Financial Performance - Generation Bio reported a quarterly loss of $3.12 per share, which was worse than the Zacks Consensus Estimate of a loss of $2.8, representing an earnings surprise of -11.43% [1] - The company posted revenues of $0.77 million for the quarter ended June 2025, missing the Zacks Consensus Estimate by 51.58%, compared to year-ago revenues of $4.09 million [2] - The current consensus EPS estimate for the upcoming quarter is -$2.62 on revenues of $1.58 million, and for the current fiscal year, it is -$11.10 on revenues of $13.46 million [7] Stock Performance - Generation Bio shares have declined approximately 61.9% since the beginning of the year, contrasting with the S&P 500's gain of 8.4% [3] - The company has surpassed consensus EPS estimates two times over the last four quarters [2] Industry Outlook - The Medical - Biomedical and Genetics industry, to which Generation Bio belongs, is currently ranked in the bottom 43% of over 250 Zacks industries, indicating potential challenges ahead [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which could impact Generation Bio's stock performance [5] Future Expectations - The earnings outlook for Generation Bio will depend on management's commentary during the earnings call and how earnings expectations may change following the recent report [4][6] - The estimate revisions trend for Generation Bio was favorable ahead of the earnings release, resulting in a Zacks Rank 2 (Buy) for the stock, suggesting it may outperform the market in the near future [6]

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 June 30, 2025 OR For the transition period from to Commission File Number: 001-39319 GENERATION BIO CO. (Exact name of registrant as specified in its charter) Delaware 81-4301284 (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification Number) 301 Binne ...

Generation Bio Announces New Data for Its Novel ctLNP Delivery System and Early T Cell Programs and Reports Second Quarter 2025 Financial Results CAMBRIDGE, MASS., August 12, 2025 -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company working to change what's possible for people living with T cell-driven autoimmune diseases, today announced new data demonstrating that its cell-targeted lipid nanoparticle (ctLNP) system selectively delivered siRNA to T cells in non-human primates, reinforcing the comp ...

Core Insights - Generation Bio Co. has demonstrated the ability of its cell-targeted lipid nanoparticle (ctLNP) system to selectively deliver siRNA to T cells in non-human primates, marking a significant advancement in genetic medicine for T cell-driven autoimmune diseases [1][2][3] Financial Position - As of June 30, 2025, the company reported a cash balance of $141.4 million, down from $185.2 million at the end of 2024 [11][14] - Research and development (R&D) expenses for Q2 2025 were $15.5 million, a decrease from $16.4 million in Q2 2024 [11][16] - General and administrative (G&A) expenses were $7.7 million for Q2 2025, down from $9.5 million in the same quarter of the previous year [11][16] - The net loss for Q2 2025 was $20.9 million, or $3.12 per share, compared to a net loss of $20.4 million, or $3.07 per share, in Q2 2024 [11][16] Strategic Developments - The company is exploring strategic alternatives to maximize the value of its delivery system and potential therapeutics, including options such as acquisition, merger, or sale of assets [5][6] - A significant workforce reduction of approximately 90% is planned as part of a strategic restructuring process, expected to begin in mid-August 2025 and conclude by the end of October 2025 [7][6] Research and Development Highlights - The ctLNP system has shown significant knockdown of beta-2 microglobulin in T cells over three weeks in a non-human primate study, indicating its selective and potent delivery capabilities [3][4] - Lead siRNA candidates have demonstrated effective knockdown of LAT1 and VAV1, which are relevant targets for T cell-driven autoimmune diseases [4][6]

Core Viewpoint - Generation Bio Co. announced a 1-for-10 reverse stock split to increase the per-share market price and regain compliance with Nasdaq listing requirements [1][3]. Group 1: Reverse Stock Split Details - The reverse stock split will be effective at 5:00 pm Eastern Time on July 21, 2025, and trading on a split-adjusted basis will begin on July 22, 2025 [1][2]. - The number of issued and outstanding shares will decrease from approximately 67.3 million to approximately 6.7 million [3]. - No fractional shares will be issued; instead, stockholders will receive cash payments for any fractional shares [4]. Group 2: Company Background - Generation Bio is focused on developing therapeutics for T cell-driven autoimmune diseases using cell-targeted lipid nanoparticles to deliver small interfering RNA to T cells [7]. - The company's innovative approach aims to address historically undruggable disease-driving genes in autoimmunity [7].

