GBIO Generation Bio Co.

30.84
-0.9  -3%
Previous Close 31.74
Open 31.25
52 Week Low 17
52 Week High 55.72
Market Cap $1,746,579,083
Shares 56,633,563
Float 29,929,964
Enterprise Value $1,395,234,289
Volume 146,466
Av. Daily Volume 492,422
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Latest News

  1. Patrik De Haes M.D. becomes Non-executive Chairman

    Leuven, BE and Boston, MA, US May 17, 20217:30 AM CETOxurion NV (Euronext Brussels: OXUR), a biopharmaceutical company developing next generation standard of care ophthalmic therapies, with a clinical stage portfolio in retinal vascular disorders, announces in a planned succession, the promotion of Tom Graney, CFA from Chief Financial Officer (CFO) to Chief Executive Officer (CEO) of the company. He takes over from Dr. Patrik De Haes, who after 14 years as CEO, has decided to move away from day-to-day management of the company to become the Non-executive Chairman of Oxurion. Thomas Clay, the outgoing Chairman of Oxurion, will remain on the Board as a Non-executive Director of the company…

    Patrik De Haes M.D. becomes Non-executive Chairman

    Leuven, BE and Boston, MA, US May 17, 20217:30 AM CETOxurion NV (Euronext Brussels: OXUR), a biopharmaceutical company developing next generation standard of care ophthalmic therapies, with a clinical stage portfolio in retinal vascular disorders, announces in a planned succession, the promotion of Tom Graney, CFA from Chief Financial Officer (CFO) to Chief Executive Officer (CEO) of the company. He takes over from Dr. Patrik De Haes, who after 14 years as CEO, has decided to move away from day-to-day management of the company to become the Non-executive Chairman of Oxurion. Thomas Clay, the outgoing Chairman of Oxurion, will remain on the Board as a Non-executive Director of the company.

    Mr. Graney has extensive capital markets and business development experience in biotech as the CFO of several successful public companies. This includes having raised >$500M in capital for highly innovative companies like Oxurion and completing a number of value creating business development transactions. Tom also has an impressive track record of developing high performing teams and talent everywhere he has worked both in the United States and Europe.

    Tom's transatlantic experience in creating significant company and shareholder value makes him uniquely qualified to lead Oxurion as its next CEO as it moves its highly differentiated assets through clinical development.

    Tom joined Oxurion as CFO in October 2020, and has over 25 years' experience in senior finance, strategy, business development and operational roles including most recently as CFO of Generation Bio (NASDAQ:GBIO), and before that CFO of Vertex Pharmaceuticals (NASDAQ:VRTX), and CFO and Head of Corporate Strategy at Ironwood Pharmaceuticals (NASDAQ:IRWD). Earlier in his career he spent 20 years with Johnson and Johnson, including CFO roles at Ethicon, Tibotec and Janssen. Tom also serves on the board of AC Immune (NASDAQ:ACIU).

    Tom has an MBA in finance, marketing and international business from the Leonard N. Stern School of Business at New York University and a BS in accounting from the University of Delaware. He is also a CFA charterholder.

    Tom Graney, the incoming CEO of Oxurion, said, "I am very excited and honored to become the CEO of Oxurion as we build out our plan to create an important ophthalmic company grounded in exceptional science. I am confident that with THR-149 and THR-687, two highly promising and differentiated new drug candidates, coupled with our extremely talented team, Oxurion has the potential to successfully address a broad range of retinal vascular disorders. This would allow us to generate significant value for patients, payors and shareholders as we look to disrupt and grow the $12 billion market for retinal vascular disorders."

    Patrik De Haes M.D., the incoming Chairman of Oxurion, said, "I am looking forward to supporting Tom and the rest of the Oxurion team in my new role as Chairman as we deliver a number of important clinical development milestones in the months ahead. I am confident that our drug candidates that have the potential to make a significant difference to the lives of the large number of patients with sight threatening disease, who require better treatment options." 

    Thomas Clay, outgoing Chairman of Oxurion, commented, "I would like to thank Patrik for his high level of commitment and leadership to Oxurion over the past 14 years and his desire to continue as Chairman of the company. Oxurion is well placed for future success given its industry-leading pipeline of next generation standard of care ophthalmic therapies and a highly experienced team. I look forward to working with Tom, Patrik and the rest of the board as a Non-executive Director of Oxurion as we look to realize a significant opportunity to bring value to patients and shareholders as we progress the clinical development of THR-149 and THR-687 in diabetic macular edema and other important retinal vascular disorders."

