AVRO AVROBIO Inc.

13.8
+0.03  (+0%)
Previous Close 13.77
Open 13.96
52 Week Low 9.76
52 Week High 29.32
Market Cap $572,023,110
Shares 41,450,950
Float 36,014,667
Enterprise Value $351,233,581
Volume 617,247
Av. Daily Volume 260,744
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Drug Pipeline

Drug Stage Notes
AVR-RD-02
Gaucher disease
Phase 1/2
Phase 1/2
Phase 1/2 trial ongoing.
AVR-RD-01 (FAB-201)
Fabry disease
Phase 1/2
Phase 1/2
Phase 2 trial ongoing.
AVR-RD-04
Cystinosis
Phase 1/2
Phase 1/2
Phase 1/2 trial ongoing - third patient has been dosed November 2020.
AVR-RD-05
Hunter syndrome / MPS II
Phase 1/2
Phase 1/2
Phase 1/2 trial to commence 2H 2021.

Latest News

  1. AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company, today announced the closing of its previously announced underwritten public offering of 5,000,000 shares of its common stock. The aggregate gross proceeds to the company from the offering, before deducting underwriting discounts and commission and estimated offering expenses, totaled $75 million. As previously announced, the company has granted the underwriters a 30-day option to purchase up to 750,000 additional shares of its common stock at the public offering price, less underwriting discounts and commissions.

    Morgan Stanley, Cowen, Wells Fargo Securities and Barclays acted as joint book-running managers for the offering, and Wedbush PacGrow acted as co-manager…

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company, today announced the closing of its previously announced underwritten public offering of 5,000,000 shares of its common stock. The aggregate gross proceeds to the company from the offering, before deducting underwriting discounts and commission and estimated offering expenses, totaled $75 million. As previously announced, the company has granted the underwriters a 30-day option to purchase up to 750,000 additional shares of its common stock at the public offering price, less underwriting discounts and commissions.

    Morgan Stanley, Cowen, Wells Fargo Securities and Barclays acted as joint book-running managers for the offering, and Wedbush PacGrow acted as co-manager.

    AVROBIO intends to use the net proceeds from the offering, in addition to its existing cash resources, to fund its current programs in Fabry disease, cystinosis, Gaucher disease type 1, Hunter syndrome, Pompe disease and Gaucher disease type 3, fund external and internal manufacturing and process development activities and fund research and development activities that relate to its current and future clinical and preclinical activities, including the cost of research and development personnel. The company intends to use the remainder for planned general and administrative expenses, working capital and other general corporate purposes.

    The securities described above were offered pursuant to a shelf registration statement on Form S-3 (File No. 333-235641), including a base prospectus. The securities were offered only by means of a prospectus. A final prospectus supplement related to the offering was filed with the Securities and Exchange Commission (SEC) on November 20, 2020 and is available on the SEC's website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to the securities being offered may also be obtained by contacting: Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014, or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About AVROBIO

    AVROBIO, Inc. is a leading clinical-stage gene therapy company. Our clinical-stage programs include Fabry disease, Gaucher disease type 1 and cystinosis and we also are advancing preclinical programs in Hunter syndrome, Pompe disease and Gaucher disease type 3.

    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 the anticipated use of proceeds from the public offering. 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 only as of the date of this release 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 development of the company's current and future product candidates and the use of proceeds from the public offering to support such development. 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and our subsequent periodic reports on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our other filings with the SEC, including those contained or incorporated by reference in the preliminary prospectus supplement related to the public offering filed with the SEC. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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  2. AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company, today announced the pricing of an underwritten public offering of 5,000,000 shares of its common stock at a public offering price of $15.00 per share. Gross proceeds from the underwritten public offering will total approximately $75 million, before deducting underwriting discounts and commissions and other offering expenses payable by the company. In addition, the company has granted the underwriters a 30-day option to purchase up to 750,000 additional shares of its common stock at the public offering price, less underwriting discounts and commissions. All of the shares in the offering are to be sold by the company. The offering is expected to close on or about Nov…

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company, today announced the pricing of an underwritten public offering of 5,000,000 shares of its common stock at a public offering price of $15.00 per share. Gross proceeds from the underwritten public offering will total approximately $75 million, before deducting underwriting discounts and commissions and other offering expenses payable by the company. In addition, the company has granted the underwriters a 30-day option to purchase up to 750,000 additional shares of its common stock at the public offering price, less underwriting discounts and commissions. All of the shares in the offering are to be sold by the company. The offering is expected to close on or about Nov. 24, 2020, subject to customary closing conditions.

