Methods and systems for treatment of neurological diseases of the central nervous system
First Claim
1. A system comprising:
- a) a therapeutic protein formulation that has been modified for enhanced cellular uptake properties; and
b) an implantable catheter system to physically deliver said therapeutic protein formulation across the blood-brain barrier of patients for the purpose of treating said patients having neurological diseases of the central nervous system.
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Abstract
The present invention is directed to methods and systems for the treatment of inborn genetic errors or other defects that cause deficiencies of active enzymes or proteins within the cells of the central nervous system. Such methods and systems generally comprise an implantable catheter system designed for the chronic delivery of specially formulated proteins to intrathecal, intracerebroventricular, and/or intraparenchymal regions of the central nervous system. The invention has application in the neuropathic aspects of the broad category of lysosomal storage diseases. These genetic based diseases are the result of insufficient enzyme activity to catabolize specific substances, which thereby accumulate in the cellular lysosomes.
73 Citations
128 Claims
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1. A system comprising:
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a) a therapeutic protein formulation that has been modified for enhanced cellular uptake properties; and
b) an implantable catheter system to physically deliver said therapeutic protein formulation across the blood-brain barrier of patients for the purpose of treating said patients having neurological diseases of the central nervous system. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, 27)
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28. A system comprising:
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a) a means of providing for a therapeutic protein formulation that facilitates cellular uptake of proteins within said formulation; and
b) a means of physically bypassing the blood-brain barrier, via an implantable catheter system, so as to deliver said therapeutic protein formulation to target cells for the purpose of treating neurological diseases of the central nervous system. - View Dependent Claims (29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 43, 44, 45, 46, 47, 48, 49, 50, 51, 52, 53)
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54. A system comprising:
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a) a therapeutic protein formulation; and
b) an implantable catheter system comprising a programmable pump to physically deliver said therapeutic protein formulation across the blood-brain barrier at a programmed delivery rate for the purpose of treating patients diagnosed with at least one neurological disease of the central nervous system. - View Dependent Claims (55, 56, 57, 58, 59, 60, 61, 62, 63, 64, 65, 66, 67, 68, 69, 70, 71, 72, 73, 74, 75, 76)
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77. A method comprising the steps of:
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a) providing a therapeutic protein formulation comprising proteins that have been modified for enhanced cellular uptake; and
b) physically delivering said therapeutic protein formulation across the blood brain barrier of patients, via an implantable catheter system, for the purpose of treating neurological diseases of the central nervous system. - View Dependent Claims (78, 79, 80, 81, 82, 83, 84, 85, 86, 87, 88, 89, 90, 91, 92, 93, 94, 95, 96, 97, 98, 99, 100, 101, 102)
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103. A therapy comprising:
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a) a therapeutic protein formulation comprising proteins that have been modified for enhanced cellular uptake; and
b) the physical delivery of said therapeutic protein formulation across the blood brain barrier of patients, via an implantable catheter system, for the purpose of treating neurological diseases of the central nervous system. - View Dependent Claims (104, 105, 106, 107, 108, 109, 110, 111, 112, 113, 114, 115, 116, 117, 118, 119, 120, 121, 122, 123, 124, 125, 126, 127, 128)
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Specification