“We are building a leading non-viral genetic medicine company with the potential to transform treatments within multiple therapeutic areas,” said
Business Highlights
- Reported Preclinical Data Demonstrating Broad Delivery of ceDNA and mRNA to Photoreceptors Using ctLNP: In
October 2021 ,Generation Bio presented preclinical data at theEuropean Society of Gene andCell Therapy (ESGCT) AnnualVirtual Congress showing that sub-retinal delivery of closed-ended DNA using cell-targeted lipid nanoparticle (ctLNP-ceDNA) in rodents demonstrated broad distribution and durable expression that was comparable to that achieved with adeno-associated virus Type 5 (AAV5) (the current standard of delivery) in rodents, and tolerability. These findings support the use of ctLNP-ceDNA to potentially achieve full gene replacement for inherited retinal diseases. Similar results were observed for sub-retinal delivery of mRNA using ctLNP in both rodents and non-human primates. This first-ever demonstration of species translation with comparable tolerability and uniform photoreceptor expression across species using ctLNP suggests ctLNP could be a best-in-class non-viral delivery system for mRNA to the retina, potentially enabling gene editing in the retina.
Third Quarter 2021 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$398.4 million as ofSeptember 30, 2021 , compared with$262.3 million as ofDecember 31, 2020 . - R&D Expenses: Research and development (R&D) expenses were
$22.0 million for the quarter endedSeptember 30, 2021 , compared to$15.3 million for the quarter endedSeptember 30, 2020 . - G&A Expenses: General and administrative (G&A) expenses were
$9.7 million for the quarter endedSeptember 30, 2021 , compared to$5.7 million for the quarter endedSeptember 30, 2020 . - Net Loss: Net loss was
$31.9 million , or$0.56 basic and diluted net loss per share, for the quarter endedSeptember 30, 2021 , compared to a net loss of$20.8 million for the quarter endedSeptember 30, 2020 .
About
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 investigational new drug, or 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 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; uncertainties regarding the timing and ability to complete the build-out of the company’s 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 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 and quarterly report on Form 10-Q, which are on file with the
Contacts:
Investors
541-646-2420
mkillackey@generationbio.com
Media
847-254-4275
awebb@generationbio.com
Ten
617-903-8783
lisa@tenbridgecommunications.com
CONSOLIDATED BALANCE SHEET DATA
(Unaudited)
(In thousands)
Earnings Release Balance Sheet | ||||||||
Cash, cash equivalents and marketable securities | $ | 398,363 | $ | 262,327 | ||||
Working capital | 388,764 | 256,515 | ||||||
Total assets | 464,033 | 294,155 | ||||||
Total stockholders’ equity | 407,210 | 268,013 | ||||||
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(Unaudited)
(in thousands, except share and per share data)
Three Months Ended | ||||||||
2021 | 2020 | |||||||
Operating expenses: | ||||||||
Research and development | $ | 21,991 | $ | 15,308 | ||||
General and administrative | 9,667 | 5,661 | ||||||
Total operating expenses | 31.658 | 20,969 | ||||||
Loss from operations | (31,658 | ) | (20,969 | ) | ||||
Other (expense) income: | ||||||||
Other (expense) and interest income, net | (197 | ) | 120 | |||||
Net loss and net loss attributable to common stockholders | $ | (31,855 | ) | $ | (20,849 | ) | ||
Net loss per share attributable to common stockholders, basic and diluted | $ | (0.56 | ) | $ | (0.46 | ) | ||
Weighted average common shares outstanding, basic and diluted | 56,629,193 | 45,468,838 | ||||||
Comprehensive loss: | ||||||||
Net loss | $ | (31,855 | ) | $ | (20,849 | ) | ||
Other comprehensive loss: | ||||||||
Unrealized (losses) gains on marketable securities | (3 | ) | 30 | |||||
Comprehensive loss | $ | (31,858 | ) | $ | (20,819 | ) | ||
Source:
2021 GlobeNewswire, Inc., source