    Morgan Stanley, Cowen, Wells Fargo Securities and Barclays are acting as joint book-running managers for the offering, and Wedbush PacGrow is acting as co-manager.

    AVROBIO intends to use the net proceeds from the offering, in addition to its existing cash resources, to fund its current programs in Fabry disease, cystinosis, Gaucher disease type 1, Hunter syndrome, Pompe disease and Gaucher disease type 3, fund external and internal manufacturing and process development activities and fund research and development activities that relate to its current and future clinical and preclinical activities, including the cost of research and development personnel. The company intends to use the remainder for planned general and administrative expenses, working capital and other general corporate purposes.

    The securities described were offered pursuant to a shelf registration statement on Form S-3 (File No. 333-235641), including a base prospectus. The securities were offered only by means of a prospectus. A preliminary prospectus supplement related to the offering was filed with the Securities and Exchange Commission (SEC) on Nov. 19, 2020, and is available on the SEC's website at www.sec.gov. A final prospectus supplement will be filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus relating to the securities being offered may also be obtained by contacting: Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014, or Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About AVROBIO

    AVROBIO, Inc. is a leading clinical-stage gene therapy company. Our clinical-stage programs include Fabry disease, Gaucher disease type 1 and cystinosis and we also are advancing preclinical programs in Hunter syndrome, Pompe disease and Gaucher disease type 3.

    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 completion, timing and anticipated size of the offering and the anticipated use of proceeds therefrom. 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 only as of the date of this release 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, completion of the proposed public offering on the anticipated terms, or at all, market conditions and the satisfaction of customary closing conditions related to the proposed public offering. 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and our subsequent periodic reports on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our other filings with the SEC, including those contained or incorporated by reference in the preliminary prospectus supplement related to the public offering filed with the SEC. 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, today announced that it intends to offer and sell, subject to market and other conditions, $75 million of its common stock in an underwritten public offering.

    The company expects to grant the underwriters a 30-day option to purchase up to $11.25 million of additional shares of its common stock (15 percent) offered in the public offering. All of the shares in the proposed offering are to be sold by the company. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    Morgan Stanley, Cowen, Wells Fargo Securities and Barclays are acting…

    AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company, today announced that it intends to offer and sell, subject to market and other conditions, $75 million of its common stock in an underwritten public offering.

    The company expects to grant the underwriters a 30-day option to purchase up to $11.25 million of additional shares of its common stock (15 percent) offered in the public offering. All of the shares in the proposed offering are to be sold by the company. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    Morgan Stanley, Cowen, Wells Fargo Securities and Barclays are acting as joint book-running managers for the offering.

    AVROBIO intends to use the net proceeds from the offering, in addition to its existing cash resources, to fund its current programs in Fabry disease, cystinosis, Gaucher disease type 1, Hunter syndrome, Pompe disease and Gaucher disease type 3, fund external and internal manufacturing and process development activities and fund research and development activities that relate to its current and future clinical and preclinical activities, including the cost of research and development personnel. The company intends to use the remainder for planned general and administrative expenses, working capital and other general corporate purposes.

    The securities described may be offered pursuant to a shelf registration statement on Form S-3 (File No. 333-235641), including a base prospectus. The securities may be offered only by means of a prospectus. A preliminary prospectus supplement related to the offering and a final prospectus supplement related to the offering will be filed with the U.S. Securities and Exchange Commission (SEC) and will be available on the SEC's website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to the securities being offered may also be obtained by contacting: Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014, or Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About AVROBIO

    AVROBIO, Inc. is a leading clinical-stage gene therapy company. Our clinical-stage programs include Fabry disease, Gaucher disease type 1 and cystinosis and we also are advancing preclinical programs in Hunter syndrome, Pompe disease and Gaucher disease type 3.

