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EX - 99.1 2 gbio - 20211110xex99d1.htm EX - 99.1 generation bio Generation Bio Reports Business Highlights and Third Quarter 2021 Financial Results New preclinical retinal data illustrating potential for expansive therapeutic applications of ctLNP delivery presented at ESGCT 2021 Annual Virtual Congress CAMBRIDGE , MASS . , Nov. 10 , 2021 -- Generation Bio Co. ( Nasdaq : GBIO ) , a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases , reported recent business highlights and third quarter 2021 financial results . " We are building a leading non - viral genetic medicine company with the potential to transform treatments within multiple therapeutic areas , ” said Geoff McDonough , M.D. , chief executive officer of Generation Bio . " We continue to advance our ceDNA , and next - generation rapid enzymatic synthesis technologies , and plan to provide data on factor VIII expression in non - human primates for our hemophilia A program by year- end . We also continue to develop our ctLNP delivery platform , and with the data recently presented at the ESGCT Congress , demonstrated a breakthrough for the retinal field , potentially paving the way for a wave of novel retinal therapies . ” 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 the European Society of Gene and Cell Therapy ( ESGCT ) Annual Virtual 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 of September 30 , 2021 , compared with $ 262.3 million as of December 31 , 2020 . • • R & D Expenses : Research and development ( R & D ) expenses were $ 22.0 million for the quarter ended September 30 , 2021 , compared to $ 15.3 million for the quarter ended September 30 , 2020 . G & A Expenses : General and administrative ( G & A ) expenses were $ 9.7 million for the quarter ended September 30 , 2021 , compared to $ 5.7 million for the quarter ended September 30 , 2020 . 1