Royalty Report: Drugs, Disease, Therapeutic – Collection: 68761

License Grant

This agreement grants the French Licensee the following;
•An exclusive license for the research, development and sale of a drug using specific patented virus vectors for a specific commercial field (AATD field);
•An option to be granted an exclusive license for the research, development and sale of a drug using specific patented virus vectors for another specific commercial field (Allergy field)

License Property

The virus vectors are for two disease indications Alpha-1 anti-trypsin (A1AT) deficiency and Allergy.  Alpha-1 Antitrypsin Deficiency is an inherited condition that raises your risk for lung and liver disease.

Field of Use

Licensee is a gene therapy company focused on discovering and developing new therapeutic products for people living with severe diseases where continuous expression of a therapeutic protein may lead to meaningful clinical benefit. Annapurna’s initial programs address alpha-1 antitrypsin (“A1AT”) deficiency, hereditary angioedema, the cardiomyopathy associated with Friedreich’s ataxia, and severe allergy.

License Grant

This agreement  between French parties grants the following
•An exclusive license for the research, development and sale of a drug using specific patented virus vectors for a specific commercial field;
•A non-exclusive research license for the research and development of a drug using other patented virus vectors for another commercial field.

Field of Use

Licensee is a gene therapy company focused on discovering and developing new therapeutic products for people living with severe diseases where continuous expression of a therapeutic protein may lead to meaningful clinical benefit. Annapurna’s initial programs address alpha-1 antitrypsin (“A1AT”) deficiency, hereditary angioedema, the cardiomyopathy associated with Friedreich’s ataxia, and severe allergy.

License Grant

This agreement grants the French Licensee following
•An exclusive license for the research, development and sale of a drug using specific patented virus vectors for a specific commercial field;
•A non-exclusive research license for the research and development of a drug using other patented virus vectors for another commercial field;
•An option to be granted a non-exclusive license for the sale of the drugs related to the developed under the non-exclusive research license.

Field of Use

Licensee is a gene therapy company focused on discovering and developing new therapeutic products for people living with severe diseases where continuous expression of a therapeutic protein may lead to meaningful clinical benefit. Initial programs address alpha-1 antitrypsin (A1AT) deficiency, hereditary angioedema, the cardiomyopathy associated with Friedreich’s ataxia, and severe allergy.

License Grant

The University grants the French Licensee three licenses to use technology to make and have made, to use and have used, to sell and have sold, to offer for sale, and to import and have imported licensed products and to practice licensed method, in three specific fields of application.

License Property

ANN-001, a gene therapy product candidate, is designed to deliver A1AT protein in patients with A1AT deficiency.

ANN-002 is a gene therapy product candidate designed for the treatment of patients with hereditary angioedema (“HAE”).

Hereditary angioedema (HAE) is a disorder that results in recurrent attacks of severe swelling.  Hereditary angioedema (HAE) is caused by a low level or improper function of a protein called the C1 inhibitor. It affects the blood vessels. An HAE attack can result in rapid swelling of the hands, feet, limbs, face, intestinal tract, larynx (voicebox), or trachea (windpipe).

ANN-004 is a gene therapy program for the treatment of severe allergies, which cause extreme discomfort and can be fatal.

Hereditary angioedema is a disorder characterized by recurrent episodes of severe swelling (angioedema). The most common areas of the body to develop swelling are the limbs, face, intestinal tract, and airway. Minor trauma or stress may trigger an attack, but swelling often occurs without a known trigger

Field of Use

Licensee is a gene therapy company focused on discovering and developing new therapeutic products for people living with severe diseases where continuous expression of a therapeutic protein may lead to meaningful clinical benefit. Annapurna’s initial programs address alpha-1 antitrypsin (“A1AT”) deficiency, hereditary angioedema, the cardiomyopathy associated with Friedreich’s ataxia, and severe allergy.

License Grant

The Parties will co-develop the Licensor’s ARO-AAT program.

License Property

ARO-AAT is a program,  second-generation, subcutaneously administered RNAi therapeutic candidate. ARO-AAT is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients. Reducing production of the inflammatory Z-AAT protein is expected to halt the progression of liver disease and potentially allow it to regenerate and repair.

Field of Use

The field of use is as a treatment for liver disease associated with alpha-1 antitrypsin deficiency (AATD), which is a rare genetic disorder that severely damages the liver and lungs of affected individual, children and adults and pulmonary disease in adults.