    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 completion, timing and anticipated size of the proposed offering and the anticipated use of proceeds therefrom. 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 only as of the date of this release 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, completion of the proposed public offering on the anticipated terms, or at all, market conditions and the satisfaction of customary closing conditions related to the proposed public offering. 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and our subsequent periodic reports on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our other filings with the SEC, including those contained or incorporated by reference in the preliminary prospectus supplement related to the proposed public offering to be filed with the SEC. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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  4. Three months post-gene therapy, first patient in Gaucher trial shows reductions in the toxic metabolite plasma lyso-Gb1 and plasma chitotriosidase as compared to baseline when the patient was on ERT

    Ongoing Fabry disease trials continue to demonstrate sustained durability, with first patient out 3.5 years

    One year post-gene therapy, first patient in cystinosis trial remains off cysteamine, with positive data across multiple measures, including substantial reduction in cystine crystals in cornea

    Clinical trial recruitment gaining momentum with five new patients expected to be dosed, enrolled or consented in 4Q 2020

    Gaucher disease type 3 program added to pipeline; recently added Hunter syndrome program planned to enter clinic next year

    Three months post-gene therapy, first patient in Gaucher trial shows reductions in the toxic metabolite plasma lyso-Gb1 and plasma chitotriosidase as compared to baseline when the patient was on ERT

    Ongoing Fabry disease trials continue to demonstrate sustained durability, with first patient out 3.5 years

    One year post-gene therapy, first patient in cystinosis trial remains off cysteamine, with positive data across multiple measures, including substantial reduction in cystine crystals in cornea

    Clinical trial recruitment gaining momentum with five new patients expected to be dosed, enrolled or consented in 4Q 2020

    Gaucher disease type 3 program added to pipeline; recently added Hunter syndrome program planned to enter clinic next year

    Virtual R&D Day to be webcast today starting at 9 a.m. ET

    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 positive new data across its clinical programs in Gaucher disease type 1, Fabry disease and cystinosis, further reinforcing the potential of ex vivo lentiviral gene therapy for lysosomal disorders. Additionally, AVROBIO is further expanding its lysosomal disorder pipeline with a new program in Gaucher disease type 3, which joins the recently announced program in Hunter syndrome in a synergistic portfolio of six programs designed to prevent, halt or reverse genetic disease.

    "We're delighted to report substantial new data across our three clinical programs. Three months post-gene therapy infusion, the first Gaucher disease patient's levels of the toxic metabolite plasma lyso-Gb1, as well as plasma chitotriosidase, were lower than the baseline levels when the patient was still on enzyme replacement therapy (ERT). With our Fabry disease data continuing to reflect sustained and durable results, with our first patient now out 3.5 years from dosing, we are planning our strategy to seek accelerated approvals in one or more major markets," said Geoff MacKay, president and CEO of AVROBIO. "Additionally, the first patient in the investigator-sponsored trial for cystinosis, now out one year, remains off both oral and eye drop cysteamine and we are thrilled to announce that a third patient has been dosed.

    "As we move into the next stage of company growth, we're expanding our lysosomal disorder pipeline with a new program for Gaucher disease type 3 and we plan to dose the first Hunter syndrome patient next year. We expect to be the first lentiviral gene therapy to the clinic across all six of these indications – and in some cases, the first to be in the clinic with an investigational gene therapy of any type. We believe the new data we've announced today help de-risk our portfolio which leverages the same lentiviral gene therapy approach across indications," MacKay added. "With strong clinical trial enrollment momentum coming out of the COVID-19-related slowdown, we anticipate dosing, enrolling or consenting five patients across our clinical trials this quarter, and dosing a total of 30 patients cumulatively across our clinical programs by the end of 2021."

    Positive clinical data out as far as 3.5 years across a broad lysosomal disorder gene therapy pipeline

    New clinical updates announced today include:

    • AVR-RD-02 for Gaucher disease type 1: Positive early reductions in plasma lyso-Gb1 and chitotriosidase activity at three months as compared to baseline, when Patient 1 was on ERT; additional positive trends observed across multiple other measures

      Three months post-gene therapy, the first patient dosed had a 22-percent reduction in the toxic metabolite plasma lyso-Gb1, a sensitive and clinically validated biomarker for Gaucher disease, compared to a baseline taken when she was stable on ERT, the current standard of care. Additionally, she had a 17-percent drop from her ERT baseline in plasma chitotriosidase, a biomarker of activated macrophages or "Gaucher cells" which lead to inflammation and severe organ damage. The vector copy number (VCN) at three months was 0.6 vcn/dg. Additionally, hemoglobin concentration and platelet counts, which are typically low in Gaucher disease patients, remained in the normal range three months after gene therapy. Patient 1 discontinued ERT one month before the gene therapy infusion and remains off ERT.



