AVRO News and Press, AVROBIO Inc. From 09/20/22

The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to Avrobio's ( NASDAQ: AVRO ) gene therapy AVR-RD-04 to treat cystinosis. Cystinosis is a rare, multisystem genetic disorder characterized by the excess build up of an amino acid cal...

Second AVROBIO gene therapy program that has been granted rare pediatric disease designation by FDA AVR-RD-04 has previously received orphan drug designation from FDA and EMA AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to ...

Avrobio ( NASDAQ: AVRO ) said its autologous hematopoietic stem cell (HSC) gene therapy AVR-RD-05 was cleared to enter a phase 1/2 trial in U.K. to treat infants with neuronopathic mucopolysaccharidosis type II (nMPS-II) or Hunter syndrome. nMPS-II is a rare genetic meta...

First in-human pediatric Phase 1/2 study to evaluate the safety and tolerability of first-in-class gene therapy targeting infants age ≥3 months and ≤12 months, diagnosed with nMPS-II University of Manchester (U.K.) expects to initiate clinical trial later this ye...

Summary Shares have fallen by 30% so far in 2022. Durability and safety for the Fabry disease gene therapy program continues to look best-in-class and enrollment should accelerate due to investigator enthusiasm. Excitement is palpable for CAR-Treg programs as renal transplant ...

AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that members of its senior management team are scheduled to participate in four upcoming investor conferences: Citi's 17 th...

AVROBIO press release ( NASDAQ: AVRO ): Q2 GAAP EPS of -$0.64 misses by $0.09 . As of June 30, 2022, AVROBIO had $132.4 million in cash and cash equivalents, as compared to $189.6 million in cash and cash equivalents as of December 31, 2021. For further details s...

Presented positive data from first five patients in Phase 1/2 cystinosis trial 1 showing systemic gene therapy effect at the American Society of Gene and Cell Therapy Annual Meeting; all five patients remain off oral cysteamine Comprehensive Gaucher disease fra...

AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced that members of its senior management team are scheduled to participate in two upcoming investor conferences: H...

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...