AVRO AVROBIO Inc.

16.94
-0.44  -3%
Previous Close 17.38
Open 17.35
52 Week Low 9.76
52 Week High 29.32
Market Cap $617,337,729
Shares 36,442,605
Float 31,006,322
Enterprise Value $388,992,474
Volume 93,787
Av. Daily Volume 288,090
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Drug Pipeline

Drug Stage Notes
AVR-RD-02
Gaucher disease
Phase 1/2
Phase 1/2
Phase 1/2 dosing commenced July 2020.
AVR-RD-04 (ASGCT)
Cystinosis
Phase 1/2
Phase 1/2
Phase 1/2 updated data presented at American Society of Gene & Cell Therapy (ASGCT) May 13, 2020.
AVR-RD-01 (FAB-201)
Fabry disease
Phase 1/2
Phase 1/2
Phase 2 trial ongoing.

Latest News

  1. New data indicate stable kidney function across the two Fabry disease clinical studies up to 32 months

    New six-month Phase 2 FAB-201 data from first patient treated using AVROBIO's plato® gene therapy platform show continued reduction in toxic metabolite plasma lyso-Gb3 and increased leukocyte and plasma enzyme activity

    Sustained clinical data across multiple other measures for Phase 1 and Phase 2 trials for Fabry disease

    First patient has been dosed in global Phase 1/2 clinical trial for Gaucher disease type 1 and second patient has been dosed in investigator-sponsored Phase 1/2 clinical trial for cystinosis

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic…

    New data indicate stable kidney function across the two Fabry disease clinical studies up to 32 months

    New six-month Phase 2 FAB-201 data from first patient treated using AVROBIO's plato® gene therapy platform show continued reduction in toxic metabolite plasma lyso-Gb3 and increased leukocyte and plasma enzyme activity

    Sustained clinical data across multiple other measures for Phase 1 and Phase 2 trials for Fabry disease

    First patient has been dosed in global Phase 1/2 clinical trial for Gaucher disease type 1 and second patient has been dosed in investigator-sponsored Phase 1/2 clinical trial for cystinosis

    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 second quarter ended June 30, 2020 and provided a business update.

    "We have continued to progress our clinical trials despite the challenges of the COVID-19 global pandemic and have now dosed 12 patients across four clinical trials, with initial data from the first patient dosed in our Gaucher program expected later this year," said Geoff MacKay, AVROBIO's president and CEO. "As we look at the new data that we released this quarter, we're pleased to see that longer-term measures suggest kidney function is stable across our Fabry trials. This is a critically important functional measure for people living with Fabry disease, as Fabry disease progression is characterized by a marked decrease in kidney function. We believe these clinically meaningful data further reinforce the potential of our single-dose investigational gene therapies to deliver a new standard of care for lysosomal disorders."

    Clinical Trials of AVR-RD-01 in Fabry Disease: Data Updates

    AVROBIO is conducting two clinical trials for its investigational gene therapy for Fabry disease, which continue to generate data relating to durability and tolerability with interim results that could support potential first-line use.

    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:

    • All four patients show sustained increased leukocyte and plasma enzyme activity, suggesting the continued production of an endogenous supply of functional alpha-galactosidase (AGA) enzyme.
    • Levels of the toxic metabolite plasma lyso-Gb3, a key biomarker for monitoring Fabry disease, have decreased 86, 49 and 59 percent from baseline for Patients 1, 3 and 4 at 22, 12 and six months, respectively. Patient 2, a cardiac variant who does not have classic Fabry disease, did have very low levels of plasma lyso-Gb3 at baseline and hence did not show a meaningful decrease in plasma lyso-Gb3 levels, as expected for such a variant.
    • A kidney biopsy was successfully obtained on Patient 3, but due to human error at the external laboratory vendor, appropriate handling of the biopsy failed and the kidney Gb3 inclusions could not be evaluated and will not be available. AVROBIO's Quality Assurance team has worked closely with the external vendor to identify the cause of the error and determine additional protocols for implementation by the vendor that are designed to prevent similar errors in the future.
    • Other data and important functional measures obtained from Patient 3 include:
      • Kidney function measures, including estimated glomerular filtration rate (eGFR) and measured glomerular filtration rate (mGFR), which indicated the patient's kidney function is stable compared to baseline, consistent with the other patients in the AVR-RD-01 Phase 1 and Phase 2 trials at the 48-week timepoint.
      • Cardiac function measures, including ejection fraction (EF), left ventricular (LV) mass and LV mass index, remain stable and in the normal range.
    • As previously reported, the kidney biopsy for Patient 1 in the trial showed an 87-percent reduction in Gb3 inclusions per peritubular capillary compared to baseline. Patient 2, a cardiac variant who does not have classic Fabry disease, did not have any Gb3 accumulated in the kidney at baseline and as such did not demonstrate any response in this endpoint, as expected for such a variant. The kidney biopsy for Patient 4 is expected in 1Q 2021.
    • Patient 4, now six months following dosing with drug product manufactured using AVROBIO's plato® gene therapy platform, showed a stable vector copy number (VCN) of 1.17 and AGA enzyme activity 5.1-fold higher in leukocytes and 3.7-fold higher in plasma compared to the mean activity level of the first three patients in the same trial at the same timepoint, who were treated using an academic platform.
    • New eGFR data, which is a measure of kidney function, was stable across all patients in the trial up to 22 months following dosing. eGFR is well documented in natural history studies to decline in classic male Fabry disease patients and we interpret the consistent emerging data set showing stable eGFR as positive.
    • 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. We have multiple patients identified as possible trial participants once hospitals and travel restrictions allow.