      At three months post-gene therapy, no unexpected safety events or trends have been identified in the trial, with no serious adverse events related to AVR-RD-02 reported in the first patient dosed as of the safety data cut-off date, Nov. 3, 2020.



    • AVR-RD-01 for Fabry disease: Potential accelerated approval strategy planning underway as clinical data across Phase 1 and Phase 2 trials continue to show positive and durable clinical activity and safety data

      With data from both trials showing consistently favorable results up to 3.5 years post-gene therapy, AVROBIO is in advanced planning of its strategy toward potential accelerated approval pathways. The company intends to submit its briefing book in 4Q 2020 to the U.S. Food and Drug Administration (FDA) with the goal to align on a potential accelerated approval strategy.



      The company reported durable and sustained response in enzyme activity, substrate levels and VCN across patients in both the Phase 1 and Phase 2 trials as of the data cut-off date, indicating successful engraftment of genetically modified cells and endogenous production of the functional enzyme needed to break down toxic substrate and metabolites in patients. Updated biomarker data on kidney function show generally stable estimated glomerular filtration rate (eGFR) in both Phase 1 and Phase 2 patients. Historically, people living with Fabry disease experience a progressive, faster-than-normal rate of decline in kidney function, as measured by eGFR, whether or not they are on ERT, the current standard of care. AVROBIO believes the stability in eGFR for patients in its clinical trials to be clinically significant and relevant.



      No unexpected safety events or trends have been identified in the trials as of the safety data cut-off date, Oct. 8, 2020. The eight serious adverse events reported in the two Fabry disease trials have been consistent with the conditioning regimen, stem cell mobilization, underlying disease or pre-existing conditions. Pre-existing low anti-AGA antibody titers have been detected in four patients in the Fabry Phase 1 trial and a transient low titer was observed but not detectable in subsequent measures in one patient in the Fabry Phase 2 trial.



    • AVR-RD-04 for cystinosis: Functional and clinical improvements for the first patient at 1 year; third patient in the trial dosed

      The first patient dosed in the Phase 1/2 trial, now 12 months post-dosing, remains off both oral and eye drop cysteamine. Biopsy data showed a 56-percent decrease in the number of crystals in his skin, suggesting that the patient may now be producing his own functional cystinosin protein – which he was unable to do before receiving AVR-RD-04 – and that the protein is potentially preventing the toxic accumulation of cystine crystals. Images of the patient's cornea also showed a substantial decline in corneal crystals. His eGFR has been stabilizing post-infusion, though it is important to note that he had pre-existing, irreversible chronic kidney disease prior to trial enrollment. VCN has reached its therapeutic plateau, as expected, measuring 0.9 vcn/dg at 12-months post-dosing, mirroring trends seen in AVROBIO's other clinical programs. Patient 2 had a VCN of 2.2 vcn/dg at three months post-gene therapy, as of Oct. 13, 2020. A new patient has been dosed this month in the investigator-sponsored1 study of AVR-RD-04 for cystinosis, marking the halfway point for enrollment with three patients dosed in total.



      No unexpected safety events or trends have been identified in the trial, with no serious adverse events reported as of the Nov. 2, 2020, safety data cut-off date.

    Pioneering approach to personalized conditioning to leverage advantages of busulfan

    The company also shared new data on the safety and tolerability profile of precision conditioning with busulfan prior to gene therapy administration. AVROBIO is pioneering a new approach called targeted concentration intervention (TCI) that enables precise dosing designed to optimize engraftment durability and head-to-toe reach of ex vivo lentiviral gene therapies. TCI aims to maximize the likelihood of engraftment while minimizing the risk of out-of-range side effects by targeting busulfan exposure to an area under the curve of 90 mg x hr/L over four days, called Bu90-TCI.

    In AVROBIO's clinical trials to date, data suggest that side effects from its single-agent, single-cycle approach to Bu90-TCI conditioning may be predictable, manageable and transient. The side effects have tended to be mild to moderate in nature and typically presented one week after dosing and peak over three to four days before quickly subsiding. Unlike other conditioning agents, Bu90-TCI is lymphocyte sparing, meaning that important components of the adaptive immune system, B and T cells, are expected to be minimally affected.

