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Generation Bio Reports Business Highlights and Fourth Quarter and Full Year 2024 Financial Results

1. Generation Bio targets T cell-driven autoimmune diseases with innovative siRNA therapies. 2. Lead target announcement expected in mid-2025; IND submission by late 2026. 3. Company cash balance at $185.2 million, funding operations until late 2027. 4. R&D expenses reduced significantly; net loss shows slight improvement year-over-year. 5. Losses tied to lease terminations reflect strategic realignments in operations.

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Why Bullish?

The company's focus on a novel therapeutic approach positions it well in a competitive market. Historical examples show similar innovations like CAR-T therapies have led to substantial institutional and retail interest, positively impacting stock prices.

How important is it?

The article discusses critical developments and cash liquidity, enhancing the company's outlook. Innovations targeting autoimmune diseases possess a high market potential, increasing investor interest and potential stock performance.

Why Long Term?

Therapeutic developments such as the one being proposed require time for clinical validation and market uptake. Companies focusing on innovative treatments often see price increases over time as milestones are met.

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March 13, 2025 16:05 ET  | Source: Generation Bio Co. -  Company is applying its T cell-selective lipid nanoparticle to develop siRNA therapeutics for T cell-driven autoimmune diseases -  Lead target and indication to be announced MY 2025 -  Cash balance of $185.2 million expected to fund operations into 2H 2027 CAMBRIDGE, MASS., March 13, 2025 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO), a biotechnology company working to change what’s possible for people living with T cell-driven autoimmune diseases, reported business highlights and fourth quarter and full year 2024 financial results. “Our T cell-selective lipid nanoparticle (LNP) is designed to overcome a long-standing challenge for siRNA therapeutics, which is to reach T cells while sparing broader immune cells and achieve potent knockdown of genetic targets in vivo,” said Geoff McDonough, M.D., chief executive officer of Generation Bio. “Significant classes of drugs in immune and inflammation indications modulate T cells, but their impact is constrained due to off-target effects. We believe that by combining the selective delivery of our cell-targeted LNP (ctLNP) platform with the genetic precision of siRNA, we can reach high-value targets that cause T cell-driven autoimmune diseases but are undruggable or poorly drugged by conventional modalities. This approach has the potential to offer predictable pharmacology and expand the therapeutic index, creating a new application space for T cell-specific therapies.” Business Highlights Applying T cell-selective LNP Delivery System to Develop siRNA Therapeutics for T Cell-driven Autoimmune Diseases: Generation Bio has designed a ctLNP that is specifically targeted to T cells to achieve selective delivery of siRNA and knockdown of targets in vivo, overcoming significant historical challenges for the development of siRNA therapeutics in T cells. The company has shown that its T cell-selective LNP containing an unoptimized siRNA achieved efficient delivery and approximately 98% knockdown of the B2M protein in human T cells in both in vitro and mouse studies.Delivering siRNA with a T cell-selective LNP could enable selective modulation of T cell activity involved in tissue inflammation and damage while sparing the broader immune system. The development of T cell-selective LNP-siRNA therapeutics could address targets that are undruggable or poorly drugged by conventional modalities and offer the potent, precise protein knockdown and wide therapeutic index required to fulfill unmet need in certain T cell-driven autoimmune diseases. Generation Bio 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. Fourth Quarter and Full Year 2024 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $185.2 million as of December 31, 2024, compared with $264.4 million in cash, cash equivalents, and marketable securities as of December 31, 2023. 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 $15.5 million for the quarter ended December 31, 2024 and $61.3 million for the year ended December 31, 2024, compared to $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. G&A Expenses: General and administrative (G&A) expenses were $8.7 million for the quarter ended December 31, 2024 and $37.8 million for the year ended December 31, 2024, compared to $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. Loss on lease termination: During the three months and year ended December 31, 2024, we recognized a non-cash charge of $3.6 million and $63.2 million, respectively, in connection with the termination of the lease for our manufacturing facility in Waltham, Massachusetts.Net Loss: Net loss was $21.4 million, or $0.32 basic and diluted net loss per share, for the quarter ended December 31, 2024, and $131.7 million, or $1.98 basic and diluted net loss per share, for the year ended December 31, 2024, compared to a net loss of $35.2 million, or $0.53 basic and diluted net loss per share, for the quarter ended December 31, 2023, and $126.6 million, or $1.96 basic and diluted net loss per share, for the year ended December 31, 2023. About Generation Bio Generation Bio is a biotechnology company working to change what’s possible for people living with T cell-driven autoimmune diseases. The company is developing redosable therapeutics that reprogram T cells in vivo to reduce or eliminate the production and persistence of autoreactive T cells, which erroneously recognize and attack the body’s own tissues, causing autoimmune diseases. Generation Bio’s innovative approach leverages cell-targeted lipid nanoparticles (ctLNP) to selectively deliver small interfering RNA (siRNA) to T cells. This combination of selective delivery and an intracellular, genetically precise mechanism of target engagement could unlock a series of high-value, historically undruggable disease-driving genes in autoimmunity. 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 anticipated timing of identification of the company’s product candidates, 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 technologies; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties regarding the anticipated timing of the submission of an investigational new drug application; 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 ContactMaren KillackeyGeneration Biomkillackey@generationbio.com857-371-4638       GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA (Unaudited)(In thousands)      Earnings Release Balance SheetDecember 31, 2024 December 31, 2023Cash, cash equivalents and marketable securities$185,223 $264,364Working capital 157,848  232,704Total assets 231,197  374,758Total stockholders’ equity 86,204  203,128           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,   2024   2023   2024   2023 Revenues:                Collaboration Revenue $4,188  $2,878  $19,892  $5,904                  Operating expenses:                Research and development  15,494   27,923   61,305   93,617 General and administrative  8,656   13,376   37,780   50,850 Loss on lease termination  3,601   —   63,197   — Total operating expenses  27,751   41,299   162,282   144,467 Loss from operations  (23,563)  (38,421)  (142,390)  (138,563)Other income:                Other income and interest income, net  2,181   3,235   10,722   11,951 Net loss $(21,382) $(35,186) $(131,668) $(126,612)Net loss per share, basic and diluted $(0.32) $(0.53) $(1.98) $(1.96)Weighted average common shares outstanding, basic and diluted  66,790,803   66,062,208   66,624,249   64,483,520                  Comprehensive loss:                Net loss $(21,382) $(35,186) $(131,668) $(126,612)Other comprehensive income (loss):                Unrealized gains (losses) on marketable securities  (160)  278   (115)  357 Comprehensive loss $(21,542) $(34,908) $(131,783) $(126,255)

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