CD33

Remain in active dialogue with the FDA regarding IND clearance for GTB-3650, a 2nd generation nanobody TriKE® for treatment of CD33+ leukemia Phase 1 trial with GTB-3650 anticipated to start in 2H 2024 Anticipate submitting an IND for GTB-5550 targeting B7H3 for multiple solid tumors, including prostate and breast, in Q4 2024 Cash of approximately $14 million as of December 31, 2023, provides sufficient runway to fund operations into 2025 BRISBANE, CALIFORNIA, March 26, 2024 (GLOBE NEWSWIRE) -- GT Biopharma, Inc. (the “Company”) (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company's proprietary natural killer (NK) cell engager, TriKE® platform, today announced fourth quarter and full-year 2023 results for the period ended December 31, 2023. “We continue to make good progress with the FDA regarding IND clearance for GTB-3650, and eagerly anticipate the start of Phase 1 this year,” said Michael Breen, Executive Chairman and Interim Chief Executive Office of GT Biopharma. “We are also anticipating an IND submission for our 2nd asset, GTB-5550, for multiple solid tumors, including breast and prostate cancers, in Q4 of this year. We have sufficient cash runway into 2025 and anticipate having initial data from our Phase 1 trial with GTB-3650 by the end of 2024/early 2025.” Fourth Quarter and Year End 2023 Financial Summary Cash Position: The Company had cash, cash equivalents and short-term investments of $13.97 million as of December 31, 2023 compared to $16.51 million as of December 31, 2022. This is anticipated to provide sufficient runway to fund operations into 2025. Research and Development (R&D) Expenses: R&D expenses for the fourth quarter of 2023 were $1.36 million compared to $2.84 million for the same quarter of 2022. R&D expenses for the year ended December 31, 2023 were $6.47 million compared to $8.81 million for the year ended December 31, 2022. The $2.34 million reduction in R&D for the year ended December 31, 2023 over 2022 was primarily due to a $3.59 million reduction in licensing and administrative costs, offset by an increase of $1.25 million in R&D costs related to our continued development and production of our most advanced TriKE® product candidates GTB-3650 and GTB-5550 along with the progression of other promising product candidates. The reduction of $3.59 million in licensing and administrative costs over the previous year was primarily due to better management of R&D expenses with consultants and reduction in stock compensation to employees. We anticipate our direct clinical and preclinical expenses to continue to increase in 2024 as we plan to advance our next generation GTB-3650 camelid nanobody product into the clinic and enroll patients, perform tests for data collection, complete the product development of GTB-5550 and anticipate submission of IND application for GTB-5550 in the fourth quarter of 2024. We do not, however, anticipate an increase in related R&D licensing and administrative costs. General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2023 were $1.81 million compared to $2.94 million for the same quarter in 2022. G&A expenses for the year ended December 31, 2023 was $7.11 million compared to $12.45 million for the year ended December 31, 2022. The decrease in G&A of $5.34 million for the year ended December 31, 2023 as compared to 2022 was primarily attributable to a reduction of $1.68 million in stock compensation expenses for officers, employees and board of directors and $1.92 million for outside consultants, a reduction of $1.31 million in consulting board advisory board fees, and a reduction of $0.50 million in patents and insurance costs. G&A decreased due to better managing and use of consultants and advisors, and overall reductions in other general and administrative expenses in 2023. Other Income Other income net of other expenses, for the fourth quarter ended December 31, 2023 was $0.21 million compared to $0.19 million for the same quarter ended December 31, 2022. Other income, net of other expenses for the year ended December 31, 2023 was $5.98 million compared to $0.37 million for the year ended December 31, 2022. Other income and expenses consisted of interest income, interest expense, change in the fair value of warrant lability, gain on extinguishment of debt, and unrealized gain and loss on marketable securities. The increase in other income net of expenses for the year ended December 31, 2023 was primarily due to the change in fair value of warrant liability as a result of fair value remeasurement which resulted in a gain of $4.8 million and $0.12 million for the years ended December 31, 2023 and 2022, respectively. Net Loss: The Company reported a net loss of $2.96 million for the fourth quarter ended December 31, 2023 compared to a loss of $5.58 million for the same quarter in 2022. The Company reported a loss of $7.60 million for the year ended December 31, 2023, compared to a net loss of $20.88 million for the year ended December 31, 2022. About Camelid Antibodies Camelid antibodies are single domain antibodies (sdAbs) from the Camelidae family of mammals that include llamas, camels, and alpacas. These animals produce two main types of antibodies. One