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12:42 PM, Oct 05, 2018  |  BC Week In Review | Clinical News  |  Clinical Results

Vector copy number declines over time in four Fabry disease patients who received Avrobio gene therapy

Avrobio Inc. (NASDAQ:AVRO) said peripheral blood average vector copy number (VCN) declined over time in four Fabry disease patients who received a single dose of gene therapy AVR-RD-01 in either a Phase I or Phase...
12:15 PM, Jun 22, 2018  |  BC Week In Review | Financial News  |  Completed Offerings

IPO roundup: Avrobio, Aptinyx, Magenta, Kezar, Xeris

lead candidate AVR-RD-01 to treat Fabry's disease. The therapy comprises stem cells engineered to express alpha galactosidase
12:14 PM, Jun 01, 2018  |  BC Week In Review | Financial News  |  Proposed Offerings

Avrobio proposes $86.3M NASDAQ IPO

slated to enter a Phase II trial mid-year -- comprises stem cells engineered to express alpha galactosidase
8:38 AM, Mar 30, 2018  |  BC Week In Review | Clinical News  |  Regulatory

Japan approvals include Hemlibra, Shingrix Galafold

plans to launch the drug "in the coming months." Galafold, a small molecule stabilizer of alpha galactosidase
7:25 AM, Feb 23, 2018  |  BC Week In Review | Financial News  |  Completed Offerings

Amicus raises $300M in bumped-up follow-on

disease in patients aged 16 and older with amenable mutations. The small molecule stabilizer of alpha galactosidase
5:04 AM, Feb 16, 2018  |  BC Week In Review | Clinical News  |  Regulatory

Priority Review for Amicus' migalastat

Migalastat has Orphan Drug and Fast Track designations from FDA. The small molecule stabilizer of alpha galactosidase
11:47 AM, Dec 15, 2017  |  BC Week In Review | Clinical News  |  Regulatory

Amicus submits NDA for Fabry candidate

the basis for accelerated approval (see BioCentury, July 14 ). The small molecule stabilizer of alpha galactosidase
5:33 PM, Oct 19, 2017  |  BC Week In Review | Company News  |  Deals

Chiesi licenses Protalix's Fabry's disease candidate

Protalix BioTherapeutics Inc. (NYSE-M:PLX; Tel Aviv:PLX) granted Chiesi Farmaceutici S.p.A. (Parma, Italy) ex-U.S. rights to pegunigalsidase alfa
PRX-102), which is in Phase III testing to treat Fabry's disease. Protalix will receive $25 million …
320 million in regulatory and commercial milestones, plus royalties. Protalix will maintain full rights to PRX-102
12:31 PM, Jul 14, 2017  |  BC Week In Review | Clinical News  |  Regulatory

FDA clears Amicus to submit migalastat NDA without new trial

patient access scheme (see BioCentury, March 6 ). Migalastat is a small molecule stabilizer of alpha galactosidase
3:00 PM, Mar 02, 2017  |  BC Week In Review | Company News  |  Sales & Marketing

Amicus sales and marketing update

Galafold is one capsule taken every other day. Galafold is a small molecule that enhances alpha galactosidase

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