Delving into the Latest Updates on Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor(RegenxBio) with Synapse

This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease. This study is intended to follow those subjects for up to 5 years since they received treatment to look for any long-term safety concerns. There is no investigational drug or therapy provided as part of this study.

Start Date30 Sep 2019

Sponsor / Collaborator

RegenxBio, Inc.

NCT02651675 / TerminatedPhase 1/2

AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)

This first-in-human study is intended to evaluate the safety and preliminary effectiveness of AAV (Adeno-associated virus)-based liver-directed gene therapy in the treatment of adults with Homozygous Familial Hypercholesterolemia (HoFH).

Start Date01 Mar 2016

Sponsor / Collaborator

RegenxBio, Inc.

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100 Clinical Results associated with Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor(RegenxBio)

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100 Translational Medicine associated with Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor(RegenxBio)

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100 Patents (Medical) associated with Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor(RegenxBio)

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100 Deals associated with Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor(RegenxBio)

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hyperlipidemias	Phase 2	-	University of Pennsylvania	-
Hyperlipidemia Type IIa	Preclinical	US	-	-
Hyperlipoproteinemia Type II	Discovery	NL	RegenxBio, Inc.	01 Mar 2016
Hyperlipoproteinemia Type II	Discovery	US	RegenxBio, Inc.	01 Mar 2016
Hyperlipoproteinemia Type II	Discovery	CA	RegenxBio, Inc.	01 Mar 2016
Hyperlipoproteinemia Type II	Discovery	IT	RegenxBio, Inc.	01 Mar 2016