type of antibody camelids produce is the conventional antibody that is made up of two heavy chains and two light chains. They also produce another type of antibody that is made up of only two heavy chains and no light chain. This is known as heavy chain IgG (hcIgG). While these antibodies do not contain the CH1 region, they retain an antigen binding domain called the VHH region. VHH antibodies, also known as single domain antibodies, contain only the VHH region from the camelid antibody. Camelid antibodies have key characteristics, which include high affinity and specificity (equivalent to conventional antibodies), high thermostability, good solubility and strictly monomeric behavior, small size, relatively low production cost, ease of genetic engineering, format flexibility or modularity, low immunogenicity, and a higher penetration rate into tissues. About GT Biopharma, Inc. GT Biopharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of immuno-oncology therapeutic products based on our proprietary TriKE® NK cell engager platform. Our TriKE® platform is designed to harness and enhance the cancer killing abilities of a patient’s immune system’s natural killer cells. GT Biopharma has an exclusive worldwide license agreement with the University of Minnesota to further develop and commercialize therapies using TriKE® technology. For more information, please visit gtbiopharma.com. Forward-Looking Statements Certain statements in this press release may constitute "forward-looking statements" regarding future events and our future results. All statements other than statements of historical facts are statements that could be deemed to be forward-looking statements. These statements are based on current expectations, estimates, forecasts, and projections about the markets in which we operate and the beliefs and assumptions of our management. Words such as "expects," "anticipates," "targets," "goals," "projects", "intends," "plans," "believes," "seeks," "estimates," "endeavors," "strives," "may," or variations of such words, and similar expressions are intended to identify such forward-looking statements. Readers are cautioned that these forward-looking statements are subject to a number of risks, uncertainties and assumptions that are difficult to predict, estimate or verify. Therefore, actual results may differ materially and adversely from those expressed in any forward-looking statements. Such risks and uncertainties include those factors described in our most recent annual report on Form 10-K, as such may be amended or supplemented by subsequent quarterly reports on Form 10-Q, or other reports filed with the Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements. Examples of forward-looking statements in this press release include statements regarding our IND applications, Phase 1 trials and operating expenses and cash runway. The forward-looking statements are made only as of the date hereof, and we undertake no obligation to publicly release the result of any revisions to these forward-looking statements. For more information, please refer to our filings with the Securities and Exchange Commission. TriKE® is a registered trademark owned by GT Biopharma, Inc. Investor Relations Contact: LifeSci Advisors Corey Davis, Ph.D. cdavis@lifesciadvisors.com 212-915-2577

Trem-cel and VCAR33ALLO clinical trial data expected in the second half of 2024VCAR33ALLO granted Fast Track and Orphan Drug Designation by U.S. Food & Drug Administration Cash runway extends into second half of 2025 CAMBRIDGE, Mass., March 20, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period and full year ended December 31, 2023, and provided a business update. “We are very pleased with the foundational progress we made in 2023. Building on this, we expect 2024 to be an exciting year in which we demonstrate the Vor Bio approach to be instrumental in reducing the disease burden of AML, a devastating cancer where we desperately need effective treatment options,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. Corporate Updates Multiple patients expected to be dosed with VCAR33ALLO in the first half of 2024; initial data anticipated in second half of 2024 VBP301, a Phase 1/2, multicenter, open-label, first-in-human study of VCAR33ALLO, is a transplant donor-derived anti-CD33 CAR-T cell therapy for patients with AML who have relapsed following a standard-of-care or trem-cel transplant. The first patient was dosed in January 2024, and multiple patients are expected to be dosed in the first half of 2024. We anticipate initial data in the second half of 2024.The U.S. Food & Drug Administration (FDA) has granted Fast Track Designation and Orphan Drug Designation to VCAR33ALLO. The FDA Fast Track process aims to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. Orphan Drug Designation entitles companies to development incentives including tax credits for clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity in the event of regulatory approval. VCAR33ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell therapy that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. VBP101 clinical trial of trem-cel enrolling steadily, with the next data readout expected