Business
AVROBIO Reports Third Quarter 2022 Financial Results and Provides Business Update
Patient dosing completed in collaborator-sponsored Phase 1/2 clinical trial for cystinosis Rare pediatric disease designations received for Gaucher disease
About this update from Tectonic Therapeutic, Inc.
[{"type":"text","content":"\nPatient dosing completed in collaborator-sponsored Phase 1/2 clinical trial for cystinosis\n\nRare pediatric disease designations received for Gaucher disease and cystinosis investigational gene therapies\n\nComprehensive Gaucher disease program update planned for Wednesday, Dec. 7\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the third quarter ended Sept. 30, 2022 and provided a business update.\n\n“We had a steady cadence of news this quarter highlighting our steadfast efforts to bring our gene therapies to patients,” said Geoff MacKay, president and CEO of AVROBIO. “We look forward to providing a comprehensive update next month on our HSC gene therapy targeting Gaucher disease, the most common lysosomal disorder, impacting an estimated 30,000 patients worldwide. This update will contain interim clinical data from our Gaucher disease type 1 Phase 1/2 trial, including data out over two years. Notably, we plan to provide an update on our regulatory interactions for a planned Gaucher disease type 3 clinical study, as well as our overall Gaucher clinical development strategy.”\n\nProgram Updates\n\nAVR-RD-02 in Gaucher disease:\n\n\nReceived rare pediatric disease designation (RPDD) from the U.S. Food and Drug Administration (FDA). Read full press release here\n\n\nReceived Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). Read full press release here\n\n\nAVR-RD-02 previously has been granted Fast Track status from FDA and orphan drug designation (ODD) in the U.S. and EU\n\n\nAVR-RD-04 in cystinosis:\n\n\nPatient dosing completed in collaborator-sponsored Phase 1/2 trial. Interim efficacy data from first five patients show systemic gene therapy effect across multiple tissues evaluated, including peripheral blood leukocytes, eyes, skin, gastrointestinal mucosa and neurocognitive system. Read full press release here\n\n\nReceived RPDD for AVR-RD-04 from FDA. Read full press release here\n\n\nAVR-RD-04 previously has been granted ODD in the U.S. and EU\n\n\nSubmitted a meeting request to the MHRA to discuss a company-sponsored clinical trial for AVR-RD-04 and expect a...