    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.
    • Toxic metabolite plasma lyso-Gb3 levels are lower or similar to the levels observed when the patient received only enzyme replacement therapy (ERT) prior to administration of AVR-RD-01. Plasma lyso-Gb3 levels for Patient 4, who discontinued ERT six months post gene therapy administration, showed a slight increase in plasma lyso-Gb3 levels but his lyso-Gb3 levels remain within range for Fabry disease patients on ERT observed in this study and he remains off ERT.
    • VCN across all patients is stable, with a range between 0.1 and 0.6, up to 32 months following dosing.
    • New eGFR data was stable up to 32 months following dosing across four patients in this trial. The other patient who entered the trial with moderate chronic kidney disease and an eGFR below 50 mL/min/1.73m2, has not stabilized his kidney function, which is to be expected given the low initial eGFR. He also remains on ERT.

    Overall, all patients in the Phase 1 trial who discontinued ERT after dosing with AVR-RD-01 remain off ERT.

    As of the most recent safety data cut-off date of April 23, 2020, there have been no serious adverse events (SAEs) attributed to the AVR-RD-01 drug product in either the Phase 1 or Phase 2 trial. Through the safety data cut-off date, four SAEs have been reported in the FAB-201 trial and two SAEs in the Phase 1 trial. Across both studies, each of the SAEs has been consistent with expectations of the stem cell mobilization, conditioning regimen, underlying disease or pre-existing conditions. Anti-AGA antibody titers have been observed in four patients in the Phase 1 trial and two patients in the Phase 2 trial. We believe none of these are of clinical significance.

    Slides with these new data can be found in the company's current corporate overview presentation here.

    Other Program Updates and Milestones

    AVR-RD-04 Phase 1/2 investigator-sponsored clinical trial for cystinosis

    AVROBIO's investigational gene therapy for cystinosis is being evaluated in a single-arm, Phase 1/2 trial sponsored by the University of California, San Diego (UCSD)1 and is expected to enroll up to six patients.

    • Patient 2 was dosed in June 2020.
    • Six-month data for both eGFR and serum creatinine measures from Patient 1 was presented at ASGCT in May 2020.
    • Patient recruitment activities continue.

    AVR-RD-02 Phase 1/2 trial for Gaucher disease

    AVROBIO's investigational gene therapy for Gaucher disease is being studied in the GuardOne clinical trial, a Phase 1/2 trial to evaluate the safety and efficacy in individuals with Gaucher disease type 1. The trial is expected to enroll eight to 16 patients between the ages of 18 and 35 who are treatment-naïve and on ERT.

    • Patient 1 was dosed in AVROBIO's global Phase 1/2 clinical trial in July 2020.
    • Subsequent new patient dosing and enrollment timelines have been impacted by the COVID-19 pandemic. However, patient recruitment activities continue for our clinical sites in Australia and Canada.
    • New clinical sites expected to open in the U.S. and Israel in the fourth quarter of the year.

    AVR-RD-03 preclinical program in Pompe disease

    AVROBIO's research program for Pompe disease, AVR-RD-03, is currently advancing a candidate through pre-clinical studies.