    END

    For further information please contact:

    Oxurion NV

    Wouter Piepers,

    Global Head of Investor Relations

    & Corporate Communications

    Tel: +32 478 33 56 32





     
    EU

    Citigate Dewe Rogerson

    David Dible/ Sylvie Berrebi/Frazer Hall

    Tel: +44 20 7638 9571





     



    US

    Westwicke, an ICR Company

    Christopher Brinzey

    Tel: +1 617 835 9304

    About Oxurion

    Oxurion (Euronext Brussels: OXUR) is a biopharmaceutical company developing next generation standard of care ophthalmic therapies, which are designed to better preserve vision in patients with retinal vascular disorders including diabetic macular edema (DME), the leading cause of vision loss in diabetic patients worldwide as well as other conditions, including wet age-related macular degeneration (AMD) and retinal vein occlusion (RVO).

    Oxurion is aiming to build the leading global franchise in the treatment of retinal vascular disorders based on the successful development of its two novel therapeutics:

    • THR-149, a plasma kallikrein inhibitor being developed as a potential new standard of care for the 40% of DME patients who respond suboptimally to anti-VEGF therapy. THR-149 has shown positive topline Phase 1 results for the treatment of DME. The company is currently conducting a Phase 2 clinical trial evaluating multiple injections of THR-149 in DME patients who previously responded suboptimally to anti-VEGF therapy.



    • THR-687 is a pan-RGD integrin antagonist that is initially being developed as a potential first line therapy for DME patients. Positive topline results in a Phase 1 clinical study assessing THR-687 as a treatment for DME were announced in 2020. THR-687 is expected to enter a Phase 2 clinical trial in mid-2021. THR-687, also has the potential to deliver improved treatment outcomes for patients with wet AMD and RVO.



    Oxurion is headquartered in Leuven, Belgium, and is listed on the Euronext Brussels exchange under the symbol OXUR. More information is available at www.oxurion.com.

    Important information about forward-looking statements

    Certain statements in this press release may be considered "forward-looking". Such forward-looking statements are based on current expectations, and, accordingly, entail and are influenced by various risks and uncertainties. The Company therefore cannot provide any assurance that such forward-looking statements will materialize and does not assume an obligation to update or revise any forward-looking statement, whether as a result of new information, future events, or any other reason. Additional information concerning risks and uncertainties affecting the business and other factors that could cause actual results to differ materially from any forward-looking statement is contained in the Company's Annual Report. This press release does not constitute an offer or invitation for the sale or purchase of securities or assets of Oxurion in any jurisdiction. No securities of Oxurion may be offered or sold within the United States without registration under the U.S. Securities Act of 1933, as amended, or in compliance with an exemption therefrom, and in accordance with any applicable U.S. state securities laws.



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  2. Durable, therapeutically relevant levels of anti-SARS-CoV-2 spike antibodies produced from the liver in a mouse model

    Next-generation rapid enzymatic synthesis of closed-ended DNA further increases the efficiency and scale of the manufacturing platform

    Potent factor VIII construct optimized by taking advantage of the increased cargo capacity of closed-ended DNA

    CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, today reported data from multiple digital presentations during the ongoing 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting as well as first quarter 2021 financial results.

    "We are excited…

    Durable, therapeutically relevant levels of anti-SARS-CoV-2 spike antibodies produced from the liver in a mouse model

    Next-generation rapid enzymatic synthesis of closed-ended DNA further increases the efficiency and scale of the manufacturing platform

    Potent factor VIII construct optimized by taking advantage of the increased cargo capacity of closed-ended DNA

    CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, today reported data from multiple digital presentations during the ongoing 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting as well as first quarter 2021 financial results.

