AVR-RD-01 / AVROBIO - LARVOL DELTA

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease (clinicaltrials.gov) - P1/2 | N=15 | Terminated | Sponsor: AVROBIO | N=11 ➔ 15

Enrollment change • Gene therapy • Viral vector • Fabry Disease • Gene Therapies • Genetic Disorders

Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201 (clinicaltrials.gov) - P=N/A | N=5 | Terminated | Sponsor: AVROBIO | Trial completion date: Dec 2036 ➔ Aug 2023 | Enrolling by invitation ➔ Terminated | Trial primary completion date: Dec 2036 ➔ Aug 2023; This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety or efficacy issue.

Gene therapy • Trial completion date • Trial primary completion date • Trial termination • Viral vector • Fabry Disease • Gene Therapies • Genetic Disorders

AVROBIO announces updated clinical data for AVR-RD-01 gene therapy in Fabry disease (GlobeNewswire) - P1, N=6; P2, N=12; NCT03454893; Sponsor: AVROBIO; "Preliminary safety data from these three subjects indicate AVR-RD-01 was generally well tolerated and no serious adverse events (SAEs) related to AVR-RD-01 have been reported (as of the safety data cut-off date of August 24, 2018)....Preliminary safety data from patient FAB-201-1 indicate that AVR-RD-01 was generally well tolerated; no SAEs related to AVR-RD-01 have been reported (as of the safety data cut-off date of August 28, 2018). Enrollment in the FAB-201 Study is ongoing....These interim results...are also scheduled to be presented at...CLSD...and...ESCGT..."

P1/2 data • Genetic Disorders

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease (clinicaltrials.gov) - P1/2 | N=11 | Terminated | Sponsor: AVROBIO | Trial completion date: Nov 2021 ➔ Mar 2022 | Recruiting ➔ Terminated | Trial primary completion date: Jul 2021 ➔ Mar 2022; AVROBIO is deprioritizing its Fabry disease program

Preclinical • Trial completion date • Trial primary completion date • Trial termination • Fabry Disease • Gene Therapies • Genetic Disorders

[VIRTUAL] AVRRD01, an investigational lentiviral gene therapy for Fabry disease: interim results from Phase 1 and Phase 2 studies (ESGCT 2021) - "This presentation will include updated safety data for all patients, including data on those patients in the Phase 2 trial conditioned with a precision dose of busulfan 90mg.h/L target concentration intervention (TCI). AVRRD01 has generally been well tolerated. Adverse events have been assessed as not related to the drug product but related to the conditioning regimen, underlying disease or preexisting conditions."

P1 data • P2 data • Fabry Disease • Gene Therapies • Genetic Disorders • Heart Failure • Nephrology

[VIRTUAL] Safety Profile of Conditioning Regimens of AVROBIO's Investigational ex vivo Lentiviral Gene Therapy for Fabry Disease (ESGCT 2021) - "In AVRRD01's clinical development program, two currently available conditioning agents, lowdose melphalan or busulfan (Bu90TCI), have each been used as single agents. Preliminary data suggest that use of both conditioning agents for gene therapy has predictable and welltolerated acute and subacute safety profile. Additional data, including ISA, will be presented."

Preclinical • Alopecia • Fabry Disease • Fatigue • Gene Therapies • Genetic Disorders • Hematological Disorders • Leukopenia • Neutropenia • Thrombocytopenia

Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201 (clinicaltrials.gov) - P; N=12; Enrolling by invitation; Sponsor: AvroBio

New trial • Fabry Disease • Gene Therapies • Genetic Disorders

"$AVRO anticipates initiating registration trial with kidney biopsy endpoint in mid-2022 to support potential full approval of AVR-RD-01" (@BioStocks)

Renal Disease

"$AVRO AVROBIO Provides Regulatory Update on Investigational AVR-RD-01 for Fabry Disease https://t.co/EwlMmKWxxY" (@stock_titan)