    • Data presented at ASGCT in May 2020 showed the ability of AVROBIO's optimized lentiviral vectors, in combination with a proprietary Glycosylation-Independent Lysosomal Targeting (GILT)-tag technology, to demonstrate significant glycogen reduction in both the muscle and central nervous system of a Pompe disease mouse model.
    • A preclinical Investigational New Drug (IND)-enabling proof-of-concept study is currently underway and expected to conclude in 2020.

    Business Updates

    • Kim Raineri has been appointed as chief manufacturing and technology officer (CMTO). Kim Warren, Ph.D., AVROBIO's founding CMTO, will retire at the end of July but will continue in a consultant role during the transition phase.
    • AVROBIO expects to host its first R&D Day virtually in 4Q 2020 and intends to provide an update on ongoing clinical programs, a review of other pipeline programs and an in-depth overview of its plato platform.

    Second Quarter 2020 Financial Results

    AVROBIO reported a net loss of $28.8 million for the second quarter of 2020 as compared to a net loss of $16.1 million for the comparable period in 2019. This increase was due to increased research and development expenses, as well as increased general and administrative expenses.

    Research and development expenses were $20.9 million for the second quarter of 2020 as compared to $12.3 million for the comparable period in 2019. This increase was driven by increased program development activities related to the advancement of the company's pipeline, as well as increased personnel-related costs resulting from an increase in employee headcount, which includes the impact of non-cash stock-based compensation.

    General and administrative expenses were $8.0 million for the second quarter of 2020 as compared to $4.3 million for the comparable period in 2019. This increase was primarily due to an increase in employee headcount, which includes the impact of non-cash stock-based compensation, as well as professional fees and consulting costs.

    As of June 30, 2020, AVROBIO had $244.4 million in cash and cash equivalents, as compared to $187.0 million in cash and cash equivalents as of December 31, 2019. Based on the company's current operating plan, AVROBIO expects its cash and cash equivalents as of June 30, 2020 will enable the company to fund its operating expenses and capital expenditure requirements into the second half of 2022.

    About AVROBIO

    Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato® gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains forward-looking statements, including 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 prospective product candidates; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals, including the expected timing of the kidney biopsy for the fourth patient in our Phase 2 FAB-201 clinical trial for AVR-RD-01; the timing of our ongoing preclinical studies, including our IND-enabling proof-of-concept study of AVR-RD-03 for Pompe disease; 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 implementation of the plato platform in our clinical trials and gene therapy programs; the expected safety profile of our investigational gene therapies; the potential impact of the COVID-19 outbreak on our clinical trial programs and business generally, as well as our plans and expectations with respect to the timing and resumption of any development activities that may be temporarily paused as a result of the COVID-19 outbreak; and statements regarding our financial and cash position and expected cash runway. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

    Any forward-looking statements in this press release are based on AVROBIO's 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 AVROBIO's product candidates will not be successfully developed or commercialized; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that AVROBIO 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 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 pre-clinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIO's 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 COVID-19 outbreak 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 materially and adversely 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.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (In thousands)

    (Unaudited)

     
     

    June 30,

     

    December 31,

     

    2020

     

    2019

         

    Cash and cash equivalents

     

    $

    244,380

     

    $

    187,043

    Prepaid expenses and other current assets

     

     

    6,640

     

     

    8,658

    Property and equipment, net

     

     

    3,580

     

     

    3,696

    Other assets

     

     

    968

     

     

    1,117

    Total assets

     

    $

    255,568

     

    $

    200,514

         

    Accounts payable

     

    $

    2,619

     

    $

    3,949

    Accrued expenses and other current liabilities

     

     

    12,792

     

     

    10,068

    Deferred rent, net of current portion

     

     

    370

     

     

    484

    Total liabilities

     

     

    15,781

     

     

    14,501

     

     

     

     

     

    Total stockholders' equity

     

     

    239,787

     

     

    186,013

    Total liabilities and stockholders' equity

     

    $

    255,568

     

    $

    200,514

         
       

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    (In thousands, except share and per share data)

    (Unaudited)

       
     

    Three Months Ended June 30,

    Six Months Ended June 30,

     

    2020

     

    2019

     

    2020

     

    2019

       

    Operating expenses:

     

     

    Research and development

     