in the second half of 2024 Dose escalation of Mylotarg to 1.0 mg/m2 has commenced with multiple patients now treated.Patients receiving a trem-cel transplant who become measurable residual disease (MRD) positive or relapse have the option to receive induction-course Mylotarg or VCAR33ALLO.The Company expects to report further engraftment and protection data from the VBP101 clinical trial in the second half of 2024.The latest data update from VBP101, the Phase 1/2a clinical study of trem-cel, was presented at the ASH Annual Meeting on December 10, 2023, showing primary neutrophil engraftment in all eight patients and hematologic protection from acute Mylotarg toxicity through repeat doses. Trem-cel is a shielded transplant in development for patients with AML, in which healthy transplant donor cells are genetically engineered by removing CD33, with the potential to enable targeted therapies such as Mylotarg and CD33-targeted CAR-T therapy post-transplant, while avoiding on-target toxicities. Continued progress on dual-targeted CAR-T and multiplex engineering approach The Company continues to make progress with a dual-specific CAR-T targeting CD33 and CLL-1, two of the most promising antigens in AML. This is being paired with a hematopoietic stem cell (HSC) multiplex engineering approach, which could allow removal or modification of these two genes. The Company has demonstrated in vitro proof of concept for this approach. IND-enabling work is progressing for the Company’s dual-specific CAR-T with key in vivo proof-of-concept experiments underway. Upcoming Milestones Trem-cel clinical data update expected in the second half of 2024VCAR33ALLO clinical data update expected in the second half of 2024 Fourth Quarter and Full Year 2023 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $137.2 million as of December 31, 2023, which is projected to fund operations into the second half of 2025. Research & Development (R&D) Expenses: R&D expenses for the fourth quarter of 2023 were $20.9 million, compared to $17.1 million for the fourth quarter of 2022, and for the year ended December 31, 2023, were $94.3 million, compared to $64.6 million for the year ended December 31, 2022. The increase in R&D expenses was attributable primarily to an increase in clinical trial, manufacturing and personnel expenses, and the execution of our non-exclusive license agreement with Editas Medicine.General & Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2023 were $7.2 million, compared to $7.7 million for the fourth quarter of 2022, and for the year ended December 31, 2023, were $31.7 million, compared to $28.9 million for the year ended December 31, 2022. The increase in G&A expenses was primarily attributable to an increase in personnel expenses, including an increase in share-based compensation expense.Net Loss: Net loss for the fourth quarter of 2023 was $26.3 million, compared to $23.9 million for the fourth quarter of 2022, and for the year ended December 31, 2023, was $117.9 million, compared to $92.1 million for the year ended December 31, 2022. About Vor BioVor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant. For more information, visit: www.vorbio.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “aim,” “anticipate,” “can,” “continue,” “could,” “design,” “enable,” “expect,” “initiate,” “intend,” “may,” “on-track,” “ongoing,” “plan,” “potential,” “should,” “target,” “update,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include Vor Bio’s statements regarding the potential of its product candidates to positively impact quality of life and alter the course of disease in the patients it seeks to treat, the timing an pace of patient enrollment and dosing in clinical trials and the availability of data therefrom, the expected safety profile of its product candidates, its intentions to use VCAR33ALLO in combination with trem-cel as a Treatment System, its potential upcoming milestones, its cash runway and expected capital requirements. Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Vor Bio’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; the success of Vor Bio’s in-house manufacturing capabilities and efforts; and availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law. Condensed Consolidated Balance Sheet Data(in thousands) December 31, December 31, 2023 2022 Cash, cash equivalents and marketable securities $137,175 $230,245 Total assets 198,126 299,366 Total liabilities 47,402 48,759 Total stockholders' equity 150,724 250,607 Consolidated Statement of Operations(in thousands, except share and per share data) Three Months Ended Twelve Months Ended December 31, December 31, 2023 2022 2023 2022 Operating expenses: Research and development $20,897 $17,062 $94,315 $64,550 General and administrative 7,227 7,663 31,721 28,868 Total operating expenses $28,124 $24,725 $126,036 $93,418 Loss from operations $(28,124) $(24,725) $(126,036) $(93,418)Other income: Interest income 1,863 814 8,173 1,324 Total other income 1,863 814 8,173 1,324 Net loss $(26,261) $(23,911) $(117,863) $(92,094)Net loss per share attributable to common stockholders, basic and diluted $(0.39) $(0.53) $(1.75) $(2.33)Weighted-average common shares outstanding, basic and diluted 67,839,463 45,394,089 67,191,973 39,551,420 Contact:Investors & MediaSarah Spencer +1 857-242-6076sspencer@vorbio.com