    "We are excited to highlight two significant new developments for our novel, non-viral gene therapy platform that showcase its potential to address a broad range of diseases at a manufacturing scale unprecedented for gene therapy," said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. "We have long thought that therapeutic antibodies could be produced by the liver, potentially expanding the durability and reach for certain classes of biologics. Our data demonstrate endogenous therapeutic antibody production from the livers of mice for anti-SARS-CoV-2 spike antibodies. Combining these data with our proprietary rapid enzymatic synthesis method for closed-ended DNA (ceDNA) could open the door to address a number of highly prevalent infectious diseases. 2021 is set to be milestone-rich for the company, and we remain on track to execute our goals for the year."

    Highlights from Digital Presentations at ASGCT

    • Novel non-viral gene therapy unlocks significant potential for genetic medicine: In a digital presentation entitled "Non-Viral Gene Delivery of Human FVIII to Hemophilia A Mice and Non-Human Primates," the company presented insights into the scientific breakthroughs underpinning its unique non-viral approach, specifically the on-target biodistribution of its cell-targeted lipid nanoparticle (ctLNP) and the access to the nucleus afforded by ceDNA. To view the digital presentation, please visit Generation Bio's website.



    • Durable expression of therapeutically relevant levels of anti-SARS-CoV-2 spike monoclonal antibody (mAb) expressed from the liver in mice: Building on its core technologies, Generation Bio has designed a ceDNA-based approach to generate persistent expression of therapeutic antibodies from the liver. Endogenous therapeutic antibody production may provide an alternative means of delivering specific mAbs, equipping patients with the ability to produce their own biotherapeutics for an extended period of time per dose. In a digital presentation entitled, "Vector Optimization for Non-Viral Antibody Gene Therapy and Expression of Human Monoclonal Antibodies in Mice," the data presented show that ceDNA delivered via LNP enabled mice to generate persistent anti-spike protein human antibody concentrations reaching peak expression of 8 µg/ml, which corresponds to a level that may be therapeutically relevant in humans. Importantly, endogenously produced antibodies in the serum of ceDNA-treated mice retained binding and functional activity, neutralizing SARS-CoV-2 ex vivo at the same level as recombinantly produced monoclonal antibodies. Generation Bio developed these data as part of its collaboration with Vir Biotechnology, Inc. To view the digital presentation, please visit Generation Bio's website.



    • Next-generation rapid enzymatic synthesis further expands efficiency and scale of manufacturing platform: In the digital presentation on optimization of non-viral endogenous therapeutic antibody production, Generation Bio outlined a proprietary, next-generation rapid enzymatic synthesis of ceDNA directly from nucleic acid components, without relying on Sf9 cells. The structure and sequence of ceDNA from enzymatic synthesis is comparable to Sf9-produced ceDNA but shortens the ceDNA production cycle from 28 days to one day. This enzymatic process is similar to the one used to manufacture messenger RNA (mRNA) vaccines, with what the company believes will be a comparable trajectory of cost and scale.



    • Construct optimization improves potency of ctLNP-ceDNA in mouse models of hemophilia A: Generation Bio's ceDNA constructs are not constrained by the 4.7 kb transgene size limitation faced by adeno-associated virus (AAV) gene therapy. In a poster entitled, "When Size Matters: FVIII Construct Optimization Leveraging ceDNA, a Non-Viral Gene Therapy Platform," the company described its semi-combinatorial approach to optimizing factor VIII expressing ceDNA, which resulted in 34-times higher expression than an analogous wild-type factor VIII construct. This demonstrates one aspect of the potential that derives from ceDNA's greater cargo capacity, which includes improved construct potency, larger or multiple genes and the inclusion of regulatory elements that can modulate transgene expression. To view the digital presentation, please visit Generation Bio's website.

    First Quarter 2021 Financial Results

    • Cash Position: Cash, cash equivalents and marketable securities were $451.1 million as of March 31, 2021, compared with $262.3 million as of December 31, 2020.
    • R&D Expenses: Research and development (R&D) expenses were $18.8 million for the quarter ended March 31, 2021, compared to $13.4 million for the quarter ended March 31, 2020.
    • G&A Expenses: General and administrative (G&A) expenses were $6.9 million for the quarter ended March 31, 2021, compared to $4.6 million for the quarter ended March 31, 2020.
    • Net Loss: Net loss was $25.6 million, or $0.46 basic and diluted net loss per share, for the quarter ended March 31, 2021, compared to a net loss of $17.7 million, or $3.22 basic and diluted net loss per share, for the quarter ended March 31, 2020.