    $

    20,866

    $

    12,267

    $

    39,140

    $

    24,713

    General and administrative

     

     

    7,991

     

     

    4,345

     

     

    16,306

     

     

    9,599

    Total operating expenses

     

     

    28,857

     

     

    16,612

     

     

    55,446

     

     

    34,312

     

     

     

     

     

     

     

     

    Loss from operations

     

     

    (28,857)

     

     

    (16,612)

     

     

    (55,446)

     

     

    (34,312)

    Total other income (expense), net

     

     

    29

     

     

    557

     

     

    645

     

     

    1,154

    Net loss

     

    $

    (28,828)

    $

    (16,055)

    $

    (54,801)

    $

    (33,158)

    Net loss attributable to common stockholders – basic and diluted

     

    $

    (28,828)

     

    $

    (16,055)

     

    $

    (54,801)

     

    $

    (33,158)

     

     

       

    Net loss per share attributable to common stockholders — basic and diluted

     

    $

    (0.80)

    $

    (0.67)

    $

    (1.57)

    $

    (1.38)

       

    Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted

     

     

    36,104,919

     

    24,046,262

     

    34,885,804

     

    23,985,717

    __________________

    1
    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).

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  2. AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced Geoff MacKay, president and CEO of AVROBIO, will present at the 2020 Wedbush PacGrow Healthcare Virtual Conference at 1:10 p.m. ET on Wednesday, Aug. 12, 2020.

    A live webcast of the presentation can be accessed under "Events and Presentations" in the Investors & Media section of the company's website at www.avrobio.com. An archived webcast recording of the presentation will be available on the website for approximately 30 days.

    About AVROBIO

    Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout…

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced Geoff MacKay, president and CEO of AVROBIO, will present at the 2020 Wedbush PacGrow Healthcare Virtual Conference at 1:10 p.m. ET on Wednesday, Aug. 12, 2020.

    A live webcast of the presentation can be accessed under "Events and Presentations" in the Investors & Media section of the company's website at www.avrobio.com. An archived webcast recording of the presentation will be available on the website for approximately 30 days.

    About AVROBIO

    Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato® gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains forward-looking statements, including 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 prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

    Any forward-looking statements in this press release are based on AVROBIO's 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 AVROBIO's product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO 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 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 AVROBIO's 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 COVID-19 outbreak 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 materially and adversely from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIO's most recent Annual or 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.

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  3. AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the company has granted non-statutory stock options for the purchase of up to an aggregate of 138,200 shares of the company's common stock to five new employees as inducement awards under the company's 2019 Inducement Plan, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock options were granted as inducements material to each new employee's acceptance of employment with the company and were approved by the Compensation Committee of the company's Board of Directors. The stock options were granted on July 1, 2020, with an exercise price of $17.83 per share, representing the…

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the company has granted non-statutory stock options for the purchase of up to an aggregate of 138,200 shares of the company's common stock to five new employees as inducement awards under the company's 2019 Inducement Plan, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock options were granted as inducements material to each new employee's acceptance of employment with the company and were approved by the Compensation Committee of the company's Board of Directors. The stock options were granted on July 1, 2020, with an exercise price of $17.83 per share, representing the closing price of AVROBIO's common stock as reported by Nasdaq on the grant date.

    Each stock option award has a 10-year term and vests over four years, with 25 percent of the original number of shares vesting on the first anniversary of the employee's new hire date and the remainder vesting in equal monthly installments over the following three years. Vesting of each option award is subject to continued service with AVROBIO by the employee through the applicable vesting dates.

    About AVROBIO

    Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato® gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains forward-looking statements, including 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 prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

    Any forward-looking statements in this press release are based on AVROBIO's 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 AVROBIO's product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO 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 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 AVROBIO's 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 COVID-19 outbreak 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 materially and adversely from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIO's most recent Annual or 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.

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  4. First patient dosed in AVROBIO's global Phase 1/2 clinical trial of AVR-RD-02 for Gaucher disease type 1

    Second patient dosed in investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, 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 also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.

    "The first patient dosed is an important milestone for the Gaucher…

    First patient dosed in AVROBIO's global Phase 1/2 clinical trial of AVR-RD-02 for Gaucher disease type 1

    Second patient dosed in investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, 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 also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.

