FXN Gene Therapy Treats Heart Problems in New FA Mouse Model

A gene therapy that delivers a healthy version of the FXN gene can correct heart problems in a new mouse model with cardiac-specific symptoms of Friedreich’s ataxia[2] (FA), a study reports.[1] This animal model more accurately mirrors the early cardiac abnormalities seen in FA patients, the researchers wrote, providing new opportunities to study FA symptoms and investigate disease-modifying therapies. The...

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Exicure Announces Agenda for Upcoming Neuroscience Pipeline Update at Virtual R&D Day

CHICAGO & CAMBRIDGE, Mass.–(BUSINESS WIRE[1])–Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, will host a virtual R&D Day on Thursday, January 7, 2021 from 09:00 am to 10:30 am ET to discuss Exicure’s neuroscience pipeline, including its lead program for Friedreich’s Ataxia (FA). Exicure will discuss its progress with XCUR-FXN,...

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Exicure Announces Agenda for Upcoming Neuroscience Pipeline Update at Virtual R&D Day

CHICAGO & CAMBRIDGE, Mass.–(BUSINESS WIRE[1])–Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, will host a virtual R&D Day on Thursday, January 7, 2021 from 09:00 am to 10:30 am ET to discuss Exicure’s neuroscience pipeline, including its lead program for Friedreich’s Ataxia (FA). Exicure will discuss its progress with XCUR-FXN,...

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AavantiBio Launches With $107M to Advance Gene Therapy for FA

The gene therapy[1] startup AavantiBio[2] has launched with $107 million in funding to advance its lead clinical program for the treatment of Friedreich’s ataxia[3] (FA). Early this year, the Boston-based company received[4] a $1 million grant from the Muscular Dystrophy Association[5] (MDA)’s Venture Philanthropy Fund[6] to start producing its gene therapy candidate, expected to be tested in a Phase 2 clinical...

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