Genzyme Corporation

Sarepta Therapeutics, Inc.

Cold Spring Harbor Laboratory

Isis Pharmaceuticals, Inc.

Biogen Ma Inc.

Marco A Passini

Seng H Cheng

Lamya S Shihabuddin

Gunnar J Hanson

James C Dodge

James Dodge

C Frank Bennett

Frank Rigo

Gene Hung

Catherine R O'Riordan

Adrian R Krainer

Katherine W Klinger

Frederick Joseph Schnell

Yimin Hua

Greg Stewart

Gregory R Stewart

Robin J Ziegler

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.

Exon skipping oligomer conjugates for muscular dystrophy

Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

Gene therapy for neurodegenerative disorders

The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.

Modified antisense oligomers for exon inclusion in spinal muscular atrophy

The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.

Gene therapy for neurometabolic disorders

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.

Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.

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Northborough, MA, United States of America

Marco A Passini