– Significant progress made in the development of the PRGN-2012 AdenoVerse immunotherapy for the treatment of RRP; Precigen plans to submit a BLA under an accelerated approval pathway in the second half of 2024; ramping up commercial readiness activities for a potential launch in 2025 – – Precigen's PRGN-2012 received the first Breakthrough Therapy Designation and accelerated approval pathway from the FDA for the treatment of RRP – – Precigen received IND clearance for a randomized Phase 2 study of PRGN-2009 AdenoVerse immunotherapy in combination with pembrolizumab in HPV-associated recurrent/metastatic cervical cancer; study now active and recruiting patients – – Interim data from the ongoing Phase 1b study of PRGN-3006 UltraCAR-T in relapsed/refractory AML anticipated in the second half of 2024 – – Preliminary data from the Phase 1 study of PRGN-3007 next generation UltraCAR-T in ROR1+ advanced cancers anticipated in the second half of 2024 – – Cash, cash equivalents, short-term and long-term investments totaled $62.9 million as of December 31, 2023 – – Continued focus on cost containment resulted in a reduction in SG&A costs of 16% for the twelve months ended December 31, 2023, compared to the prior year period – GERMANTOWN, Md., March 19, 2024 /PRNewswire/ -- Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced full year 2023 financial results and business updates. "2024 is poised to be a transformative year for Precigen, as we are on track to present pivotal Phase 2 data in the second quarter and submit a BLA for PRGN-2012 in the second half, a milestone bolstered by the Breakthrough Therapy Designation and accelerated approval pathway granted by the FDA," said Helen Sabzevari, PhD, President and CEO of Precigen. "We are preparing our manufacturing facility and commercial operations in anticipation of the launch of PRGN-2012 in 2025. We are also looking forward to exciting updates from our UltraCAR-T programs, which offer a novel approach compared to traditional CAR-T therapies and have garnered significant interest from potential partners due to the safety, preliminary efficacy, and manufacturing advantages." "With multiple value inflection points anticipated in 2024, we remain steadfastly committed to a strategy of sound financial management," said Harry Thomasian Jr., CFO of Precigen. "We are evaluating various financing opportunities to strengthen our balance sheet as we prepare our lead asset PRGN-2012 for potential commercial launch in 2025." Key Program Highlights AdenoVerse™ Immunotherapies PRGN-2012 in RRP: PRGN-2012 is an investigational off-the-shelf AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 has received Breakthrough Therapy Designation and Orphan Drug Designation from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission. PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP (clinical trial identifier: NCT04724980). PRGN-2012 demonstrated strong efficacy and a favorable safety profile in the Phase 1 portion of the study with 50% of patients (N=12) in durable and ongoing Complete Response more than two years after PRGN-2012 treatment. Results of the Phase 1 portion of the Phase 1/2 study were published in the peer-reviewed journal, Science Translational Medicine, a leading publication from the American Association for the Advancement of Science (AAAS). Enrollment and dosing in the Phase 2 portion of the study is complete and a Phase 2 data presentation is anticipated in the second quarter of 2024. The Company has received agreement from the FDA that PRGN-2012 is eligible for consideration of a rolling Biologics License Application (BLA) review. A planned BLA submission under an accelerated approval pathway is anticipated in the second half of 2024. Commercial readiness preparations are underway for a potential launch in 2025. PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is an investigational off-the-shelf AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-associated cancers. The Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) is enrolling patients (clinical trial identifier: NCT05996523). The Phase 2 randomized, open-label study of PRGN-2009 in combination with pembrolizumab in patients with HPV-associated recurrent/metastatic cervical cancer is active and recruiting patients (clinical trial identifier: NCT06157151). UltraCAR-T® Cell Therapies PRGN-3006 in AML/MDS: PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a safety/kill switch. PRGN-3006 has been granted Orphan Drug Designation in patients with acute myeloid leukemia (AML) and Fast Track Designation in patients with relapsed/refractory (r/r) AML by the FDA. PRGN-3006 is currently under investigation in a Phase 1b dose expansion clinical trial (clinical trial identifier: NCT03927261) for the treatment of patients with r/r AML or higher-risk myelodysplastic syndromes (MDS). The first-in-human Phase 1 dose escalation study data show that PRGN-3006 was well-tolerated with no dose-limiting toxicities (DLTs) and a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients infused