    About Generation Bio  

    Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The company's non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is engineered to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The company's efficient, scalable manufacturing process supports Generation Bio's mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

    Forward-Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the company, including statements about our strategic plans or objectives, our technology platforms, our research and clinical development plans, and other statements containing the words "believes," "anticipates," "plans," "expects," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the company's product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the Company's cash resources are sufficient to fund the company's operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the company's business and operations; as well as the other risks and uncertainties set forth in the "Risk Factors" section of our most recent annual report on Form 10-K, which is on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company's views as of the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the company's views as of any date subsequent to the date on which they were made.

    Contacts:

    Investors

    Maren Killackey

    Generation Bio

    541-646-2420

    Media

    Alicia Webb

    Generation Bio

    847-254-4275

    Stephanie Simon

    Ten Bridge Communications

    617-581-9333

    GENERATION BIO CO.

    CONSOLIDATED BALANCE SHEET DATA

    (Unaudited)

    (In thousands)

    Earnings Release Balance SheetMarch 31, 2021 December 31, 2020
    Cash, cash equivalents and marketable securities$451,083 $262,327
    Working capital 444,991  256,515
    Total assets 514,081  294,155
    Total stockholders' equity 458,736  268,013

    GENERATION BIO CO.

    CONSOLIDATED STATEMENTS OF OPERATIONS

    (Unaudited)

    (in thousands, except share and per share data)

     Three Months Ended March 31, 
     2021

        2020

    Operating expenses:     
    Research and development$18,753  $13,394 
    General and administrative 6,902   4,642 
    Total operating expenses 25,655   18,036 
    Loss from operations (25,655)  (18,036)
    Other income:     
    Interest income 93   319 
    Net loss and net loss attributable to common stockholders$(25,562) $(17,717)
    Net loss per share attributable to common stockholders, basic and diluted$(0.46) $(3.22)
    Weighted average common shares outstanding, basic and diluted 55,366,238   5,495,013 
          
    Comprehensive loss:     
    Net loss$(25,562) $(17,717)
    Other comprehensive income:     
    Unrealized gains on marketable securities 1    
    Comprehensive loss$(25,561) $(17,717)

     



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  3. CAMBRIDGE, Mass., April 27, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, today announced three digital presentations at the American Society of Gene & Cell Therapy (ASGCT) 2021 Virtual Annual Meeting taking place May 11-14. The presentations will cover data supporting use of the company's platform for antibody gene therapy, a summary of previously released preclinical data from the hemophilia A program and insight into ceDNA construct engineering for factor VIII.

    "We look forward to sharing preclinical data supporting the broad potential of our platform in antibody gene therapy and to providing an overview of our lead hemophilia A program…

    CAMBRIDGE, Mass., April 27, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, today announced three digital presentations at the American Society of Gene & Cell Therapy (ASGCT) 2021 Virtual Annual Meeting taking place May 11-14. The presentations will cover data supporting use of the company's platform for antibody gene therapy, a summary of previously released preclinical data from the hemophilia A program and insight into ceDNA construct engineering for factor VIII.

    "We look forward to sharing preclinical data supporting the broad potential of our platform in antibody gene therapy and to providing an overview of our lead hemophilia A program," said Matthew Stanton, Ph.D., chief scientific officer of Generation Bio. "The hemophilia A program is on track and we continue to expect additional non-human primate data by mid-year."

    Digital presentations will be available online to registered attendees beginning May 11.

    Generation Bio will present:

    • Vector Optimization for Non-Viral Antibody Gene Therapy and Expression of Human Monoclonal Antibodies in Mice



    • Non-Viral Gene Delivery of Human FVIII to Hemophilia A Mice and Non-Human Primates



    • When Size Matters: FVIII Construct Optimization Leveraging ceDNA, a Non-Viral Gene Therapy Platform

    About Generation Bio

    Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The company's non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The company's efficient, scalable manufacturing process supports Generation Bio's mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

    For more information, please visit www.generationbio.com.

    Forward-Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the company, including statements about our strategic plans or objectives, our technology platforms, our research and clinical development plans, and other statements containing the words "believes," "anticipates," "plans," "expects," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the company's product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the company's cash resources are sufficient to fund the company's operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the company's business and operations; as well as the other risks and uncertainties set forth in the "Risk Factors" section of our most recent annual report on Form 10-K, which is on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company's views as of the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the company's views as of any date subsequent to the date on which they were made.

