BioCentury | Oct 17, 2020
Regulation

CHMP’s October recommendations

...CHMP’s support for full marketing authorization. If approved, the gene edited hematopoietic stem cells encoding ARSA...
BioCentury | Feb 15, 2020
Translation in Brief

COVID-19 therapy from Emory and diagnostic from Sherlock co-founders; plus Genentech, Homology and more

...tropism in the central and peripheral nervous systems. The company also showed HMI-202, which encodes ARSA...
...the BBB in non-human primates. In a mouse model of MLD, the gene therapy increased ARSA...
...the compound restored tumor growth suppression. Targets APOE4 - Apolipoprotein E ε 4 ARSA - Arylsulfatase A...
BioCentury | Feb 4, 2020
Finance

Co-founded by UPenn’s Wilson, Passage Bio outlines clinical goals in new IPO filing

...other preclinical therapies for genetically-driven forms of disease: PBML04 for metachromatic leukodystrophy in patients with ARSA...
...in Black Diamond Therapeutics Inc. (NASDAQ:BDTX) (see “Arcutis, Black Diamond Gain” ). Targets ARSA - Arylsulfatase A...
BioCentury | Mar 29, 2019
Clinical News

Orchard planning MAA for leukodystrophy stem cell gene therapy

...to FDA for the ex vivo autologous hematopoietic stem cell-based gene therapy that delivers the arylsulfatase A (ARSA)...
..."Orchard Gains GSK's Rare Disease Gene Therapy Portfolio" ). Sandi Wong, Staff Writer OTL-200 (2696274, GSK2696274) Orchard Therapeutics plc Arylsulfatase A (ARSA) Leukodystrophy metachromatic...
BioCentury | May 11, 2018
Strategy

Takeda hits the gas

...and its principal binding protein IGF binding protein 3 (IGFBP3). SHP611 is a recombinant human arylsulfatase A (ARSA)...
BioCentury | Mar 31, 2018
Strategy

Refashioning Takeda

...testing in Japan to treat convulsive seizures. Shire does have SHP611, a first-in-class recombinant human arylsulfatase A (ARSA)...
BioCentury | Jul 21, 2014
Strategy

AbbVie's plan B

...syndrome). SHP611 is in Phase I/II testing for metachromatic leukodystrophy. SHP611 is a recombinant human arylsulfatase A...
BioCentury | Nov 18, 2013
Strategy

Shire goes vertical

...is in Phase I/II testing for mucopolysaccharidosis IIIA, or Sanfilippo syndrome. SHP611 , a human arylsulfatase A...
...NYSE:SNY) has marketing rights in Japan and certain Asian Pacific countries SHP611 (HGT 1110) Human arylsulfatase A...
BioCentury | Aug 5, 2013
Clinical News

Autologous CD34+ cells: Phase I/II data

...cells transfected with a lentiviral vector encoding functional ARSA protein led to "extensive and stable" ARSA...
...Neurology Molecular target: NA Description: Autologous CD34+ cells transfected with a lentiviral vector encoding functional arylsulfatase A (ARSA)...
...Indication: Treat metachromatic leukodystrophy (MLD) Endpoint: Safety, Gross Motor Function Measure (GMFM) score and residual ARSA...
BioCentury | May 20, 2013
Strategy

Let a thousand ideas bloom

...each candidate selected; royalties Aug-09 Zymenex A/S Product acquisition Acquires Zymenex's Metazym, a recombinant human arylsulfatase A...
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