Generation Bio Reports Business Highlights and Fourth Quarter and Full Year 2023 Financial Results
- Generation Bio is making strides in leveraging its ctLNP platform for in vivo T cell and hematopoietic stem cell programs, showcasing selective targeting and high delivery efficiency.
- The development of iqDNA by Generation Bio demonstrates immune-quiet properties, with robust expression in mice, paving the way for innovative therapeutic applications in hepatocytes and other cell types.
- The company's cash position of $264.4 million as of December 31, 2023, is projected to fund operational and capital needs until the second half of 2027, ensuring financial stability for future growth.
- None.
Insights
The reported cash balance of $264.4 million by Generation Bio Co. indicates a strong cash position, providing the company with a significant runway to fund its operations into the second half of 2027. This is a critical factor for investors as it suggests the company does not require immediate additional financing, which could dilute existing shareholders' equity. The slight decrease in cash reserves from the previous year's $279.1 million is not alarming given the R&D investments made during the period.
Regarding R&D expenses, the increase to $93.6 million for the year, despite a reduction in force, reflects the company's commitment to advancing its technology platforms and in vivo programs. The one-time charge of $5.1 million indicates a restructuring, which could be seen as a strategic move to streamline operations and allocate resources more efficiently. Investors typically look for such cost optimization strategies as they can lead to improved operational efficiencies and potentially better margins in the long term.
The net loss figures, while significant, are not uncommon for biotechnology companies in the development stage. The slight improvement from a net loss of $136.6 million in 2022 to $126.6 million in 2023 could be interpreted as a positive trend, reflecting management's control over expenses while pursuing their R&D objectives. However, the basic and diluted net loss per share of $1.96 for the year ended December 31, 2023, compared to $2.35 for 2022, should be seen in the context of the company's long-term potential to bring new treatments to market.
Generation Bio Co.'s focus on developing its proprietary cell-targeted lipid nanoparticle (ctLNP) delivery system and immune-quiet DNA (iqDNA) platforms could represent a significant advancement in the treatment of genetic diseases like sickle cell disease and beta-thalassemia. The ability to target and transduce T cells and hematopoietic stem cells (HSCs) in vivo is a notable scientific breakthrough, potentially offering a competitive edge over existing ex vivo therapies.
From a market perspective, the shift towards in vivo therapies could disrupt the current treatment paradigm, offering patients less invasive and more accessible treatment options. The company's collaboration with Moderna on T cell therapies also adds credibility and potential synergies in R&D. The focus on optimizing iqDNA for hepatocytes and T cells is equally promising, with the potential to address a wide range of genetic diseases, including hemophilia A.
The strategic importance of these developments cannot be overstated. If successful, Generation Bio's platforms could lead to new therapeutic classes with broad applications, positioning the company as a leader in genetic medicine. This could have long-term positive implications for the company's market positioning and valuation, pending successful clinical development and regulatory approvals.
The development of ctLNP and iqDNA by Generation Bio Co. represents a significant step in the field of genetic medicine. The high selectivity and potency in targeting T cells and HSCs in humanized mouse models suggest potential for a paradigm shift in the treatment of diseases like sickle cell disease and beta-thalassemia. Unlike traditional ex vivo approaches, which often require chemotherapy and are limited by the availability of treatment slots and high costs, in vivo therapies can be administered directly into the patient, potentially reducing the treatment burden and associated costs.
The reported progress in the development of immune-quiet DNA is particularly noteworthy. By avoiding innate immune detection, iqDNA could enable sustained therapeutic gene expression without eliciting an adverse immune response, which is a common challenge in gene therapy. This innovation could lead to treatments that are not only more effective but also safer for patients.
While these technologies are promising, it is important to note the challenges ahead, including the need to demonstrate efficacy and safety in clinical trials and to navigate the complex regulatory landscape. The reported financial stability of the company provides a conducive environment for these critical development phases. The implications for patients with genetic diseases could be profound, offering hope for more effective and convenient therapies in the future.
- Company is leveraging proprietary cell-targeted lipid nanoparticle delivery to develop wholly-owned in vivo program for sickle cell disease and beta-thalassemia
- Development of breakthrough immune-quiet DNA for hemophilia A program continues
- Cash balance of
CAMBRIDGE, Mass., March 06, 2024 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and fourth quarter and full year 2023 financial results.
“Late last year we announced important advances in our cell-targeted lipid nanoparticle and immune-quiet DNA platforms that we are now translating to our portfolio strategy,” said Geoff McDonough, M.D., chief executive officer of Generation Bio. “The potency and selectivity of our ctLNP platform open up significant opportunities for in vivo delivery of T cell and hematopoietic stem cell therapies, and this year we are laying the foundation for a wholly-owned program in sickle cell disease and beta-thalassemia. We are also continuing to optimize iqDNA to progress our hemophilia A program and to explore therapeutic applications in cell types beyond hepatocytes. With our current cash balance, we believe we have sufficient runway to reach meaningful development milestones for our platforms and programs.”
