Group 1 - Generation Bio Co. reported a quarterly loss of $0.32 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.28, and an improvement from a loss of $0.53 per share a year ago, indicating an earnings surprise of -14.29% [1] - The company posted revenues of $4.19 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 6.95%, and showing an increase from year-ago revenues of $2.88 million [2] - Generation Bio shares have declined approximately 50.5% since the beginning of the year, contrasting with the S&P 500's decline of -4.8% [3] Group 2 - The earnings outlook for Generation Bio is mixed, with the current consensus EPS estimate for the coming quarter at -$0.28 on revenues of $0.86 million, and for the current fiscal year at -$0.95 on revenues of $9.52 million [7] - The Medical - Biomedical and Genetics industry, to which Generation Bio belongs, is currently ranked in the top 26% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 OR Delaware 81-4301284 (State or other jurisdiction of incorporation or organization) 301 Binney Street Cambridge, Massachusetts 02142 (Address of principal executive offices) (Zip Code) ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT ...

Financial Performance - Generation Bio reported a cash balance of $185.2 million as of December 31, 2024, down from $264.4 million as of December 31, 2023, expected to fund operations into the second half of 2027[5][11]. - The net loss for Q4 2024 was $21.4 million, or $0.32 per share, compared to a net loss of $35.2 million, or $0.53 per share, in Q4 2023[6][12]. - The total operating expenses for the year ended December 31, 2024, were $162.3 million, an increase from $144.5 million in 2023[6][12]. - A non-cash charge of $3.6 million was recognized in Q4 2024 related to the termination of the lease for the manufacturing facility, totaling $63.2 million for the year[6][12]. - Total assets decreased to $231.2 million as of December 31, 2024, from $374.8 million as of December 31, 2023[11]. Research and Development - Research and development (R&D) expenses for Q4 2024 were $15.5 million, a decrease from $27.9 million in Q4 2023, while total R&D expenses for the year were $61.3 million compared to $93.6 million in 2023[6][12]. - Generation Bio achieved approximately 98% knockdown of the B2M protein in human T cells using its T cell-selective lipid nanoparticle (LNP) in both in vitro and mouse studies[4]. - The company plans to announce the target and indication for its lead ctLNP-siRNA program in mid-2025 and submit an investigational new drug (IND) application in the second half of 2026[4][5]. General and Administrative Expenses - General and administrative (G&A) expenses for Q4 2024 were $8.7 million, down from $13.4 million in Q4 2023, with total G&A expenses for the year at $37.8 million compared to $50.9 million in 2023[6][12]. Collaboration Revenue - Collaboration revenue for Q4 2024 was $4.2 million, compared to $2.9 million in Q4 2023, with total collaboration revenue for the year at $19.9 million versus $5.9 million in 2023[6][12].

Core Insights - Generation Bio is developing T cell-selective lipid nanoparticles (LNP) for siRNA therapeutics targeting T cell-driven autoimmune diseases, with a lead target and indication expected to be announced by mid-2025 [1][3][4] - The company reported a cash balance of $185.2 million as of December 31, 2024, which is projected to fund operations into the second half of 2027 [4][5] Business Highlights - The T cell-selective LNP is designed to deliver siRNA specifically to T cells, achieving approximately 98% knockdown of the B2M protein in human T cells in studies [2][3] - This innovative approach aims to selectively modulate T cell activity involved in autoimmune diseases while minimizing off-target effects on the broader immune system [2][3] - Generation Bio plans to submit an investigational new drug (IND) application in the second half of 2026 [3] Financial Results - Cash, cash equivalents, and marketable securities decreased from $264.4 million in 2023 to $185.2 million in 2024 [4][9] - Research and development (R&D) expenses for Q4 2024 were $15.5 million, down from $27.9 million in Q4 2023, while total R&D expenses for the year were $61.3 million compared to $93.6 million in 2023 [4][11] - General and administrative (G&A) expenses for Q4 2024 were $8.7 million, down from $13.4 million in Q4 2023, with total G&A expenses for the year at $37.8 million compared to $50.9 million in 2023 [4][11] - The net loss for Q4 2024 was $21.4 million, or $0.32 per share, compared to a net loss of $35.2 million, or $0.53 per share, in Q4 2023 [5][11]

Company Overview - Generation Bio is a biotechnology company focused on developing therapeutics for T cell-driven autoimmune diseases [3] - The company utilizes a novel approach with T cell-selective cell-targeted lipid nanoparticles (ctLNP) to deliver siRNA in vivo, aiming to modulate T cell activity responsible for inflammation and tissue destruction [3] - This new modality is designed to effectively target areas that are challenging to address with existing therapies, potentially expanding treatment options for various autoimmune diseases [3] Upcoming Event - Geoff McDonough, M.D., the president and CEO of Generation Bio, will present at the TD Cowen 45th Annual Health Care Conference on March 3, 2025, at 9:10 a.m. ET in Boston [1] - A live webcast of the presentation will be accessible on the investor section of the company's website, with a replay available for 30 days post-event [2]