Company Overview - Generation Bio is a biotechnology company focused on developing therapeutics for T cell-driven autoimmune diseases [3] - The company aims to create redosable therapeutics that reprogram T cells in vivo to reduce or eliminate autoreactive T cells, which attack the body's own tissues [3] - Generation Bio utilizes cell-targeted lipid nanoparticles (ctLNP) to deliver small interfering RNA (siRNA) selectively to T cells, potentially addressing previously undruggable disease-driving genes in autoimmunity [3] Recent Developments - On July 1, 2025, Generation Bio granted equity awards to a new employee as part of its 2025 Inducement Stock Incentive Plan [1] - The employee received non-statutory stock options to purchase 128,600 shares of common stock at an exercise price of $0.32 per share, equal to the closing price on the grant date [2] - The options have a 10-year term and will vest over four years, with 25% vesting on the first anniversary and 6.25% vesting quarterly thereafter, contingent on the employee's continued service [2]

Summary of Generation Bio (GBIO) Conference Call Company Overview - **Company**: Generation Bio (GBIO) - **Focus**: Development of targeted siRNA therapies for T cell-driven autoimmune diseases - **CEO**: Jeff McDonough Key Points Industry and Market Context - Generation Bio aims to address significant unmet needs in autoimmune diseases driven by T cells, which are critical in both the initiation and maintenance of these conditions [4][5] - The company is developing a first-in-class delivery system for siRNA that selectively targets T cells, a significant advancement in the field of RNA therapeutics [3][4] Therapeutic Approach - The delivery system utilizes a cell-targeted lipid nanoparticle (LNP) technology that allows for selective delivery of siRNA to T cells while avoiding off-target effects in other immune cells [11][12] - The approach is designed to knock down "undruggable" T cell targets, which have historically been difficult to address with existing therapies [3][4] Target Indications - Generation Bio is focusing on several autoimmune diseases, including: - **Alopecia and Vitiligo**: Approximately 3 million patients affected, with current therapies having significant off-target side effects [6] - **Ulcerative Colitis and Crohn's Disease**: Current therapies only benefit about one-third of patients, indicating a substantial opportunity for improvement [6] - **Primary Biliary Cholangitis (PBC)**: No disease-modifying therapies currently available [7] - **Type 1 Diabetes**: Potential to preserve islet function and insulin independence [7] Competitive Landscape - The company believes it has a unique position in the market due to its ability to selectively deliver siRNA to T cells, a capability not currently matched by other technologies [27] - The competitive landscape is evolving with increased interest in RNAi technologies, but Generation Bio maintains a strong advantage due to its innovative delivery system [26][27] Clinical Development Timeline - Generation Bio plans to file its first Investigational New Drug (IND) application in the second half of 2026, with lead targets to be announced in August 2025 [3][21][33] - The company has a robust pipeline of preclinical data and plans to present additional data from non-human primate studies over the next year [34] Financial Position - As of the last quarter, Generation Bio reported a cash position of approximately $150 million, which is expected to fund operations into the second half of 2027 [38] Collaboration and Partnerships - The collaboration with Moderna is progressing well, with both companies satisfied with the advancements made in the delivery system [36] Future Outlook - Generation Bio is optimistic about the potential of its targeted siRNA therapies to provide disease-modifying treatments for autoimmune diseases, with a focus on achieving durable responses and improving patient outcomes [30][31] Additional Insights - The company emphasizes the importance of selectively targeting T cells without activating them, which has been a challenge for antibody-based therapies [23][24] - Generation Bio's approach may allow for a more durable therapeutic effect, potentially reducing the frequency of treatment needed for patients [30]

Core Insights - Generation Bio Co. is focused on developing innovative therapeutics for T cell-driven autoimmune diseases [3] - The company will present at the Jefferies Global Healthcare Conference on June 4, 2025 [1] - A live webcast of the presentation will be available on the company's investor website [2] Company Overview - Generation Bio is developing redosable therapeutics that aim to reprogram T cells in vivo to combat autoimmune diseases [3] - The company utilizes cell-targeted lipid nanoparticles (ctLNP) for selective delivery of small interfering RNA (siRNA) to T cells [3] - This approach targets historically undruggable genes associated with autoimmune diseases, potentially unlocking new treatment avenues [3]