    Contacts:

    Investors

    Maren Killackey

    Generation Bio

    541-646-2420

    Media

    Alicia Webb

    Generation Bio

    847-254-4275

    Stephanie Simon

    Ten Bridge Communications

    617-581-9333



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  4. CAMBRIDGE, Mass., April 08, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Geoff McDonough, M.D., president and chief executive officer, will participate in a fireside chat at the 20th Annual Needham Virtual Healthcare Conference on Thursday, April 15, at 3:00 p.m. ET.

    A live webcast of the fireside chat will be available on the investor section of the company's website at investors.generationbio.com. A replay will be available there for 30 days following the event.

    About Generation Bio

    Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable…

    CAMBRIDGE, Mass., April 08, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Geoff McDonough, M.D., president and chief executive officer, will participate in a fireside chat at the 20th Annual Needham Virtual Healthcare Conference on Thursday, April 15, at 3:00 p.m. ET.

    A live webcast of the fireside chat will be available on the investor section of the company's website at investors.generationbio.com. A replay will be available there for 30 days following the event.

    About Generation Bio

    Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The company's non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is engineered to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The company's efficient, scalable manufacturing process supports Generation Bio's mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

    For more information, please visit www.generationbio.com

    Contact:

    Investors 

    Maren Killackey

    Generation Bio



    541-646-2420

    Media 

    Alicia Webb

    Generation Bio



    847-254-4275

    Stephanie Simon 

    Ten Bridge Communications



    617-581-9333



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  5. $262.3 million cash balance at the end of 2020 bolstered by additional net proceeds of $211.3 million from follow-on offering, funding key milestones into 2024

    Ron Cooper appointed to Board of Directors

    Company remains on track to begin IND-enabling studies for hemophilia A in 2021

    CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, reported recent business highlights and fourth quarter and full year 2020 financial results.

    "2020 was a foundational year for Generation Bio in which we strengthened the leadership team, advanced our platform science, and added substantially to our balance sheet to fund our lead programs…

    $262.3 million cash balance at the end of 2020 bolstered by additional net proceeds of $211.3 million from follow-on offering, funding key milestones into 2024

    Ron Cooper appointed to Board of Directors

    Company remains on track to begin IND-enabling studies for hemophilia A in 2021

    CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (NASDAQ:GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, reported recent business highlights and fourth quarter and full year 2020 financial results.

    "2020 was a foundational year for Generation Bio in which we strengthened the leadership team, advanced our platform science, and added substantially to our balance sheet to fund our lead programs to human data," said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. "We entered 2021 with preclinical data in mice and non-human primates that we believe demonstrate our platform's potential to go beyond what is currently possible with gene therapy for hemophilia A. We expect to announce additional data throughout the remainder of this year that will support the rapid expansion of our pipeline and showcase our differentiated approach to creating a new class of genetic medicine."

    Recent Business Highlights

    • Raised $225.4 million in gross proceeds from follow-on offering: In January 2021, Generation Bio closed a public offering of 9,200,000 shares of its common stock at a public offering price of $24.50 per share, including 1,200,000 additional shares of common stock issued upon the exercise in full by the underwriters of their option to purchase additional shares. The gross proceeds of the offering, before deducting the underwriting discount and commissions and other offering expenses payable by Generation Bio, were approximately $225.4 million.



    • Appointed Ron Cooper to Board of Directors: In February 2021, Generation Bio announced the appointment of Ron Cooper to its Board of Directors. Mr. Cooper is a global biopharmaceutical executive who currently serves as president, chief executive officer and board member of Albireo Pharma, Inc.



    • Announced preclinical data that support development candidate selection for hemophilia A program: Also in January 2021, the company reported data from a series of preclinical studies evaluating the expression of factor VIII in both mice and non-human primates (NHP). Data in mice showed that a single dose of a closed-ended DNA (ceDNA) construct delivered via the company's novel, cell-targeted lipid nanoparticle (ctLNP) system led to mean human factor VIII expression levels from 9% to 23% of normal with a dose response across the three cohorts. In two additional separate studies, a mean 2:1 species translation ratio from mice to NHPs was observed, consistent with best-in-class translation ratios seen for other nucleic acids delivered via LNPs.