following lymphodepletion. An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024. PRGN-3005 in Ovarian Cancer: PRGN-3005 is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a safety/kill switch. The Phase 1b dose expansion portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT03907527). PRGN-3007 in Advanced ROR1+ Hematological and Solid Tumors: PRGN-3007, based on the next generation UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a safety/kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression. The Phase 1 dose escalation portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT05694364). A preliminary Phase 1 dose escalation data presentation is anticipated by the end of 2024. Financial Highlights Cash, cash equivalents, short-term and long-term investments totaled $62.9 million as of December 31, 2023. Selling, general, and administrative (SG&A) costs decreased versus the prior year, 16% for the twelve months ended December 31, 2023. Full Year 2023 Financial Results Compared to Prior Year Period Research and development expenses increased $1.4 million, or 3.1%, compared to year ended December 31, 2022. Salaries, benefits, and other personnel costs increased $2.8 million due to an increase in the hiring of employees to support the growth in the Company's development activities, and to a lesser extent, increases in salaries of our continuing employees. These increases were offset by less expenses incurred related to preclinical research programs for the comparable period. SG&A expenses decreased $7.6 million, or 15.8%, compared to the year ended December 31, 2022. This decrease was primarily driven by a reduction in professional fees of $6.5 million, due to decreased legal fees associated with certain litigation matters, and $0.7 million decreased insurance-related premiums. Total revenues decreased $20.7 million, or 76.9%, compared to the year ended December 31, 2022. Collaboration and licensing revenues decreased $14.6 million, or 99.5%, compared to the year ended December 31, 2022, primarily due to the prior year period non-cash recognition of revenue related to historical collaboration agreements for which revenue was previously deferred. Product and services revenues decreased $5.9 million, or 48.8%, compared to the year ended December 31, 2022. This decrease is related to reductions in services performed at Exemplar as well as the recognition of revenue in the first quarter of 2022 related to agreements for which revenue was previously deferred that did not occur in 2023. Total other income, net, increased $8.5 million, compared to the year ended December 31, 2022. This was primarily due to $6.3 million in reduced interest expense associated with the Convertible Notes as they were fully retired in the second quarter of 2023, and $3.1 million increased interest income due to higher interest rates on investments. This increase was partially offset by a $0.9 million decrease in gain recorded on the early retirement of a portion of our Convertible Notes compared to the year ended December 31, 2022. The Company recorded a $10.4 million impairment charge in the fourth quarter of 2023 related to its Exemplar subsidiary as a result of the Company's annual goodwill impairment test. Loss from continuing operations was $95.9 million, or $(0.39) per basic and diluted share, compared to loss from continuing operations of $79.8 million, or $(0.40) per basic and diluted share, in year ended December 31, 2022. Precigen: Advancing Medicine with Precision™ Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit or follow us on X @Precigen, LinkedIn or YouTube. UltraCAR-T® UltraCAR-T is a multigenic autologous CAR-T platform that utilizes Precigen's advanced non-viral Sleeping Beauty system to simultaneously express an antigen-specific CAR to specifically target tumor cells, mbIL15 for enhanced in vivo expansion and persistence, and a kill switch to conditionally eliminate CAR-T cells for a potentially improved safety profile. Precigen has advanced the UltraCAR-T platform to address the inhibitory tumor microenvironment by incorporating a novel mechanism for intrinsic checkpoint blockade without the need for complex and expensive gene editing techniques. UltraCAR-T investigational therapies are manufactured via Precigen's overnight manufacturing process using the proprietary UltraPorator® electroporation system at the patient's medical center and administered to patients only one day following gene transfer. The overnight UltraCAR-T manufacturing process does not use viral vectors and does not require ex vivo activation and expansion of T cells, potentially addressing major limitations of current T cell therapies. UltraCAR-T® Clinical Program Precigen's UltraCAR-T platform is currently under clinical investigation for hematological and solid tumors, including a Phase 1/1b study of PRGN-3005 UltraCAR-T in patients with advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer (NCT03907527), a Phase 1/1b study of PRGN-3006 UltraCAR-T in patients with relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS) (NCT03927261) and a Phase 1/1b study of PRGN-3007 UltraCAR-T incorporating PD-1 checkpoint inhibition in patients