Fabry Disease • Genetic Disorders

[VIRTUAL] AVR-RD-01, an Investigational Lentiviral Gene Therapy for Fabry Disease: Clinical Data Trends from Phase 1 and Phase 2 Studies up to 3.5 Years (ASGCT 2021) - "Across all 4 patients enrolled to-date in Phase 2, cardiac mass and function remain stable at 1 year post AVR-RD-01 treatment.Overall, the therapy is well tolerated, and adverse events were consistent with the conditioning regimen, underlying FD and pre-existing conditions, and none were related to AVR-RD-01 drug product. The latest results from the ongoing Phase 1 and Phase 2 studies will be shared, as well as the results from the second evaluable kidney biopsy at 48 weeks post AVR-RD-01 treatment in the Phase 2 study."

Clinical data • P1 data • P2 data • Fabry Disease • Gene Therapies • Genetic Disorders • Heart Failure • Nephrology • Renal Disease

[VIRTUAL] AVR-RD-01, an Investigational Lentiviral Gene Therapy for Fabry Disease, Reduces Gb3 Substrate in Endothelial Cells of Renal Peritubular Capillaries (KIDNEY WEEK 2020) - "The latest results from this ongoing open label Phase 2 study will be shared. Funding: Commercial Support"

Fabry Disease • Gene Therapies • Genetic Disorders • Renal Disease • Transplantation

AVROBIO Reports Second Quarter 2020 Financial Results and Provides Business Update (Businesswire) - P1, N=5; "Phase 1 FACTs (Fabry disease Clinical research and Therapeutics) trial for Fabry disease: Five patients are participating in the fully enrolled Phase 1 investigator-led clinical trial. New data from these patients, now all out one year or more, up to 32 months post gene therapy, include: All five patients show sustained increased leukocyte and plasma enzyme activity....Overall, all patients in the Phase 1 trial who discontinued ERT after dosing with AVR-RD-01 remain off ERT."

P1 data • Fabry Disease • Genetic Disorders

AVROBIO Reports Second Quarter 2020 Financial Results and Provides Business Update (Businesswire) - P1/2, N=12; FAB-201 (NCT03454893); Sponsor: AvroBio; "Phase 2 FAB-201 clinical trial: Four patients have been dosed in the global Phase 2 FAB-201 trial evaluating treatment-naïve patients. New data from these patients, now six to 22 months post-gene therapy, include:...New eGFR data, which is a measure of kidney function, was stable across all patients in the trial up to 22 months following dosing....Anticipate patient dosing may resume incrementally, as allowed by hospitals and travel restrictions, but further delays are expected as sites remain impacted by COVID-19-related care."

P2 data • Trial status • Fabry Disease • Genetic Disorders

AVROBIO Announces New Patients Dosed in Gaucher Disease and Cystinosis Clinical Trials (Businesswire) - "AVROBIO, Inc....today announced that the first patient has been dosed in the company’s GuardOne clinical trial, a Phase 1/2 investigational study evaluating AVR-RD-02 for Gaucher disease type 1....The company’s Phase 1/2 trial of AVR-RD-02 for Gaucher disease type 1 is currently recruiting patients in Australia and Canada, with new clinical sites expected to open in the U.S. and Israel by year-end....The trial will include both patients who are treatment-naïve and who are on ERT....Patient recruitment activities for AVROBIO’s Phase 2 FAB-201 trial for Fabry disease continue for clinical trial sites in Australia, Canada and the U.S."

Enrollment status • Trial status • Fabry Disease • Gaucher Disease • Genetic Disorders • Type 1 Gaucher Disease

10th World Orphan Drug Congress (WODC) (November 12-14, 2019 - Barcelona, Spain). (PubMed, Drugs Today (Barc)) - "A co-conference on Cell and Gene Therapy was also organized. This rare disease conference addressed many different challenges in the field with numerous discussions on how to improve cross-border communications, how to better identify patients and shorten their diagnosis time using new tools such as artificial intelligence and machine learning, as well as how to improve usage of patient data and patient empowerment."