Licensee is in the rare disease and GI therapy space, and is well-positioned to work with the patient and medical community to help meet the severe unmet need of patients with Alpha-1 liver disease.

License Grant

Based on licensing and technology transfer agreement between the companies, upon initiation of sales of GLASSIA manufactured by Licensee which is expected to take place during 2021, Licensee will pay royalties to the Company.

License Property

The Companys flagship product is GLASSIA; the first liquid, ready-to-use, intravenous plasma-derived AAT product approved by the U.S. Food and Drug Administration.

The Company is focused on plasma-derived protein therapeutics for orphan indications, and has a commercial product portfolio and a late-stage product pipeline. The Company uses its proprietary platform technology and know-how for the extraction and purification of proteins from human plasma to produce Alpha-1 Antitrypsin (AAT) in a highly-purified, liquid form, as well as other plasma-derived Immune globulins.  AAT is a protein derived from human plasma with known and newly-discovered therapeutic roles given its immunomodulatory, anti-inflammatory, tissue-protective and antimicrobial properties.

Field of Use

GLASSIA is proprietary product for the treatment of Alpha-1 Antitrypsin Deficiency

Alpha-1 antitrypsin (AAT) deficiency is a condition in which the body does not make enough of AAT, a protein that protects the lungs and liver from damage. The condition can lead to COPD and liver disease (cirrhosis).
Chronic Obstructive Pulmonary Disease (COPD) is an umbrella term used to describe progressive lung diseases including emphysema, chronic bronchitis, and refractory (non-reversible) asthma. This disease is characterized by increasing breathlessness.

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.

License Grant

Licensor of Israel grants to Licensee of Switzerland a worldwide license within the Field, with the right to Sublicense, but only by Licensee, to Permitted Manufacturing Sublicensees, under the Licensor Licensed Patent Rights and Licensor Licensed Know-How, to develop, make, have made, import, export, and use Licensee Products; provided, that such license shall only be exclusive within the Licensee Territory, and provided, further, that such imports, exports and use do not involve directly or indirectly marketing and distribution of Licensee Products outside the Licensee Territory.

Licensor grants an exclusive right within the Field to enforce the Licensor Licensed Patent Rights in the Licensee Territory.

For the Trademarks, Licensor grants a non-exclusive license to use Licensor Trademarks for the sole purpose of marketing, offering for sale, selling, having sold, advertising and promoting Licensor Products under this Agreement.

License Property

Licensor owns certain intellectual property, confidential information, and regulatory licenses relating to the production of A1P1 biopharmaceutical products;

A1P1 shall mean human alpha-one antitrypsin, also known as alpha-one proteinase inhibitor.

Licensee Product shall mean any A1P1 concentrate prepared by and/or on behalf of Licensee, other than by Licensor, from human plasma IV-1 or IV-1+4 for intravenous administration that is encompassed by a claim of the Licensor Licensed Patent Rights, or produced using the Licensor Licensed Know-How.

Licensor Trademarks shall mean those trademarks owned by Licensor for A1P1 concentrate prepared from human plasma for intravenous administration registered in the Licensee Territory, expressly including the trademark GLASSIAâ„¢.

The Patents include
—  Large scale preparation of Alpha-1 Proteinase inhibitor and use thereof;
—  Compositions comprising purified, stable, active Alpha-1 Proteinase inhibitor, process for producing same and use thereof;
—  Method for purification of Alpha-1 Proteinase inhibitor.

Field of Use

GLASSIATM is the first available ready-to-use liquid alpha1-proteinase inhibitor (Alpha1-PI) and is indicated as a chronic augmentation and maintenance therapy in adults with emphysema due to congenital deficiency of alpha-1 antitrypsin (AAT), an under-diagnosed hereditary condition characterized by a low level of alpha-1 protein in the blood.

Alpha-1 antitrypsin deficiency is an inherited disorder that may cause lung disease and liver disease.  Alpha-1-antitrypsin (AAT) is a protein produced in the liver that protects the body's tissues from being damaged by infection-fighting agents released by its immune system. In alpha-1 antitrypsin deficiency, the body's normal production of AAT is reduced, resulting in the destruction of sensitive lung tissue.

The Field shall mean the use of an A1P1 concentrate produced from human plasma for intravenous administration in humans.

This agreement is specifically for the purpose of processing A1P1 from human plasma derived Cohn fraction IV-1 for Licensee Products for sale in the Licensee Territory.