    Selected Anticipated Company Milestones in 2021

    • NHP factor VIII expression data for hemophilia A development candidate
    • NHP factor IX expression, durability and redosing data
    • Initiation of IND-enabling studies for phenylketonuria (PKU)
    • Preclinical data for Gaucher disease and Wilson disease
    • Preclinical data for expression of therapeutic antibodies

    Fourth Quarter and Full Year 2020 Financial Results

    • Cash Position: Cash, cash equivalents and marketable securities were $262.3 million as of December 31, 2020, compared with $15.1 million as of December 31, 2019. Cash at December 31, 2020 does not include the $211.3 million in net proceeds received from the follow-on public offering completed in January 2021.

       
    • R&D Expenses: Research and development (R&D) expenses were $16.4 million for the quarter ended December 31, 2020 and $58.5 million for the year ended December 31, 2020, compared to $12.7 million for the quarter ended December 31, 2019 and $50.1 million for the year ended December 31, 2019.

       
    • G&A Expenses: General and administrative (G&A) expenses were $8.0 million for the quarter ended December 31, 2020 and $22.6 million for the year ended December 31, 2020, compared to $3.4 million for the quarter ended December 31, 2019 and $12.2 million for the year ended December 31, 2019.



    • Net Loss: Net loss was $24.2 million, or $0.53 basic and diluted net loss per share, for the quarter ended December 31, 2020 and $80.5 million, or $2.95 basic and diluted net loss per share, for the year ended December 31, 2020, compared to a net loss of $16.1 million, or $3.09 basic and diluted net loss per share, for the quarter ended December 31, 2019 and $61.3 million, or $12.96 basic and diluted net loss per share, for the year ended December 31, 2019.

    About Generation Bio  

    Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The company's non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is engineered to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The company's efficient, scalable manufacturing process supports Generation Bio's mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

    Forward-Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about our strategic plans or objectives, our technology platforms, our research and clinical development plans, and other statements containing the words "believes," "anticipates," "plans," "expects," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the Company's cash resources are sufficient to fund the Company's operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the Company's business and operations; as well as the other risks and uncertainties set forth in the "Risk Factors" section of our most recent quarterly report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in subsequent filings the Company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date on which they were made.

    Contacts:

    Investors

    Maren Killackey

    Generation Bio

    541-646-2420

    Media

    Alicia Webb

    Generation Bio

    847-254-4275

    Stephanie Simon

    Ten Bridge Communications

    617-581-9333



    GENERATION BIO CO.

    CONSOLIDATED BALANCE SHEET DATA

    (Unaudited)

    (In thousands)

      December 31,
      2020  2019 
    Cash, cash equivalents and marketable securities $262,327  $15,076 
    Working capital  256,515   8,998 
    Total assets  294,155   42,140 
    Convertible preferred stock     115,593 
    Total stockholders' equity (deficit)  268,013   (98,592)
             

    GENERATION BIO CO.

    CONSOLIDATED STATEMENTS OF OPERATIONS

    (Unaudited)

    (in thousands, except share and per share data)

       Three Months Ended December 31,   Year Ended December 31,
       2020   2019   2020  2019 
    Operating expenses:                
    Research and development $16,374  $12,731  $58,532  $50,134 
    General and administrative  7,971   3,427   22,582   12,168 
    Total operating expenses  24,345   16,158   81,114   62,302 
    Loss from operations  (24,345)  (16,158)  (81,114)  (62,302)
    Other income (expense):                
    Interest income and other income (expense), net  119   95   591   985 
    Net loss and net loss attributable to common stockholders $(24,226) $(16,063) $(80,523) $(61,317)
    Net loss per share attributable to common stockholders, basic and diluted $(0.53) $(3.09) $(2.95) $(12.96)
    Weighted average common shares outstanding,

    basic and diluted
      45,855,896   5,205,942   27,256,494   4,731,519 
                     
    Comprehensive loss:                
    Net loss $(24,226) $(16,063) $(80,523) $(61,317)
    Other comprehensive income:                
    Unrealized (loss) gain on marketable securities  (17)  (2)  9   9 
    Comprehensive loss $(24,243) $(16,065) $(80,514) $(61,308)



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