2022 Strategic Priorities
Generation Bio has created a unique non-viral genetic medicine platform comprised of closed-ended DNA (ceDNA), rapid enzymatic synthesis (RES) manufacturing for ceDNA and a proprietary portfolio of ctLNPs. The company is applying its platform technologies in three areas: the liver, retina and vaccines.
Liver: In December 2021, the company reported preclinical data demonstrating that enhanced manufacturing and production controls for its ceDNA and ctLNPs achieved peak mean human factor VIII expression of 205% in mice with a single dose, a near nine-fold improvement from studies using materials produced by prior processes. In non-human primates (NHPs), peak human factor VIII expression reached 2%. The demonstration of therapeutic protein expression in mice and NHPs represents a milestone achievement for a non-viral DNA delivery platform. The difference in expression between species is attributed to differences in biodistribution. Generation Bio plans to continue to optimize the biodistribution profile of its liver ctLNP in NHP to support the selection of a development candidate for its hemophilia A program and to advance additional liver programs.
Retina: Generation Bio has developed ctLNPs for the retina that may allow the company to address a variety of inherited retinal diseases using full gene replacement or gene editing. In October 2021, the company presented preclinical findings at the European Society of Gene and Cell Therapy (ESGCT) demonstrating that sub-retinal delivery of messenger RNA (mRNA) or ceDNA via retina ctLNP resulted in broad photoreceptor distribution, expression, and good tolerability in rodents and in NHPs. Generation Bio plans to evaluate more advanced generations of its retina ctLNP in NHPs in order to enable potential selection of a development candidate for its lead Stargardt disease program, as well as for use in subsequent retina programs.
Vaccines: Generation Bio aims to develop a novel vaccine platform using a vaccine ctLNP to deliver ceDNA or mRNA that could meet or exceed the efficacy of existing mRNA vaccines. In January 2022, the company reported preclinical findings from studies of the company’s mRNA-vaccine ctLNP. At a dose of 10μg, mRNA delivered via vaccine ctLNP demonstrated a neutralizing antibody response in mice similar to the benchmark established by Moderna Therapeutics’ mRNA1273, the COVID-19 vaccine approved for individuals ages 18 and older. These murine results translated to NHP a similar neutralizing antibody response was observed at a dose of 100μg. The company is also exploring ceDNA delivered with its vaccine ctLNP, which could enable more durable protection and be stored at ambient temperatures potentially allowing for greater shelf stability than current mRNA-LNP vaccines. Generation Bio plans to evaluate a next generation vaccine ctLNP in NHPs that has demonstrated enhanced potency in preclinical murine studies for both mRNA and ceDNA.
Fourth Quarter and Full Year 2021 Financial Results
Cash Position: Cash and cash equivalents were $375.1 million as of December 31, 2021, compared with $262.3 million in cash, cash equivalents and marketable securities as of December 31, 2020. The company continues to believe that its cash position is expected to fund its operating plan into 2024.
R&D Expenses: Research and development (R&D) expenses were $21.8 million for the quarter ended December 31, 2021, and $85.2 million for the year ended December 31, 2021, compared to $16.4 million for the quarter ended December 31, 2020, and $58.5 million for the year ended December 31, 2020.
G&A Expenses: General and administrative (G&A) expenses were $9.1 million for the quarter ended December 31, 2021, and $33.9 million for the year ended December 31, 2021, compared to $8.0 million for the quarter ended December 31, 2020, and $22.6 million for the year ended December 31, 2020.
Net Loss: Net loss was $30.9 million, or $0.54 basic and diluted net loss per share, for the quarter ended December 31, 2021, and $119.2 million, or $2.12 basic and diluted net loss per share, for the year ended December 31, 2021, compared to a net loss of $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.
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