GlobeNewswire Inc | Aug 11, 2021 04:20PM EDT

August 11, 2021

CAMBRIDGE, Mass., Aug. 11, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported recent business highlights and second quarter 2021 financial results.

This quarter we announced our shift to rapid enzymatic synthesis, or RES, for production of our closed-ended DNA, ceDNA, constructs and our signing of a lease to establish significant internal current Good Manufacturing Practice, or cGMP, manufacturing capacity. These are important steps toward our goal of extending the reach of our durable, redosable genetic medicines to patients with prevalent diseases, said Geoff McDonough, M.D., chief executive officer of Generation Bio. We will continue to advance the platform throughout the rest of the year and expect factor VIII expression data with ceDNA produced using RES in non-human primatesfor our hemophilia A program by year-end.

Business Highlights

-- Transition of Portfolio to Next-Generation Rapid Enzymatic Manufacturing Process Underway: In July 2021, Generation Bio announced plans to deploy next-generation RES for manufacturing its closed-ended DNA (ceDNA) constructs across all portfolio programs. The company also signed a lease agreement to build out a state-of-the-art cGMP facility of approximately 104,000 square feet in Waltham, Mass. to scale RES for clinical and initial commercial supply. The facility is expected to be operational in 2023, enabling an Investigational New Drug (IND) application and clinical development for the companys lead program in hemophilia A. The company is using its existing infrastructure for current production of research material using RES. As Generation Bio transitions to RES, it expects to update its program development strategy and timelines for its pipeline.

Second Quarter 2021 Financial Results

-- Cash Position:Cash, cash equivalents and marketable securities were$425.2 millionas ofJune 30, 2021, compared with $262.3 million as of December 31, 2020. -- R&D Expenses:Research and development (R&D) expenses were$22.7millionfor the quarter ended June 30, 2021, compared to $13.5 million for the quarter ended June 30, 2020. -- G&A Expenses:General and administrative (G&A) expenses were$8.2millionfor the quarter ended June 30, 2021, compared to $4.3 million for the quarter ended June 30, 2020. -- Net Loss:Net loss was$30.8 million, or $0.55 basic and diluted net loss per share,for the quarter ended June 30, 2021, compared to a net loss of $17.7 million, or $1.50 basic and diluted net loss per share, for the quarter ended June 30, 2020.

About Generation Bio

Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral genetic medicine platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bios manufacturing scale to hundreds of millions of doses to support its mission to extend the reach of genetic medicine to more people, living with more diseases, 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 platform, our research and clinical development plans, the expected timing of the submission of IND applications and preclinical data, our manufacturing plans, our expectations regarding our new facility 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 companys 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; uncertainties regarding the timing and ability to complete the build-out of the companys manufacturing facility and regarding the new manufacturing process; expectations regarding the timing of submission of IND applications; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the companys cash resources are sufficient to fund the companys operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the companys 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 and quarterly report on Form 10-Q, which are 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 companys views as of the date hereof. The company anticipates that subsequent events and developments will cause the companys 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 companys views as of any date subsequent to the date on which they were made.

Contacts:

InvestorsMaren KillackeyGeneration Bio541-646-2420mkillackey@generationbio.com

MediaAlicia WebbGeneration Bio847-254-4275awebb@generationbio.com

Lisa RaffenspergerTen Bridge Communications617-903-8783lisa@tenbridgecommunications.com

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA (Unaudited)(In thousands)

Earnings Release Balance Sheet June30,2021 December31,2020Cash, cash equivalents and marketable $ 425,193 $ 262,327securitiesWorking capital 418,810 256,515Total assets 490,792 294,155Total stockholders? equity 433,402 268,013

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Unaudited)

(in thousands, except share and per share data)

Three Months Ended June30, 2021 2020 Operating expenses: Research and development $ 22,656 $ 13,456 General and administrative 8,186 4,308 Total operating expenses 30,842 17,764 Loss from operations (30,842 ) (17,764 )Other income: Interest income 51 33 Net loss and net lossattributable to common $ (30,791 ) $ (17,731 )stockholdersNet loss per share attributableto common stockholders, basic and $ (0.55 ) $ (1.50 )dilutedWeighted average common shares 56,318,025 11,801,704 outstanding, basic and diluted Comprehensive loss: Net loss $ (30,791 ) $ (17,731 )Other comprehensive loss: Unrealized losses on marketable (6 ) (4 )securitiesComprehensive loss $ (30,797 ) $ (17,735 )