1. Homepage
  2. Equities
  3. United States
  4. Nasdaq
  5. AVROBIO, Inc.
  6. News
  7. Summary
    AVRO   US05455M1009


Delayed Nasdaq  -  04:00 2022-08-05 pm EDT
0.9900 USD   +2.52%
08/04AVROBIO Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
08/01AVROBIO to Participate in Two Upcoming Investor Conferences in August
07/13Avrobio Gets US FDA's Orphan Drug Designation for AVR-RD-05 for Treatment of Hunter Syndrome
SummaryMost relevantAll NewsAnalyst Reco.Other languagesPress ReleasesOfficial PublicationsSector news

AVROBIO : Corporate Presentation June 2022

06/30/2022 | 07:13am EDT

Corporate Presentation

J U N E 2 0 2 2


This presentation has been prepared by AVROBIO, Inc. ("AVROBIO") for informational purposes only and not for any other purpose. Certain information contained in this presentation and statements made orally during this presentation relate to or are based on studies, publications, surveys and other data obtained from third-party sources and AVROBIO's own internal estimates and research. While AVROBIO believes these third-party sources to be reliable as of the date of this presentation, it has not independently verified, and AVROBIO makes no representation as to the adequacy, fairness, accuracy or completeness of any information obtained from third-party sources. While AVROBIO believes its internal research is reliable, such research has not been verified by any independent source.

This presentation may contain forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as "aims," "anticipates," "believes," "could," "designed to," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our current and prospective product candidates; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; our plans and expectations with respect to the development of our clinical and preclinical product candidates, including timing, design and initiation of our potential clinical and registration trials and anticipated interactions with regulatory agencies; the timing of anticipated clinical and regulatory updates; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the timing and results of our ongoing preclinical studies; the anticipated benefits of our gene therapy

platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our manufacturing technology, including the implementation of our plato® platform in our clinical trials and gene therapy programs; the expected safety profile of our investigational gene therapies; and our financial position and cash runway expectations. Any such statements in this presentation that are not statements of historical fact may be deemed to be forward-looking statements.

Any forward-looking statements in this presentation are based on our current expectations, estimates and projections about our industry as well as management's current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of our product candidates will not be successfully developed or commercialized; the risk that regulatory agencies may disagree with our anticipated development approach for any one or more of our product candidates; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that we may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that we may not realize the intended benefits of our gene therapy platform, including the features of our plato® platform; the risk that our product candidates or procedures in connection with the administration thereof, including our use of busulfan as a conditioning agent or potential use of monoclonal antibody conditioning agents, will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving our product candidates; the risk that we will be unable to obtain and maintain

regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for additional financing; risks relating to clinical trial and business interruptions resulting from the ongoing COVID-19 pandemic or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIO's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIO's most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIO's subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Note regarding trademarks: plato® is a registered trademark of AVROBIO. Other trademarks referenced in this presentation are the property of their respective owners.

Copyright© 2022 AVROBIO, Inc. All rights reserved.




Freedom from a lifetime

Bring personalized gene

of genetic disease.

therapy to the world.


Investment highlights

Leading hematopoietic stem cell (HSC) gene therapy company

Targeting lysosomal disorders representing a multi-billion dollar revenue opportunity

Strong efficacy and safety profile to date across two clinical-stage programs

Regulatory discussions planned for 2H 2022 to frame approval pathways for multiple indications

Strong balance sheet with cash runway into Q1 2024


Near-term opportunities in leading gene therapy pipeline

Potential billion-dollar revenue opportunities

AVR-RD-04 for cystinosis

    • First and only gene therapy for cystinosis in clinic
    • Proof-of-conceptdemonstrated in adults
    • Secured U.S./EU Orphan Disease Designation and U.S. Fast Track Designation
  • Plan to meet with regulators in 2H 2022 to discuss company-sponsored trial
  • Plan to initiate company-sponsored trial in 2023

AVR-RD-02* for Gaucher disease type 3

    • Second program in Gaucher disease franchise
    • Leverages clinical and CMC work conducted in Gaucher disease type 1, which was first gene therapy for Gaucher to enter clinical trials
  • Plan to engage with regulators on potential Phase 2/3 trial in 2H 2022
  • Plan to initiate potential Phase 2/3 trial in 2023

Other anticipated 2022 catalysts:

  • AVR-RD-02for Gaucher disease type 1 - planned clinical update
  • AVR-RD-05for Hunter syndrome - CTA authorization expected
  • AVR-RD-03for Pompe disease - engage with regulators on clinical trial

Planned regulatory milestones subject to regulatory agency clearance; * Formerly referred to as AVR-RD-06;collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH)


This is an excerpt of the original content. To continue reading it, access the original document here.


AVROBIO Inc. published this content on 30 June 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 30 June 2022 11:12:05 UTC.

© Publicnow 2022
All news about AVROBIO, INC.
08/04AVROBIO Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
08/01AVROBIO to Participate in Two Upcoming Investor Conferences in August
07/13Avrobio Gets US FDA's Orphan Drug Designation for AVR-RD-05 for Treatment of Hunter Syn..
07/13AVROBIO Receives Orphan Drug Designation from the U.S. Food and Drug Administration for..
07/07AVROBIO Announces Inducement Grants Under Nasdaq Listing Rule 5635(4)
07/06AVROBIO Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
06/30AVROBIO : Corporate Presentation June 2022
06/29AVROBIO, INC. : Change in Directors or Principal Officers, Regulation FD Disclosure, Finan..
06/24AVROBIO, INC.(NASDAQGS : AVRO) dropped from Russell 2500 Value Index
06/24AVROBIO, INC.(NASDAQGS : AVRO) dropped from Russell 2500 Index
More news
Analyst Recommendations on AVROBIO, INC.
More recommendations
Financials (USD)
Sales 2022 - - -
Net income 2022 -109 M - -
Net cash 2022 78,7 M - -
P/E ratio 2022 -0,44x
Yield 2022 -
Capitalization 42,3 M 42,3 M -
EV / Sales 2022 -
EV / Sales 2023 -
Nbr of Employees 122
Free-Float 97,3%
Duration : Period :
AVROBIO, Inc. Technical Analysis Chart | MarketScreener
Full-screen chart
Technical analysis trends AVROBIO, INC.
Short TermMid-TermLong Term
Income Statement Evolution
Mean consensus OUTPERFORM
Number of Analysts 9
Last Close Price 0,99 $
Average target price 4,57 $
Spread / Average Target 362%
EPS Revisions
Managers and Directors
Geoff MacKay President, Chief Executive Officer & Director
Erik John Ostrowski Chief Financial Officer & Treasurer
Bruce Lee Booth Chairman
Kim Raineri Chief Manufacturing & Technical Operations Officer
Essra Ridha Chief Medical Officer
Sector and Competitors
1st jan.Capi. (M$)
AVROBIO, INC.-74.29%42
GILEAD SCIENCES, INC.-15.87%76 626
BIONTECH SE-28.97%44 499
WUXI APPTEC CO., LTD.-19.26%41 093