Alseres Pharmaceuticals, Inc.

Children's Medical Center Corporation

Other

National Taiwan University

Ptc Therapeutics, Inc.

Precigen, Inc.

Boston Life Sciences, Inc.

Richard M Thorn

Marsha A Moses

Dmitri G Wiederschain

Marc E Lanser

Mark Pykett

Cara Ferreira

David Leyh

Chris Sterzinger

Wuh-Liang (“Paul”) Hwu

Gregory E Robinson

Stephen Ezra Schauer

Kirsten Gruis

Mark R Hurtt

Darby Thomas

Noel James Cusack

Mark A Lanser

The present invention is directed to compositions and methods for treating aromatic-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8×10vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4×10vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7×10vg/mL. This invention is also directed to methods for treating aromatic-amino acid decarboxylase (AADC) deficiency, wherein the method optionally further comprises the step of administering a therapeutically effective dose of dopamine-antagonist to the subject such as risperidone. This invention is also directed to methods for treating aromatic-amino acid decarboxylase (AADC) deficiency, wherein the method optionally comprises providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, and empty capsids.

Gene therapy for AADC deficiency

The invention provides polynucleotides, vectors and viruses expressing frataxin and methods of treating Friedreich's Ataxia.

Frataxin expression constructs

The present invention is directed to compositions and methods for treating aromatic-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8&#xd7;10vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4&#xd7;10vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7&#xd7;10vg/mL. This invention is also directed to methods for treating aromatic-amino acid decarboxylase (AADC) deficiency, wherein the method optionally further comprises the step of administering a therapeutically effective dose of dopamine-antagonist to the subject such as risperidone. This invention is also directed to methods for treating aromatic-amino acid decarboxylase (AADC) deficiency, wherein the method optionally comprises providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, and empty capsids.

A diagnostic formulation is provided comprising a tropane having a radioactive concentration of at least 1.6 mCi/mL at least about 51 hours post creation. The diagnostic formulation optionally comprises a radiolabeled dopamine transporter (DAT) ligand useful in the diagnosis of Parkinson's disease (PS). One example of a radiolabeled dopamine transporter (DAT) ligand example is [I]-2&#x3b2;-carbomethoxy-3&#x3b2;-(4-flurophenyl)-N-(3-iodo-E-allyl) nortropane.

Labeled iodinated tropane formulation

A diagnostic formulation is provided comprising a tropane having a radioactive concentration of at least 1.6 mCi/mL at least about 51 hours post creation. The diagnostic formulation optionally comprises a radiolabeled dopamine transporter (DAT) ligand useful in the diagnosis of Parkinson's disease (PS). One example of a radiolabeled dopamine transporter (DAT) ligand example is [I]-2&#x3b2;-carbomethoxy-3&#x3b2;-(4-flurophenyl)-N-(3-iodo-E-allyl)nortropane.

Methods for modulating the axonal outgrowth of central nervous system neurons are provided by means of internalized purine administration such as by intravenous, intraarterial, subcutaneous, intramuscular, intraperitoneal, and intrapleural administration. The methods are noted for stimulating the axonal outgrowth of central nervous system neurons following an injury (e.g., stroke, traumatic brain injury, cerebral aneurism, spinal cord injury and the like).

Systemic purine administration: modulating axonal outgrowth of central nervous system neurons

The present invention relates to methods of inhibiting angiogenesis associated with a disease or disorder with peptides homologous to amino acid residues 130-137 or 132-139 of human troponin subunit I.

Methods of inhibiting angiogenesis with fragments and homologs of troponin subunit 1

The present invention relates to methods of inhibiting angiogenesis associated with a disease or disorder with peptides homologous to amino acid residues 130&#x2013;137 or 132&#x2013;139 of human troponin subunit I.

Methods of inhibiting angiogenesis with fragments and homologs of troponin subunit I

The present invention relates to pharmaceutical compositions comprising therapeutically effective amounts of recombinant troponin C, I or T subunits for the treatment of diseases or disorders involving abnormal angiogenesis.

Pharmaceutical compositions comprising recombinant troponin subunits

The present invention relates to a peptide corresponding to human troponin subunit fragment I amino acid residues 118-137 and homologs thereof, as well as pharmaceutical compositions.

Troponin subunit I fragment and homologs thereof

The present invention relates to pharmaceutical compositions comprising therapeutically effective amounts of troponin C, I or T subunits, fragments, or homologs thereof, for the treatment of diseases or disorders involving abnormal angiogenesis and methods of use thereof.

Growing community of inventors

North Easton, MA, United States of America

Richard M Thorn