “PRO044” and any other Compounds that are Researched or Developed by or on behalf of Licensor or its Affiliate qualifying under this definition.

DMD Program 3 means a program of Research and Development activities for Licensor’s proprietary Compounds targeted to specific exons of the human dystrophin gene existing as of the Effective Date or arising under the collaboration that are directed to exon skipping by a mechanism intended to directly induce single exon skipping in the dystrophin gene for the treatment of DMD by inducing cells to specifically skip translation of a mutually agreed exon(s) of the dystrophin gene, including any Secondary Compounds developed by Licensor.

DMD Program 4 means a program of Research and Development activities for Licensor’s proprietary Compounds targeted to specific exons of the human dystrophin gene existing as of the Effective Date or arising under the collaboration that are directed to exon skipping by a mechanism intended to directly induce single exon skipping in the dystrophin gene for the treatment of DMD by inducing cells to specifically skip translation of a mutually agreed exon(s), but exon(s) that are different from the exon(s) of DMD Program 3, of the dystrophin gene and any Secondary Compounds developed by Licensor.

Licensor Collaboration Program means each of Licensor’s programs of Research and Development activities for Compounds targeted to specific exons of the human dystrophin gene, and collectively, the Licensor Collaboration Programs includes the “Exon 44 Program”, the “DMD Program 3” and the “DMD Program 4”.

Licensee Development Program means each of (i) the Exon 51 Program, and (ii) any Licensor Collaboration Program for which Licensee has exercised its Option and, for both (i) and (ii), where such Program has not been terminated by Licensee or terminated by Licensor (i.e for a termination by Licensor in the case of an uncured material breach by Licensee of its diligence or other obligations with respect to such Program).

Field of Use
This agreement pertains to the development and commercializeation of RNA-based therapeutics for the treatment of DMD (Duchenne muscular dystrophy).

Duchenne muscular dystrophy (DMD) is a progressive form of muscular dystrophy that occurs primarily in males, though in rare cases may affect females. DMD causes progressive weakness and loss (atrophy) of skeletal and heart muscles.

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: 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: 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: 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: 248957

License Grant
Licensor grants to Licensee an exclusive (except as to Licensor) license under the Licensor Patents to the extent necessary to conduct research and development of, and to make, have made, use, offer for sale, sell, have sold and import Licensed Products in the Shared Territory during the term of this Agreement.

Licensor hereby grants Licensee a paid-up non-exclusive license to use Licensor Know-How in the Shared Territory to the extent necessary to research, develop and Commercialize the Licensed Products during the Research Term and while Licensee is co-promoting the applicable Licensed Product in the Shared Territory.

License Property
ONYX-015 means any and all formulations of the genetically engineered adenovirus DL1520 described in US Patent Numbers 5,677,178 and 5,846,945 that selectively replicates in and kills cancer cells based on abnormal p53 pathway function and is currently in clinical trials sponsored by Licensee.

5,677,178 – Cytopathic viruses for therapy and prophylaxis of neoplasia
5,846,945 – Cytopathic viruses for therapy and prophylaxis of neoplasia

Neoplasia is the formation or presence of a new, abnormal growth of tissue.

Gene Product means the adenovirus product selected by Licensor.  The parties collaborate to identify a Gene Product based on incorporation of a specific gene, selected by Licensor and encoding a specific member of one of three classes of proteins, into ONYX-015, or another adenovirus agreed to by the Parties that selectively replicates in and kills cancer cells based on abnormal p53 pathway function, for the purpose of expressing in cancer cells a particular protein with useful anti-cancer activity.

Independent Product shall have the meaning If, Licensor either (A) is not deemed to be diligent(b) or (c) or terminates Development as to a Licensed Product, in each case after the filing of an IND therefor and prior to the End of Phase II Clinical Trials for such Licensed Product, or (B) fails to fulfill its obligations with respect to diligent Development of a Licensed Product after the End of Phase II Clinical Trials.

Pro-Drug Product means the adenovirus product selected by Licensor to identify a Pro-Drug Product based on incorporation of a gene for a prodrug activating enzyme into ONYX-015, or another adenovirus agreed to by the Parties that selectively replicates in and kills cancer cells based on abnormal p53 pathway function, for the purpose of activating a prodrug into a specific active drug form.

Licensed Product means Local/Regional ONYX-015, Systemic ONYX-015, the Pro-Drug Product (upon selection of the Selected Drug System pursuant to this agreement(b)) or the Gene Product (upon selection of the Selected Gene pursuant to this agreement(c)), but excluding those of the foregoing that become Independent Products unless and until such time as Licensor exercises its buy-back rights under this agreement or that become Terminated Products.

Local/Regional ONYX-015 means the unmodified ONYX-015 currently in clinical trials sponsored by Licensee, and including any formulation thereof, such as, but without limitation, lyophilized formulations, mouthwash formulations, and formulations that stabilize ONYX-015 for storage at refrigerated or room temperature, but excluding modified formulations that comprise Systemic ONYX-015.

Field of Use
The rights granted apply to the drug industry.
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