Journal • Orphan drug

Open-Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease (clinicaltrials.gov) - P1/2; N=12; Recruiting; Sponsor: AvroBio; Trial completion date: Dec 2020 ➔ Nov 2021; Trial primary completion date: Jun 2020 ➔ Jul 2021

Clinical • Trial completion date • Trial primary completion date

AVROBIO, Inc. reports first quarter 2019 financial results and provides business update (Businesswire) - "Completed enrollment in the investigator-sponsored FACTs Phase 1 study, and continued recruitment in AVROBIO’s FAB-201 Phase 2 trial. A total of seven patients have now been dosed across these two studies. Achieved U.S. Food and Drug Administration (FDA) clearance of AVROBIO’s Investigational New Drug (IND) application for AVR-RD-01."

Enrollment status • IND

AVROBIO, Inc (AVRO) announces FDA clearance of IND for AVR-RD-01 gene therapy for TDM (Streetinsider.com) - "AVROBIO...announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for AVR-RD-01, its gene therapy candidate for the treatment of Fabry disease. The Company now expects to move forward on two key initiatives in its Fabry clinical program: the incorporation of AVROBIO’s plato™ platform into its FAB-201 Phase 2 trial, and the dosing of patients at clinical sites in the U.S."

IND • New P2 trial

AVROBIO, Inc. reports fourth quarter and fiscal year 2018 financial results and provides business update (Businesswire) - "The AVR-RD-01 investigational gene therapy has been generally well tolerated with no serious adverse events (SAEs) related to the study drug, at up to 22- and 6-months follow-up in the Phase 1 and Phase 2 trials, respectively....AVROBIO received a no objection letter (NOL) from Health Canada to the clinical trial application (CTA) for GAU-201, its Phase 1/2 clinical trial of AVR-RD-02 in Gaucher disease."

Clinical • P1/2 data

AVROBIO, Inc. Announces Time Change for Analyst and Investor Presentation and Webcast, now 5:30 p.m. ET on Wednesday, February 6, 2019 (GlobeNewswire, AVROBIO, Inc.) - "AVROBIO...Phase 2 clinical-stage gene therapy company, today announced a time change for its analyst and investor presentation and webcast scheduled for Wednesday, February 6, 2019, at which time the Company will provide clinical trial updates related to its investigational gene therapy program for AVR-RD-01 in Fabry disease."

Clinical • P2 data

AVROBIO, Inc. announces updated clinical data from ongoing phase 1 and phase 2 studies for AVR-RD-01 gene therapy for Fabry disease (Businesswire) - "To date, six patients have been dosed in the AVR-RD-01 studies – four patients in the Phase 1 study and two patients in the Phase 2 study. Across both studies, four patients have reached the point of three-month data analysis or longer after receiving AVR-RD-01 – three patients in the Phase 1 study and one patient in the Phase 2 study....Preliminary safety data for all patients dosed in the Phase 1 and Phase 2 studies indicate that AVR-RD-01 has been generally well tolerated..."

Trial status

AVROBIO, Inc. announces time change for Analyst and Investor Presentation and webcast, now 5:30 p.m. ET on Wednesday, February 6, 2019 (GlobeNewswire) - "AVROBIO, Inc...announced a time change for its analyst and investor presentation and webcast scheduled for Wednesday, February 6, 2019, at which time the Company will provide clinical trial updates related to its investigational gene therapy program for AVR-RD-01 in Fabry disease. The Company’s presentation and webcast is now scheduled to start at 5:30 p.m. ET on Wednesday, February 6, 2019."

Clinical

AVROBIO, Inc. announces analyst and investor event and upcoming clinical data presentations at WORLDSymposium 2019 (GlobeNewswire) - "AVROBIO’s analyst and investor event will expand on the presentations at the conference and highlight AVROBIO’s progress with the Phase 1 study and Phase 2 FAB-201 study for its lead clinical program in Fabry disease; the Company’s optimized gene therapy platform; and other gene therapy pipeline programs in Gaucher, Cystinosis and Pompe."

Trial status