Business Highlights
- Developing ctLNP for in vivo T cell and hematopoeitc stem cell (HSC) programs: Generation Bio’s ctLNP platform has demonstrated a set of key characteristics that could make possible in vivo genetic medicine approaches to treating diseases for which only ex vivo therapies are currently available. The company has developed ctLNPs that selectively target and transduce
75% of circulating and splenic T cells and maintain high levels of delivery at doses as low as 0.005 mg/kg in humanized mice. In addition to continuing this work in T cells in collaboration with Moderna, Generation Bio is developing a wholly-owned in vivo program targeting HSCs to address sickle cell disease and beta-thalassemia. In vivo approaches could offer important advantages over current ex vivo approaches, such as the lack of pre-treatment chemotherapy, redosing, on-demand availability and lower cost. In 2024, the company intends to show highly selective in vivo delivery to T cells with therapeutic transgenes, and proof of concept for specific and potent in vivo HSC targeting in humanized mouse models. - Developing iqDNA for programs in hepatocytes and other cell types: In 2023, Generation Bio announced that it had developed a novel DNA cargo that was shown to avoid innate immune detection in both mice and NHPs. In addition to these immune quiet characteristics, iqDNA also exhibited robust and durable expression in mice. This profile could lead to differentiated therapeutics utilizing hepatocytes to treat a wide range of genetic diseases, including hemophilia A, as well extrahepatic tissues and cell types, such as T cells and HSCs. In 2024, the company intends to optimize iqDNA for application in hepatocytes and T cells with a focus on improving potency.
Fourth Quarter and Full Year 2023 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$264.4 million as of December 31, 2023, compared with$279.1 million in cash, cash equivalents, and marketable securities as of December 31, 2022. The company continues to believe that its cash, cash equivalents and marketable securities will fund its operating expenditures and capital expenditure requirements into the second half of 2027. - R&D Expenses: Research and development (R&D) expenses were
$27.9 million for the quarter ended December 31, 2023, including a one-time$5.1 million charge related to the company’s reduction in force, and$93.6 million for the year ended December 31, 2023, compared to$21.6 million for the quarter ended December 31, 2022, and$96.7 million for the year ended December 31, 2022. - G&A Expenses: General and administrative (G&A) expenses were
$13.4 million for the quarter ended December 31, 2023, including a one-time$1.9 million charge related to the company’s reduction in force, and$50.9 million for the year ended December 31, 2023, compared to$13.1 million for the quarter ended December 31, 2022, and$44.5 million for the year ended December 31, 2022. - Net Loss: Net loss was
$35.2 million , or$0.53 b asic and diluted net loss per share, for the quarter ended December 31, 2023, and$126.6 million , or$1.96 b asic and diluted net loss per share, for the year ended December 31, 2023, compared to a net loss of$32.4 million , or$0.55 b asic and diluted net loss per share, for the quarter ended December 31, 2022, and$136.6 million , or$2.35 b asic and diluted net loss per share, for the year ended December 31, 2022.
About Generation Bio
Generation Bio is innovating non-viral genetic medicines to provide durable and redosable treatments for hundreds of millions of patients living with rare and prevalent diseases. The company is developing two distinct and complementary platforms: a potent, highly selective cell-targeted lipid nanoparticle (ctLNP) delivery system and a novel immune-quiet DNA (iqDNA) cargo produced by a scalable capsid-free manufacturing process that uses proprietary cell-free rapid enzymatic synthesis (RES). With these platforms, Generation Bio aims to develop the next wave of non-viral genetic medicines 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 the company’s strategic plans or objectives, cash resources, technology platforms, research and clinical development plans, and preclinical data 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; uncertainties regarding our novel platforms and related technologies; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; 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; as well as the other risks and uncertainties set forth in the “Risk Factors” section of the company’s 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 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.
Investors and Media Contact
Maren Killackey
Generation Bio
mkillackey@generationbio.com
857-371-4638
| GENERATION BIO CO. CONSOLIDATED BALANCE SHEET DATA (Unaudited) (In thousands) | |||||||
| Earnings Release Balance Sheet | December 31, 2023 | December 31, 2022 | |||||
| Cash, cash equivalents and marketable securities | $ | 264,364 | $ | 279,091 | |||
| Working capital | 232,704 | 267,866 | |||||
| Total assets | 374,758 | 376,264 | |||||
| Total stockholders’ equity | 203,128 | 282,493 | |||||
| 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, | ||||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||||
| Revenues: | |||||||||||||||
| Collaboration Revenue | $ | 2,878 | $ | — | $ | 5,904 | $ | — | |||||||
| Operating expenses: | |||||||||||||||
| Research and development | 27,923 | 21,607 | 93,617 | 96,718 | |||||||||||
| General and administrative | 13,376 | 13,081 | 50,850 | 44,464 | |||||||||||
| Total operating expenses | 41,299 | 34,688 | 144,467 | 141,182 | |||||||||||
| Loss from operations | (38,421 | ) | (34,688 | ) | (138,563 | ) | (141,182 | ) | |||||||
| Other income: | |||||||||||||||
| Other income and interest income, net | 3,235 | 2,283 | 11,951 | 4,543 | |||||||||||
| Net loss | $ | (35,186 | ) | $ | (32,405 | ) | $ | (126,612 | ) | $ | (136,639 | ) | |||
| Net loss per share, basic and diluted | $ | (0.53 | ) | $ | (0.55 | ) | $ | (1.96 | ) | $ | (2.35 | ) | |||
| Weighted average common shares outstanding, basic and diluted | 66,062,208 | 59,407,296 | 64,483,520 | 58,114,893 | |||||||||||
| Comprehensive loss: | |||||||||||||||
| Net loss | $ | (35,186 | ) | $ | (32,405 | ) | $ | (126,612 | ) | $ | (136,639 | ) | |||
| Other comprehensive income (loss): | |||||||||||||||
| Unrealized gains (losses) on marketable securities | 278 | 219 | 357 | (83 | ) | ||||||||||
| Comprehensive loss | $ | (34,908 | ) | $ | (32,186 | ) | $ | (126,255 | ) | $ | (136,722 | ) | |||
FAQ
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