Core Viewpoint - Generation Bio is advancing its T cell-directed lipid nanoparticle (ctLNP) technology to develop siRNA therapeutics aimed at treating T cell-driven autoimmune diseases, with plans to submit its first Investigational New Drug (IND) application in the second half of 2026 [1][3][7]. Group 1: Technology and Development - The company is utilizing its validated ctLNP to deliver siRNA specifically to T cells, aiming to silence disease-driving targets without affecting other immune cell types [2][9]. - Generation Bio's ctLNP technology has shown strong selectivity for CD8+ and CD4+ effector T cells and NK cells, which are involved in auto-reactive tissue damage in autoimmune diseases [2][3]. - The ctLNP-siRNA approach is designed to modulate T cell functions that lead to inflammation and tissue damage, potentially addressing high-value, currently undruggable targets [3][7]. Group 2: Clinical Plans and Timeline - The company plans to enter clinical trials within its cash runway, which extends into the second half of 2027, following the submission of its first IND in late 2026 [2][3]. - Generation Bio is reorganizing to support the clinical development of its T cell-directed therapies, indicating a strategic shift towards clinical capabilities [3][7]. Group 3: Leadership Changes - The company announced changes in its executive leadership team, with Phillip Samayoa, Ph.D., becoming the new chief scientific officer, and Kevin Conway being appointed as chief financial officer [4][5]. - Matthew Stanton, the outgoing chief scientific officer, will transition to the Scientific Advisory Board, while Matthew Norkunas, M.D., will remain until mid-2025 [4][5].

Group 1 - Generation Bio Co. reported a quarterly loss of $0.23 per share, better than the Zacks Consensus Estimate of a loss of $0.28, and improved from a loss of $0.43 per share a year ago, representing an earnings surprise of 17.86% [1] - The company posted revenues of $7.55 million for the quarter ended September 2024, surpassing the Zacks Consensus Estimate by 249.24%, compared to year-ago revenues of $2.15 million [2] - Generation Bio shares have increased approximately 32.7% since the beginning of the year, outperforming the S&P 500's gain of 21.2% [3] Group 2 - The current consensus EPS estimate for the coming quarter is -$0.24 on revenues of $2.16 million, and for the current fiscal year, it is -$1.80 on revenues of $12.48 million [7] - The Medical - Biomedical and Genetics industry, to which Generation Bio belongs, is currently ranked in the top 38% of over 250 Zacks industries, indicating a favorable outlook for the sector [8]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to | --- | --- | |---------------------------------------------------------------------------------------|----------------------------------------- ...

Generation Bio Reports Recent Business Highlights and Third Quarter 2024 Financial Results ● Non-human primate data presented at ESGCT demonstrated selective in vivo delivery of mRNA with T cell-targeted lipid nanoparticle (ctLNP) ● Cash balance of $199.8 million expected to fund operations into 2H 2027 CAMBRIDGE, MASS., Nov. 6, 2024 -- Generation Bio Co. (Nasdaq:GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and t ...

Core Insights - Generation Bio Co. presented new data on its cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, highlighting advancements in genetic medicines for rare and prevalent diseases [1][2] Group 1: ctLNP Platform - A single intravenous dose of ctLNP successfully delivered mRNA encoding a reporter protein to a majority of circulating T cells in non-human primates, with a dose of 1 mg/kg transducing most T cells and showing balanced distribution to CD4+ and CD8+ cells [2] - Untargeted lipid nanoparticles (LNPs) did not achieve T cell transduction, indicating the specificity of the ctLNP platform [2] - The biodistribution of ctLNP-mRNA to other organs such as the liver, spleen, and lung was minimal, confirming improved results over previous murine studies [2] Group 2: iqDNA Platform - The iqDNA platform is a partially single-stranded DNA molecule designed to evade recognition by primary DNA sensors in both mice and non-human primates [3] - Recent research has focused on enhancing protein expression through methods such as encoding the promoter region as double-stranded and stabilizing iqDNA against degradation by exonucleases [3] - The company is pursuing targeted research areas to prepare iqDNA for the development of novel genetic medicines [3] Group 3: Company Overview - Generation Bio is focused on innovating non-viral genetic medicines aimed at providing durable and redosable treatments for patients with rare and prevalent diseases [5] - The company is developing two complementary platforms: the highly selective ctLNP delivery system and the novel iqDNA cargo produced through a scalable capsid-free manufacturing process [5] - The mission of Generation Bio is to extend the reach of genetic medicine to a broader patient population globally [5]