BioCentury | Nov 13, 2019
Finance

Allievex, backed by Pappas and Novo, scoops up BioMarin ultra-rare disease assets

...sulfate. The disorder is caused by a mutation in the NAGLU gene; tralesinidase alfa is NAGLU...
...to in-license additional candidates in the near term. Targets IGF-2 - Insulin-like growth factor 2 NAGLU...
BioCentury | Oct 24, 2019
Finance

Allievex, backed by Pappas and Novo, scoops up BioMarin ultra-rare disease assets

...sulfate. The disorder is caused by a mutation in the NAGLU gene; tralesinidase alfa is NAGLU...
...to in-license additional candidates in the near term. Targets IGF-2 - Insulin-like growth factor 2 NAGLU...
BioCentury | Feb 9, 2018
Clinical News

Abeona reports early data for AAV9 MPS IIIB gene therapy

...IIIB (MPS IIIB, Sanfilippo type B syndrome). ABO-101 is an AAV9 vector that delivers the N-acetylglucosaminidase alpha (NAGLU)...
...and plasma by 60%. ABO-101 also reduced urinary total glycosaminoglycans (GAGs) by 67% and normalized NAGLU...
...Business: Endocrine/Metabolic Molecular target: N-acetylglucosaminidase alpha (NAGLU) Description: Adeno-associated viral serotype 9 (AAV9) vector that delivers the NAGLU...
BioCentury | Feb 7, 2018
Clinical News

Abeona gains on early data for AAV9 MPS IIIB gene therapy

...mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo B syndrome). ABO-101 is an AAV9 vector that delivers the N-acetylglucosaminidase alpha (NAGLU)...
...said ABO-101 significantly reduced cerebrospinal fluid (CSF), urine and plasma heparan sulfate levels and normalized NAGLU...
...animal studies of high-dose systemic AAV9 variants (see BioCentury Extra, Feb. 5) . Alicia Parker ABO-101 Abeona Therapeutics Inc. N-acetylglucosaminidase alpha (NAGLU)...
BioCentury | Feb 2, 2018
Product Development

AA Viral meme

...AAV9 Abeona Therapeutics Inc. (NASDAQ:ABEO) ABO-101 Mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo type B syndrome) N-acetylglucosaminidase alpha (NAGLU)...
BioCentury | Nov 10, 2017
Clinical News

BioMarin reports Phase I/II data for BMN 250 in mucopolysaccharidosis IIIB

...were presented at the Inborn Errors of Metabolism meeting in Rio de Janeiro. BMN-250 is N-acetylglucosaminidase alpha (NAGLU)...
...Pharmaceutical Inc. (NASDAQ:BMRN), San Rafael, Calif. Product: BMN 250 Business: Endocrine/Metabolic Molecular target: N-acetylglucosaminidase alpha (NAGLU) Description: NAGLU...
...on brain structure assessed by MRI Status: Phase I/II data Milestone: NA Claire Quang BMN 250 BioMarin Pharmaceutical Inc. N-acetylglucosaminidase alpha (NAGLU) Inborn...
BioCentury | Mar 10, 2017
Company News

Alexion SBC-103 news

...MPS IIIB, Sanfilippo B syndrome) will continue to receive SBC-103. The product is recombinant human N-acetylglucosaminidase alpha (NAGLU)...
...May 11, 2015 ). Alexion Pharmaceuticals Inc. (NASDAQ:ALXN), New Haven, Conn. Business: Endocrine/Metabolic Chris Lieu SBC-103 Alexion Pharmaceuticals Inc. N-acetylglucosaminidase alpha (NAGLU)...
BioCentury | Nov 18, 2016
Company News

uniQure restructuring news

...will seek options, including partnerships, for AMT-110 and AMT-140. AMT-110, an AAV5 vector carrying the N-acetylglucosaminidase alpha (NAGLU)...
...the Netherlands Business: Gene/Cell therapy, Pharmaceuticals Chris Lieu AAV-S100A1 AAV2-GDNF AMT-060 AMT-110 AMT-130 Bristol-Myers Squibb Co. uniQure N.V. Factor IX Glial cell-derived neurotrophic factor (GDNF) Huntingtin (HTT) N-acetylglucosaminidase alpha (NAGLU) S100...
BioCentury | Nov 15, 2016
Company News

uniQure paring down gene therapy portfolio

...Phase I/II candidate AMT-140 to treat Parkinson's disease. AMT-110 is an AAV5 vector carrying the N-acetylglucosaminidase alpha (NAGLU)...
...shed $0.57 to $7.73 on Tuesday. Chris Lieu AAV-S100A1 AAV2-GDNF AMT-060 AMT-110 AMT-130 Bristol-Myers Squibb Co. uniQure N.V. Factor IX Glial cell-derived neurotrophic factor (GDNF) Huntingtin (HTT) N-acetylglucosaminidase alpha (NAGLU) S100...
BioCentury | May 2, 2016
Clinical News

BMN 250: Phase I/II started

...Pharmaceutical Inc. (NASDAQ:BMRN), San Rafael, Calif. Product: BMN 250 Business: Endocrine/Metabolic Molecular target: N-acetylglucosaminidase alpha (NAGLU) Description: N-acetylglucosaminidase alpha (NAGLU)...
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