Financial Performance - For the three months ended March 31, 2025 and 2024, the company reported net losses of $14.8 million and $74.5 million, respectively, with an accumulated deficit of $717.8 million as of March 31, 2025[91]. - Net loss narrowed to $14.8 million in Q1 2025 compared to a net loss of $74.5 million in Q1 2024, reflecting an improvement of 80%[112]. - Collaboration revenue increased to $8.7 million for the three months ended March 31, 2025, up from $4.1 million in the same period of 2024, representing a growth of 115%[113]. - Total operating expenses decreased to $25.3 million in Q1 2025 from $81.7 million in Q1 2024, a reduction of 69%[112]. - General and administrative expenses decreased to $8.8 million in Q1 2025 from $10.4 million in Q1 2024, a decline of 15%[115]. - Research and development expenses rose to $15.4 million in Q1 2025, up from $14.3 million in Q1 2024, an increase of 7%[114]. - Net cash used in operating activities was $28.4 million in Q1 2025, compared to $31.7 million in Q1 2024, a decrease of 10.4%[120]. - Net cash provided by investing activities was $3.0 million in Q1 2025, primarily due to $45.0 million in maturities of marketable securities[124]. Future Expectations - The company expects to incur significant expenses and increasing operating losses for at least the next several years due to ongoing research programs, technology expansion, and clinical development[91]. - The company expects to announce the target and indication of its lead T cell-selective LNP-siRNA program for autoimmune disease by mid-year 2025[88]. - The company expects to submit its investigational new drug (IND) application for its lead program in the second half of 2026[88]. - The company believes its existing cash, cash equivalents, and marketable securities will fund operations into the second half of 2027[95]. - The company expects to fund its operating expenses into the second half of 2027 with existing cash and marketable securities[128]. - The company has not yet commercialized any product candidates and does not expect to generate revenue from sales for several years[118]. Collaboration and Revenue Generation - The collaboration with Moderna includes an upfront payment of $40.0 million and eligibility for up to $1.8 billion in milestone payments, along with tiered royalties on product sales[101]. - The company has not generated any revenue from product sales and expects future revenue primarily from the collaboration with Moderna[90]. Market and Financial Risks - The company may need to curtail or discontinue research or development programs if sufficient capital is not raised[129]. - Additional funds raised through collaborations or licensing may require relinquishing valuable rights to future revenue streams[129]. - The company is exposed to market risk related to changes in interest rates, primarily due to investments in short-term securities[133]. - A hypothetical increase in interest rates of 100 basis points would have resulted in a $0.4 million decrease in the fair value of the company's holdings[133]. - The company does not currently seek to hedge exposure to fluctuations in interest rates[133]. Accounting and Financial Management - There have been no material changes to the company's critical accounting policies and estimates from those disclosed in the 2024 Annual Report[132]. - The company evaluates its estimates and assumptions on an ongoing basis, which may differ from actual results under different conditions[132]. - The company incurs costs associated with acquisitions of in-licensed products and operating as a public company[131]. - There were no impairment charges recorded for marketable securities during the three months ended March 31, 2025[133]. - As of March 31, 2025, the company had marketable securities totaling $106.7 million[133].

Financial Performance - Cash balance as of March 31, 2025, was $157.6 million, down from $185.2 million as of December 31, 2024, expected to fund operations into the second half of 2027[6] - Net loss for Q1 2025 was $14.8 million, or $0.22 per share, compared to a net loss of $74.5 million, or $1.12 per share, in Q1 2024, indicating a substantial improvement[11] - Total operating expenses for Q1 2025 were $25.3 million, down from $81.7 million in Q1 2024, a decrease of 69.0%[16] - Loss on lease termination was $1.1 million in Q1 2025, significantly lower than $56.9 million in Q1 2024[11] Revenue and Expenses - Research and development (R&D) expenses for Q1 2025 were $15.4 million, compared to $14.3 million in Q1 2024, reflecting a year-over-year increase of 7.7%[7] - General and administrative (G&A) expenses decreased to $8.8 million in Q1 2025 from $10.4 million in Q1 2024, a reduction of 15.4%[7] - Collaboration revenue for Q1 2025 was $8.7 million, significantly higher than $4.1 million in Q1 2024, representing a 114.6% increase[16] Assets and Future Plans - Total assets as of March 31, 2025, were $201.3 million, down from $231.2 million as of December 31, 2024[14] - The company plans to announce the target and portfolio strategy for its lead ctLNP-siRNA program in mid-2025[4] - Generation Bio is focused on developing first-in-class treatments for T cell-driven autoimmune diseases using its innovative ctLNP technology[6]