    "The first patient dosed is an important milestone for the Gaucher disease community and our AVR-RD-02 program. Gaucher disease type 1 leads to an array of serious symptoms and the current standard of care does not halt disease progression," said Geoff MacKay, AVROBIO's president and CEO. "With a single dose of our investigational lentiviral gene therapy, we aim to prevent the buildup of a fatty substrate in specialist immune cells called macrophages, as well as debilitating symptoms throughout the body, including the brain."

    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.

    Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) – the current standard of care – people with Gaucher disease type 1 have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

    "While the current treatments for Gaucher disease -- enzyme replacement therapy and substrate reduction therapy -- have been life changing, many unmet needs remain that significantly impact the daily lives of patients and families living with Gaucher disease, including fatigue, severe bone pain, joint destruction, increased risk of developing Parkinson's disease and other co-morbidities," said Christine White, executive director, National Gaucher Foundation of Canada. "We welcome clinical trials of new therapeutics that have the potential to stop the progression of Gaucher disease and are excited to learn more about the potential use of this lentiviral gene therapy."

    The Phase 1/2 trial of AVR-RD-02 for Gaucher disease type 1 is designed to evaluate the safety and efficacy of the investigational gene therapy and is expected to enroll eight to 16 patients between the ages of 18 and 35. AVR-RD-02 starts with the patient's own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. The trial will include both patients who are treatment-naïve and who are on ERT. Every patient in this trial will be treated using the plato® gene therapy platform, AVROBIO's foundation designed to scale gene therapy worldwide.

    Second patient dosed in cystinosis clinical trial

    The second patient has been dosed in the company's AVR-RD-04 investigational gene therapy program for cystinosis. The ongoing Phase 1/2 clinical trial is sponsored by the company's academic collaborators at the University of California, San Diego (UCSD)1 and is led by Stephanie Cherqui, Ph.D., associate professor of pediatrics at UCSD.

    Cystinosis is a progressive disease marked by the accumulation of cystine in cellular organelles known as lysosomes. This buildup can cause debilitating symptoms including kidney failure, corneal damage and thyroid dysfunction, often leading to a shortened lifespan. Currently, more than 90 percent of treated cystinosis patients require a kidney transplant in the second or third decade of life. The current standard of care for cystinosis is cysteamine, a burdensome treatment regimen that can require dozens of pills per day and may not prevent overall progression of the disease.

    The Phase 1/2 clinical trial is evaluating the safety and efficacy of AVR-RD-04 in patients at least 18 years of age who are currently being treated with cysteamine. The trial will enroll up to six patients. AVR-RD-04 starts with the patient's own hematopoietic stem cells, which are genetically modified to produce functional cystinosin, the protein that is deficient in cystinosis.

    Patient recruitment activities for Fabry Phase 2 trial ongoing

    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. While clinical trial sites are starting to reopen and patient identification activities are ongoing with a number of potential new patients identified, activities related to new patient screening, consent and enrollment in the FAB-201 clinical trial have been slowed because of the COVID-19 pandemic.

    AVROBIO is conducting two clinical trials for its AVR-RD-01 investigational gene therapy for Fabry disease. Four patients have been dosed in the global Phase 2 trial (FAB-201), which is evaluating treatment-naïve patients, and five patients are participating in the fully enrolled Phase 1 investigator-led clinical trial, known as FACTs.

    AVR-RD-01 starts with the patient's own hematopoietic stem cells, which are genetically modified to produce functional alpha-galactosidase A, the enzyme that is deficient in Fabry disease. People with the disease experience a toxic buildup of a complex cell lipid called globotriaosylceramide (Gb3 or GL3), which can damage tissues throughout the body and brain, and cause the progressive signs and symptoms of Fabry disease.

    About AVROBIO's personalized gene therapy approach

    Our investigational lentiviral gene therapies start with the patient's own hematopoietic stem cells. We use a lentiviral vector to transduce those cells in order to insert a therapeutic gene designed to enable the patient to produce a supply of the functional protein they lack. These cells are then infused back into the patient, where they are expected to engraft in the bone marrow and produce generations of daughter cells, each containing a copy or copies of the therapeutic gene. To optimize engraftment, we use a personalized conditioning regimen with precision dosing of busulfan to make space and enable durable engraftment in the patient's bone marrow and central nervous system (CNS). Busulfan is an extensively validated conditioning agent generally considered to be the gold standard for ex vivo lentiviral gene therapy and has been administered to hundreds of patients for this purpose. Our approach is designed to drive durable production of the functional protein throughout the patient's body, thereby potentially addressing symptoms from "head to toe," including those originating in the CNS.

