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AVROBIO Reports Second Quarter 2021 Financial Results and Provides Business Update

U.S. Food and Drug Administration granted Fast Track Designation to AVR-RD-04 for cystinosis
Company is planning to initiate multiple registration trials in 2022
Multiple data and regulatory updates anticipated over next 12 months

CAMBRIDGE, Mass., August 5, 2021 (BUSINESS WIRE)– AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today reported financial results for the quarter ended June 30, 2021, and provided a business update. 

“Data from the patients dosed to date across three indications continue to support our first-in-class, one-time investigational gene therapies as potentially transformative treatments for the more than 50,000 people worldwide living with the life-limiting lysosomal disorders we are researching,” said Geoff MacKay, president and CEO of AVROBIO. “In the second half of this year, we plan to share updated safety data from our ongoing clinical trials and plan to meet with regulatory agencies to discuss initiating potential registration trials in 2022 for Fabry disease, cystinosis and Gaucher disease type 3. With our Pompe disease and Hunter syndrome programs anticipated to enter the clinic in 2022, we remain fiercely committed to our purpose: to free people living with genetic disease from a lifetime of symptoms, chronic treatment and inevitable disease progression.”

Multiple program milestones anticipated over the next 12 months…
…Gaucher disease programs:

  • AVR-RD-02 in Gaucher disease type 1:
    • The first patient dosed has completed the 48-week Phase 1/2 Guard1 trial and has enrolled in the long-term follow up trial, where patients will be followed for 14 years after the initial trial period. AVROBIO is currently enrolling additional patients in the Phase 1/2 trial.
    • AVROBIO expects to present updated safety data on Patient 1 in the fourth quarter of 2021 and a full program update in the first half of 2022. 
  • AVR-RD-06 in Gaucher disease type 3:
    • AVROBIO plans to engage regulatory agencies to discuss the regulatory strategy for AVR-RD-06, the company’s program for Gaucher disease type 3, a form of Gaucher disease associated with severe neurological symptoms.
    • The company is planning to initiate a potential registration trial of AVR-RD-06 in patients with Gaucher disease type 3 in the second half of 2022, subject to regulatory clearance.

Read the complete press release.

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