    Strategic pipeline expansion into relentlessly progressive lysosomal disorders

    AVROBIO announced the addition of Gaucher disease type 3 to its pipeline, following the recent addition of Hunter syndrome, which is planned to enter the clinic next year. Together with the existing program in Pompe disease, these make up AVROBIO's second wave of clinical programs focused on life-threatening lysosomal disorders, with the goals of preventing the central nervous system and systemic deterioration that make lysosomal disorders so devastating, normalizing lifespan and lifting the burden of chronic treatment with ERT. New preclinical data suggest that AVROBIO's proprietary tagging technology, part of its industry-leading plato® gene therapy platform toolbox, further enhances the potential of its investigational gene therapies in these disorders.

    "We believe that all six of our pipeline programs share tremendous synergies in clinical development, manufacturing, regulatory processes and commercialization. This second wave of programs will evaluate our promising investigational therapies in diseases with high unmet medical need for patients and families," said Chris Mason, M.D., Ph.D., chief scientific officer at AVROBIO. "We believe the opportunity we have to potentially prevent patients, especially children, from developing the disabilities that would otherwise result from their inherited genetic code – to perhaps give them the possibility of a full and healthy life – is humbling. That is our purpose; it drives all of us at AVROBIO every day."

    Preclinical updates include:

    • AVR-RD-05 for Hunter syndrome: Normalization of multiple biomarkers in mouse model of the disease

      The presented data showed that a lentiviral gene therapy incorporating an ApoE2 tag used in AVR-RD-05 and in-licensed from the University of Manchester, U.K., substantially reduced substrate accumulation and neuroinflammation in mouse models of Hunter syndrome to levels seen in normal mice. The presence of the tag significantly improved performance across multiple metrics in preclinical models, including normalization of skeletal features such as the cheekbone dimensions and the width of the humerus and femur bones. Patient dosing is planned to begin in 2H 2021 in an investigator-sponsored trial with the University of Manchester. The company is exploring regulatory options, including expedited development programs, to advance the product for use in neuronopathic patients, the most severe form of the disease.



    • AVR-RD-06 for Gaucher disease type 3: New program leveraging the same vector as AVR-RD-02 for Gaucher disease type 1

      The disease burden for patients with Gaucher disease type 3 includes significant neurological deterioration, which AVROBIO believes AVR-RD-06 has the potential to address by replacing diseased microglia and macrophages, or "Gaucher cells," with genetically modified cells throughout the body and brain.



    • AVR-RD-03 for Pompe disease: Preclinical data show normalization of substrate levels in multiple hard-to-reach organs

      The presented data showed that a lentiviral gene therapy incorporating a proprietary Glycosylation-Independent Lysosomal Targeting (GILT) tag used in AVR-RD-03 and in-licensed from BioMarin, reduced toxic glycogen accumulation by 97 to 100 percent in tissues including the brain, spinal cord, heart and diaphragm, and by more than 85 percent in skeletal muscle in a mouse model of the most severe form of the disease, classic infantile-onset Pompe disease. Four to eight months after dosing, substrate levels in multiple tissues of the mice treated with the tagged lentiviral gene therapy were nearly indistinguishable from normal mice. AVROBIO believes that these exciting results could only be achieved with the GILT tag to drive uptake into hard-to-reach tissues. The company is exploring options to rapidly advance AVR-RD-03 into the clinic for treatment of classic infantile-onset Pompe disease.

    End-to-end plato® platform ready to enable global commercialization

    AVROBIO also presented data on its industry-leading plato® platform highlighting advances in chemistry, manufacturing and controls (CMC) to prepare for planned upcoming trials and potential global commercialization.

    The optimized processes embedded in plato are designed to enable robust product characterization and efficient production of potent, consistent drug product on two continents, with a third site slated to become operational in Europe in 2021. New advances include:

    • Development of a universal VCN assay that may be leveraged across the portfolio: AVROBIO believes that this universal assay, intended to be transferred to multiple regulatory jurisdictions, represents a significant advance in the industry. It is designed to build a strong foundation to support future biologics license applications.
    • Advances in next-generation analytics to potentially enable deep product characterization: AVROBIO leads in single-cell analytics and whole transcriptome profiling, such as characterization of tens of thousands of single cells in the apheresis starting material used to make the drug product.
    • Implementation of global infrastructure built to scale production to meet commercial demands: plato is built with efficiency, scalability and speed in mind, from large-scale plasmid and vector manufacturing to automated drug product production to cryopreservation.

