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The US Food and Drug Administration (FDA) has granted fast track designation to SB-525, a gene therapy intended for use in patients with hemophilia A.
SB-525 is designed to provide continuous therapeutic expression of factor VIII protein.
SB-525 uses a recombinant adeno-associated virus (AAV) to deliver a human factor VIII complementary DNA construct and synthetic liver-specific promoter to the nucleus of liver cells.
The therapy is being developed by Sangamo Therapeutics, Inc.
In research presented at the 2016 ASH Annual Meeting (abstract 1173), SB-525 induced the expression of human factor VIII in mice and non-human primates (NHPs). SB-525 also corrected the bleeding defect in a mouse model of hemophilia A.
Dosing studies in NHPs demonstrated a robust and reproducible dose response curve, according to researchers. In these animals, mean human factor VIII levels ranged from 5% of normal at the lowest dose to 230% at the highest (AAV doses in the 6 x 1011 – 6 x 1012 vgs/kg range).
The researchers said the peak circulating human factor VIII levels in these experiments exceeded levels previously reported in NHPs. And this could significantly reduce the dose required to achieve therapeutically relevant levels in human subjects.
Sangamo is planning to open a phase 1/2 trial of SB-525 in adults with hemophilia A by the end of the second quarter of 2017. Data from this study are expected in late 2017 or early 2018.
In addition to the fast track designation, SB-525 has orphan drug designation from the FDA.
About fast track designation
The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.
Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the new drug application or biologic license application on a rolling basis as data become available.
Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved. 
The US Food and Drug Administration (FDA) has granted fast track designation to SB-525, a gene therapy intended for use in patients with hemophilia A.
SB-525 is designed to provide continuous therapeutic expression of factor VIII protein.
SB-525 uses a recombinant adeno-associated virus (AAV) to deliver a human factor VIII complementary DNA construct and synthetic liver-specific promoter to the nucleus of liver cells.
The therapy is being developed by Sangamo Therapeutics, Inc.
In research presented at the 2016 ASH Annual Meeting (abstract 1173), SB-525 induced the expression of human factor VIII in mice and non-human primates (NHPs). SB-525 also corrected the bleeding defect in a mouse model of hemophilia A.
Dosing studies in NHPs demonstrated a robust and reproducible dose response curve, according to researchers. In these animals, mean human factor VIII levels ranged from 5% of normal at the lowest dose to 230% at the highest (AAV doses in the 6 x 1011 – 6 x 1012 vgs/kg range).
The researchers said the peak circulating human factor VIII levels in these experiments exceeded levels previously reported in NHPs. And this could significantly reduce the dose required to achieve therapeutically relevant levels in human subjects.
Sangamo is planning to open a phase 1/2 trial of SB-525 in adults with hemophilia A by the end of the second quarter of 2017. Data from this study are expected in late 2017 or early 2018.
In addition to the fast track designation, SB-525 has orphan drug designation from the FDA.
About fast track designation
The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.
Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the new drug application or biologic license application on a rolling basis as data become available.
Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
The US Food and Drug Administration (FDA) has granted fast track designation to SB-525, a gene therapy intended for use in patients with hemophilia A.
SB-525 is designed to provide continuous therapeutic expression of factor VIII protein.
SB-525 uses a recombinant adeno-associated virus (AAV) to deliver a human factor VIII complementary DNA construct and synthetic liver-specific promoter to the nucleus of liver cells.
The therapy is being developed by Sangamo Therapeutics, Inc.
In research presented at the 2016 ASH Annual Meeting (abstract 1173), SB-525 induced the expression of human factor VIII in mice and non-human primates (NHPs). SB-525 also corrected the bleeding defect in a mouse model of hemophilia A.
Dosing studies in NHPs demonstrated a robust and reproducible dose response curve, according to researchers. In these animals, mean human factor VIII levels ranged from 5% of normal at the lowest dose to 230% at the highest (AAV doses in the 6 x 1011 – 6 x 1012 vgs/kg range).
The researchers said the peak circulating human factor VIII levels in these experiments exceeded levels previously reported in NHPs. And this could significantly reduce the dose required to achieve therapeutically relevant levels in human subjects.
Sangamo is planning to open a phase 1/2 trial of SB-525 in adults with hemophilia A by the end of the second quarter of 2017. Data from this study are expected in late 2017 or early 2018.
In addition to the fast track designation, SB-525 has orphan drug designation from the FDA.
About fast track designation
The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.
Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the new drug application or biologic license application on a rolling basis as data become available.
Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.