US2022370348A1PendingUtilityA1

Slow intraventricular delivery

71
Assignee: GENZYME CORPPriority: Feb 9, 2006Filed: Jan 10, 2022Published: Nov 24, 2022
Est. expiryFeb 9, 2026(expired)· nominal 20-yr term from priority
Inventors:James DodgeMarco A. PassiniLamya ShihabuddinSeng H. Cheng
A61P 25/16A61P 25/14A61P 25/08A61P 23/00C12N 2750/14143A61K 9/0024A61K 31/00C12N 15/86A61P 11/00A61P 3/10A61P 3/00A61P 3/08C12N 9/16A61P 43/00A61P 25/28A61K 9/0085A61P 25/00A61P 1/16A61P 13/12
71
PatentIndex Score
0
Cited by
0
References
0
Claims

Abstract

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.

Claims

exact text as granted — not AI-modified
1 .- 53  (canceled) 
     
     
         54 . A method of treating a lysosomal storage disease in patient in need thereof, the method comprising administering to the brain of the patient an intraventricular dose of a vector encoding an enzyme that the patient is deficient in, wherein the dose is administered at a rate of at least 50% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         55 . The method of  claim 54 , wherein the vector is a recombinant adeno-associated (rAAV) vector. 
     
     
         56 . The method of  claim 54 , wherein the dose is administered at a rate of at least 100% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         57 . The method of  claim 56 , wherein the vector is a recombinant adeno-associated (rAAV) vector. 
     
     
         58 . The method of  claim 54 , wherein the dose is administered at a rate of at least 150% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         59 . The method of  claim 54 , wherein the dose is administered at a rate of at least 200% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         60 . The method of  claim 54 , wherein the dose is administered at a rate of between 50% and 400% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         61 . The method of  claim 60 , wherein the vector is a recombinant adeno-associated (rAAV) vector. 
     
     
         62 . The method of  claim 54 , wherein the administration of a single dose of the vector is more than 2 hours. 
     
     
         63 . The method of  claim 54 , wherein the administration of a single dose of the vector is more than 4 hours. 
     
     
         64 . The method of  claim 54 , wherein administration of a single dose of the vector is from 2 hours to 10 hours. 
     
     
         65 . The method of  claim 54 , wherein administration of a single dose of the vector is from 4 hours to 8 hours. 
     
     
         66 . The method of  claim 55 , wherein administration of a single dose of the vector is from 2 hours to 10 hours. 
     
     
         67 . The method of  claim 55 , wherein administration of a single dose of the vector is from 4 hours to 8 hours. 
     
     
         68 . A method of treating Niemann-Pick disease in patient in need thereof, the method comprising administering to the brain of the patient an intraventricular dose of a vector encoding a sphingomyelinase, wherein the dose is administered at a rate of at least 50% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         69 . The method of  claim 68 , wherein the Niemann-Pick disease is Niemann-Pick A disease. 
     
     
         70 . The method of  claim 68 , wherein the Niemann-Pick disease is Niemann-Pick B disease. 
     
     
         71 . The method of  claim 68  wherein the vector is a recombinant adeno-associated (rAAV) vector. 
     
     
         72 . The method of  claim 68 , wherein the dose is administered at a rate of at least 100% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         73 . The method of  claim 72 , wherein the vector is a recombinant adeno-associated (rAAV) vector. 
     
     
         74 . The method of  claim 68 , wherein the dose is administered at a rate of at least 150% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         75 . The method of  claim 68 , wherein the dose is administered at a rate of at least 200% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         76 . The method of  claim 68 , wherein the dose is administered at a rate of between 50% and 400% of the turn-over time of the cerebrospinal fluid of the patient. 
     
     
         77 . The method of  claim 76 , wherein the vector is a recombinant adeno-associated (rAAV) vector. 
     
     
         78 . The method of  claim 68 , wherein the administration of a single dose of the vector is more than 2 hours. 
     
     
         79 . The method of  claim 68  wherein the administration of a single dose of the vector is more than 4 hours. 
     
     
         80 . The method of  claim 68 , wherein administration of a single dose of the vector is from 2 hours to 10 hours. 
     
     
         81 . The method of  claim 68 , wherein administration of a single dose of the vector is from 4 hours to 8 hours. 
     
     
         82 . The method of  claim 71 , wherein administration of a single dose of the vector is from 2 hours to 10 hours. 
     
     
         83 . The method of  claim 71 , wherein administration of a single dose of the vector is from 4 hours to 8 hours. 
     
     
         84 . The method of  claim 71 , wherein the vector is delivered using an implantable pump.

Join the waitlist — get patent alerts

Track US2022370348A1 — get alerts on status changes and closely related new filings.

We store only your email — no account needed. See our privacy policy.