    R&D Day webcast information

    A live webcast of Virtual R&D Day and accompanying slides will be available under "Events and Presentations" in the Investors section of the company's website at www.avrobio.com. An archived webcast recording of the event will be available on the website for approximately 30 days.

    About AVROBIO

    Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by its industry leading plato® gene therapy platform, our foundation designed to deliver 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, results of preclinical studies, 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, and the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, including the use of a personalized and ultra-precision busulfan conditioning regimen. 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 enrollment and development timelines 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 on Form 10-Q, 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. Expanded ex vivo lentiviral gene therapy pipeline with program for Hunter syndrome

    Received orphan drug designation from the European Commission for two lead programs, AVR-RD-01 for Fabry disease and AVR-RD-02 for Gaucher disease type 1

    Clinical and pipeline updates to be shared at Virtual R&D Day on Tuesday, Nov. 17, 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 reported financial results for the third quarter ended Sept. 30, 2020 and provided a business update.

    "We continue to progress our clinical programs and have now dosed 12 patients across three different lysosomal disorders. We're also excited by the strategic expansion of our…

    Expanded ex vivo lentiviral gene therapy pipeline with program for Hunter syndrome

    Received orphan drug designation from the European Commission for two lead programs, AVR-RD-01 for Fabry disease and AVR-RD-02 for Gaucher disease type 1

    Clinical and pipeline updates to be shared at Virtual R&D Day on Tuesday, Nov. 17, 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 reported financial results for the third quarter ended Sept. 30, 2020 and provided a business update.

    "We continue to progress our clinical programs and have now dosed 12 patients across three different lysosomal disorders. We're also excited by the strategic expansion of our pipeline with a new program for Hunter syndrome. This devastating lysosomal disorder ravages both the brain and body, causing young boys to progressively lose cognitive and motor function. The current standard of care does not halt this tragic disease progression. We aim to halt, prevent or reverse disease with a lentiviral gene therapy designed to deliver active protein from head to toe, including throughout the central nervous system," said Geoff MacKay, president and CEO of AVROBIO. "We look forward to providing additional details on our new Hunter syndrome program and sharing new data on our three clinical programs, including initial clinical data from our Gaucher disease program, at our upcoming Virtual R&D Day."

    Expanded lentiviral gene therapy pipeline with program for Hunter syndrome (AVR-RD-05)

    • The addition of AVR-RD-05 represents a strong strategic fit that expands AVROBIO's leading lysosomal disorder gene therapy pipeline. The technology was in-licensed from The University of Manchester, UK ("UoM").
    • AVR-RD-05 involves ex vivo transduction of the patient's own hematopoietic stem cells with a lentiviral vector to integrate a therapeutic gene designed to express the functional enzyme the patient needs to maintain cellular health, coupled to a proprietary peptide tag that is designed to improve stability of the enzyme in the bloodstream and facilitate uptake in the central nervous system.
    • Hunter syndrome represents a significant unmet need, affecting an estimated one in 100,000 to 170,000 males worldwide with a high mortality rate and devastating complications throughout the body and brain, including severe cardiac and respiratory dysfunction, skeletal malformations and hearing impairment.
    • An investigator-sponsored Phase 1/2 clinical trial of AVR-RD-05 is expected to commence in the second half of 2021.

    Received orphan drug designation (ODD) from the European Commission for AVR-RD-01, an investigational gene therapy for Fabry disease, and AVR-RD-02, an investigational gene therapy for Gaucher disease type 1

    • ODD is given to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than five in 10,000 patients in the European Union.
    • AVR-RD-01 and AVR-RD-02 also have received orphan drug designation from the U.S. Food and Drug Administration.

    Broad program update planned at upcoming Virtual R&D Day on Nov. 17, 2020

    • Provide initial data on the first patient dosed in the Phase 1/2 trial for Gaucher disease, new data on the first two patients dosed in the investigator-sponsored Phase 1/2 trial for cystinosis, as well as updated patient data from the Phase 1 and Phase 2 clinical trials for Fabry disease.
    • In-depth review of the company's continued optimization of plato®, its proprietary platform designed to bring gene therapies to patients worldwide. The company will also discuss the advantages of busulfan as a conditioning agent for lentiviral gene therapy and the company's pioneering approach to personalized conditioning with precision dosing.
    • Strategic update on the company's path to market strategy and next wave of lentiviral gene therapy programs, including the recently announced program in Hunter syndrome.

