AVRO News and Press, AVROBIO Inc. From 07/13/22

The shares of gene therapy company AVROBIO ( NASDAQ: AVRO ) jumped ~9% in the pre-market Monday after the company announced that the FDA awarded the orphan drug designation for AVR-RD-05, a gene therapy candidate for mucopolysaccharidosis type II (MPSII), or Hunter syndrom...

AVR-RD-05 is the fourth AVROBIO gene therapy to receive orphan drug designation FDA previously granted rare pediatric disease designation for AVR-RD-05 AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free peop...

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BioLineRx (NASDAQ:BLRX) appointed Holly May as its chief commercial officer to bolster efforts for its investigational stem cell therapy for blood cancer. The Israeli company said that in the newly created role, which is based in the U.S., May will be responsible for commercial planning and l...

With two overwhelmingly positive U.S. FDA advisory committee votes this week, bluebird bio (NASDAQ:BLUE) is poised to see beti-cel (betibeglogene autotemcel) approved for beta-thalassemia and eli-cel (elivaldogene autotemcel) for cerebral adrenoleukodystrophy. Should the FDA follow ...

Reviewing its coverage on biotechs with negative enterprise values, Morgan Stanley says that the number of companies on the list with potential catalysts in 2022 has increased to 27 in May from 20 previously. Adding market capitalization to total debt and deducting cash and cash equivalents, ...

Genetically modified hematopoietic stem cells lead to supraphysiological levels of therapeutic protein in a mouse model of Pompe disease sustained at eight months after administration Substantial reduction in glycogen observed across cardiac and skeletal muscles, as well as CN...

Avrobio (NASDAQ:AVRO) said new interim data from a phase 1/2 trial of its gene therapy AVR-RD-04 for cystinosis showed stabilized or improved symptoms related to the disease following treatment. Cystinosis is a rare, genetic disorder in which an amino acid called cystine builds up i...

New early data show key visual motor integration, visual perception and motor coordination measures impacted by cystinosis stabilized or improved post gene therapy Systemic reach of AVR-RD-04 observed across multiple other measures All five dosed patients remain off or...