with ROR1-positive (ROR1+) chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), acute lymphoblastic leukemia (ALL), diffuse large B-cell lymphoma (DLBCL) and triple negative breast cancer (TNBC) (NCT05694364). PRGN-3006 UltraCAR-T has been granted Orphan Drug Designation and Fast Track Designation in patients with AML by the US Food and Drug Administration (FDA). UltraCAR-T® Library Approach Precigen's UltraCAR-T library approach is designed to transform the personalized cell therapy landscape for cancer patients. Precigen's goal is to develop and validate a library of non-viral plasmids to target tumor-associated antigens. Enabled by design and manufacturing advantages of UltraCAR-T, coupled with the capabilities of the UltraPorator® system, Precigen is working to empower medical centers to deliver personalized, autologous UltraCAR-T treatment with overnight manufacturing to any cancer patient. Based on the patient's cancer indication and biomarker profile, one or more non-viral plasmids would be selected from the library to build a personalized UltraCAR-T treatment. After initial treatment, this approach has the potential to allow for redosing of UltraCAR-T targeting the same or new tumor-associated antigen(s) based on the treatment response and the changes in antigen expression of the patient's tumor. Precigen believes that the combination of the advanced UltraVector® DNA construction platform and the ease of overnight manufacturing gives this library approach a proprietary advantage over traditional T-cell therapies. UltraPorator® The UltraPorator system is an exclusive device and proprietary software solution for the scale-up of rapid and cost-effective manufacturing of UltraCAR-T therapies and potentially represents a major advancement over current electroporation devices by significantly reducing the processing time and contamination risk. The UltraPorator device is a high-throughput, semi-closed electroporation system for modifying T cells using Precigen's proprietary non-viral gene transfer technology. UltraPorator is being utilized for clinical manufacturing of Precigen's investigational UltraCAR-T therapies in compliance with current good manufacturing practices. AdenoVerse™ Immunotherapy Precigen's AdenoVerse immunotherapy platform utilizes a library of proprietary adenovectors for the efficient gene delivery of therapeutic effectors, immunomodulators, and vaccine antigens designed to modulate the immune system. Precigen's gorilla adenovectors, part of the AdenoVerse library, have potentially superior performance characteristics as compared to current competition. AdenoVerse immunotherapies have been shown to generate high-level and durable antigen-specific T-cell immune responses as well as an ability to boost these responses via repeat administration. Superior performance characteristics and high yield manufacturing of AdenoVerse vectors leveraging UltraVector® technology allows Precigen to engineer cutting-edge investigational gene therapies to treat complex diseases. AdenoVerse™ Immunotherapy Clinical Program Precigen's AdenoVerse immunotherapy platform is currently under clinical investigation in a Phase 1/2 study of PRGN-2009 AdenoVerse immunotherapy alone or in combination with an anti-PDL1/TGF-Beta Trap in patients with HPV-associated cancers (NCT04432597), a Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) (NCT05996523), a Phase 2 study of PRGN-2009 AdenoVerse immunotherapy in combination with pembrolizumab in patients with recurrent or metastatic cervical cancer (NCT06157151), and a Phase 1/2 study of PRGN-2012 AdenoVerse immunotherapy in patients with recurrent respiratory papillomatosis (RRP) (NCT04724980). PRGN-2012 has been granted Orphan Drug Designation and Breakthrough Therapy Designation in patients with RRP by the FDA and Orphan Drug Designation by the European Commission. Trademarks Precigen, UltraCAR-T, UltraPorator, AdenoVerse, UltraVector and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners. Cautionary Statement Regarding Forward-Looking Statements Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon the Company's current expectations and projections about future events and generally relate to plans, objectives, and expectations for the development of the Company's business, including the timing and progress of preclinical studies, clinical trials, discovery programs and related milestones, the promise of the Company's portfolio of therapies, and in particular its CAR-T and AdenoVerse therapies. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release. The Company has no obligation to provide any updates to these forward-looking statements even if its expectations change. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. For further information on potential risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in the Company's most recent Annual Report on Form 10-K and subsequent reports filed with the Securities and Exchange Commission. Investor Contact: Steven M. Harasym Vice President, Investor Relations Tel: +1 (301) 556-9850 [email protected] Media Contacts: Donelle M. Gregory [email protected] Glenn Silver Lazar-FINN Partners [email protected] SOURCE Precigen, Inc.