    Updated statement on COVID-19 impact

    Data collection for dosed clinical trial patients is ongoing, with any delays related to COVID-19 for certain measures in our Fabry disease and cystinosis trials currently being remediated. Patient recruitment and screening activities continue across our three actively enrolling clinical trials for Fabry disease, cystinosis and Gaucher disease. The company expects to provide an update on enrollment progress across all trials at R&D day on Nov. 17, 2020.

    Third Quarter 2020 Financial Results

    AVROBIO reported a net loss of $36.8 million for the third quarter of 2020 as compared to a net loss of $17.1 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 $28.5 million for the third quarter of 2020 as compared to $13.0 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, including an $8.0 million expense related to a one-time, upfront fee we agreed to pay UoM as consideration for in-licensing the Hunter syndrome program, 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 $8.2 million for the third quarter of 2020 as compared to $5.0 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 Sept. 30, 2020, AVROBIO had $219.5 million in cash and cash equivalents, as compared to $187.0 million in cash and cash equivalents as of Dec. 31, 2019. Based on the company's current operating plan, AVROBIO expects its cash and cash equivalents as of Sept. 30, 2020 will enable the company to fund its operating expenses and capital expenditure requirements into the second half of 2022.

    About AVROBIO

    Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt and/or reverse disease throughout the body with a single dose of gene therapy designed to drive 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 preclinical programs in Hunter syndrome and 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, including the expected timing of the planned Phase 1/2 investigator-sponsored clinical trial for AVR-RD-05; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the timing of our ongoing preclinical studies; the timing and expected program updates we plan to provide at our upcoming Virtual R&D Day; 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 or delayed as a result of the COVID-19 outbreak; our plans and expectations with respect to the remediation of existing or future clinical data collection delays in connection with 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, including the planned investigator-sponsored Phase 1/2 clinical trial of AVR-RD-05; 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, that data collection efforts may be impaired or otherwise impacted by such crises, or that we may be unable to successfully remediate existing or future delays related to data collection or other clinical activities; 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)

     

    September 30,

     

    December 31,

    2020

     

    2019

     

    Cash and cash equivalents

    $

    219,546

    $

    187,043

    Prepaid expenses and other current assets

     

    9,440

     

    8,658

    Property and equipment, net

     

    3,312

     

    3,696

    Other assets

     

    957

     

    1,117

    Total assets

    $

    233,255

    $

    200,514

     

    Accounts payable

    $

    2,311

    $

    3,949

    Accrued expenses and other current liabilities

     

    22,741

     

    10,068

    Deferred rent, net of current portion

     

    331

     

    484

    Total liabilities

    $

    25,383

    $

    14,501

     

     

     

    Total stockholders' equity

     

    207,872

     

    186,013

    Total liabilities and stockholders' equity

    $

    233,255

    $

    200,514

     

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    (In thousands, except per share data)

    (Unaudited)

     

    Three Months Ended

    September 30,

     

    Nine Months Ended

    September 30,

    2020

     

    2019

     

    2020

     

    2019

     

    Operating expenses:

     

    Research and development

    $

    28,509

    $

    13,042

    $

    67,649

    $

    37,755

    General and administrative

     

    8,209

     

    5,022

     

    24,515

     

    14,621

    Total operating expenses

     

    36,718

     

    18,064

     

    92,164

     

    52,376

     

     

     

     

    Loss from operations

     

    (36,718)

     

    (18,064)

     

    (92,164)

     

    (52,376)

    Total other income (expense), net

     

    (62)

     

    919

     

    583

     

    2,073

    Net loss

    $

    (36,780)

    $

    (17,145)

    $

    (91,581)

    $

    (50,303)

    Net loss attributable to common stockholders – basic and diluted

    $

    (36,780)

    $

    (17,145)

    $

    (91,581)

    $

    (50,303)

     

     

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

    $

    (1.01)

    $

    (0.57)

    $

    (2.59)

    $

    (1.93)

     

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

     

    36,444

     

    30,297

     

    35,409

     

    26,019

     

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