    About lentiviral gene therapy

    Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patient's chromosomes. This integration is designed to maintain the therapeutic gene's presence as the patient's cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated using the vector into patients' own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the viral vector.

    About AVROBIO

    Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato® gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

    Forward-Looking Statement

    This press release contains forward-looking statements, including 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 prospective product candidates, including the ability to stop disease progression, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including new patient screening, consent and enrollment activities in our FAB-201 clinical trial, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

    Any forward-looking statements in this press release are based on AVROBIO's 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 AVROBIO's product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned preclinical or clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO 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 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 AVROBIO's 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 COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that patient enrollment and/or 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 materially and adversely 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.

    1 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).

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  5. AVROBIO co-founder and veteran gene and cell therapy pioneer Kim Warren to retire July 31, 2020

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced the appointment of Kim Raineri, as chief manufacturing and technology officer. He brings deep global experience in the cell and gene therapy industry, with a distinguished track record of innovation and implementation of Good Manufacturing Practices (GMP).

    AVROBIO's founding chief manufacturing and technology officer, Kim Warren, Ph.D., will be retiring at the end of July. Among many other accomplishments, she led the development of plato®, the company's proprietary gene therapy platform designed…

    AVROBIO co-founder and veteran gene and cell therapy pioneer Kim Warren to retire July 31, 2020

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced the appointment of Kim Raineri, as chief manufacturing and technology officer. He brings deep global experience in the cell and gene therapy industry, with a distinguished track record of innovation and implementation of Good Manufacturing Practices (GMP).

    AVROBIO's founding chief manufacturing and technology officer, Kim Warren, Ph.D., will be retiring at the end of July. Among many other accomplishments, she led the development of plato®, the company's proprietary gene therapy platform designed to bring gene therapy to patients worldwide through an efficient, automated, closed manufacturing system developed to be rapidly deployed to contracted manufacturing sites.

    "We have made it a priority to build leadership in gene therapy manufacturing by integrating and optimizing pioneering technologies as part of our plato platform. As we continue to implement our global clinical trials and prepare for eventual commercial scale, we are delighted to bring Kim Raineri on board. His expertise in global GMP for cell and gene therapy will be highly valuable in keeping AVROBIO at the forefront of this industry," said Geoff MacKay, AVROBIO's president and CEO. "I also would like to thank Kim Warren for her integral contributions to AVROBIO these past five years. As one of my co-founders, Kim has played a key role in building the company and establishing plato as an unrivaled lentiviral gene therapy platform."

    "I am thrilled to join AVROBIO, a leader in lentiviral gene therapy and a true pioneer in driving manufacturing advances that address the gene therapy field's need for faster, more scalable and more automated production," said Raineri. "The AVROBIO team has created a state-of-the-art gene therapy platform and is clearly committed to continuous innovation on behalf of the patient communities they strive to serve. I am excited to contribute to that work."

    Raineri has broad, global experience in GMP operations, including in the cell and gene therapy, biologics and medical device spaces. Prior to joining AVROBIO, he served as the vice president of operations for Nikon CeLL innovation Co., Ltd, a Japanese contract development and manufacturing organization. During his tenure, he established the company as the preferred provider of custom process development and manufacturing services for cell and gene therapy products in the Japanese market. Previously, Raineri held management positions at Lonza, serving as the business director for the cell therapy contract manufacturing operations in Singapore for five years, and prior to that as director of operations at Lonza's Maryland facilities. Raineri was also previously the senior manager of the Tissue Processing Lab at CryoLife Inc. He holds a B.S. from the University of Miami and an MBA from Kennesaw State University.

    About AVROBIO

    Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato® gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

    Forward-Looking Statement

    This press release contains forward-looking statements, including 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 the expected benefits from the appointment of Mr. Raineri to the position of chief manufacturing and technology officer, the expected benefits, timing and results of our implementation of the plato platform in our clinical trials and gene therapy programs, as well as future innovations to our plato platform and other gene therapy manufacturing technologies.

    Any forward-looking statements in this press release are based on AVROBIO's 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 AVROBIO's product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned preclinical or clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO 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 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 AVROBIO's 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 COVID-19 outbreak 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 materially and adversely 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.

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