排名前五的靶点	数量

IL-12R(白细胞介素-12受体)	1
EGFR x Hyaluronidase x VEGFR2	1
VDR(维生素D受体)	1
LOX(赖氨酰氧化酶)	1
AGER(晚期糖基化终产物特异性受体)	1

关联

项与 University of Rochester 相关的药物

Sodium Bicarbonate

碳酸氢钠

靶点-

作用机制-

在研机构

University of Rochester School of Medicine & Dentistry [+2]

原研机构

华中药业股份有限公司

在研适应症

酸中毒 [+2]

非在研适应症

发作性紧张型头痛

最高研发阶段批准上市

首次获批国家/地区

中国

首次获批日期1981-01-01

PXS-5505

靶点

LOX

作用机制

赖氨酰氧化酶抑制剂

在研机构

Syntara Ltd. [+1]

原研机构

Syntara Ltd.

在研适应症

瘢痕 [+7]

非在研适应症-

最高研发阶段临床2期

首次获批国家/地区-

首次获批日期-

Encapsulated pancreatic cancer vaccine (Therapyx)

靶点

IL-12R

作用机制

IL-12R刺激剂

在研机构

TherapyX, Inc. [+1]

原研机构

TherapyX, Inc.

在研适应症

局部晚期胰腺腺癌 [+2]

非在研适应症-

最高研发阶段临床1期

首次获批国家/地区-

首次获批日期-

900

项与 University of Rochester 相关的临床试验

NCT05744869

/ Not yet recruitingN/AIIT

School-based Asthma Therapy to Advance Health Equity Among Historically Marginalized Children

The goal of this research trial is to: 1) Solidify a population health SBAT implementation strategy with our longstanding community collaborators, 2) Perform a district-wide hybrid type 3, stepped-wedge, cluster randomized trial, and 3) Assess the supportive resource utilization and essential features of SBAT to extend sustainability and fidelity in a cost-effective manner.

开始日期2026-08-01

申办/合作机构

University of Rochester

[+3]

NCT06800053

/ SuspendedN/AIIT

FOCUS ON US: Adapting the FOCUS Program for Sexual and Gender Minority (SGM) Cancer Patients and Caregivers

Our aim for this study is to assess the feasibility and acceptability and explore potential outcomes of the adapted FOCUS On Us intervention in a two-arm pilot trial. The investigators will randomize 80 sexual and/or gender minority (SGM; e.g., lesbian, gay, bisexual, transgender, queer, etc.) cancer patients and their ≥80 informal caregivers (total N≥160) to either FOCUS On Us (adapted from the evidence-based FOCUS program) or a waitlist control.

开始日期2026-04-01

申办/合作机构

University of Rochester

[+2]

NCT06352086

/ WithdrawnN/AIIT

Understanding Visual Processing After Occipital Stroke

The purpose of this study is to investigate how visual orientation discrimination and metacognition (i.e., perceptual confidence) are affected by occipital stroke that causes hemianopia and quadrantanopia in adults. This research will provide insight as to how the residual visual system, which not directly damaged by the occipital stroke, processes orientation (assayed in terms of orientation discrimination) and metacognition (by measuring perceptual confidence for orientation discrimination). These measures will be used to refine computational models that attempt to explain how the brain copes with loss of primary visual cortex (V1) as a result of stroke. This knowledge is essential to devise more effective visual rehabilitation therapies for patients suffering from occipital strokes.

开始日期2025-12-01

申办/合作机构

University of Rochester

100 项与 University of Rochester 相关的临床结果

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0 项与 University of Rochester 相关的专利（医药）

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73,150

项与 University of Rochester 相关的文献（医药）

2025-12-31·Emerging Microbes & Infections

A polyvalent DNA prime with matched polyvalent protein/GLA-SE boost regimen elicited the most robust and broad IgG and IgG3 V1V2 binding antibody and CD4+ T cell responses among 13 HIV vaccine trials

Article

作者: Laher, Fatima ; Gilbert, Peter B. ; Zhang, Lu ; Andersen-Nissen, Erica ; Liu, Shuying ; Hosseinipour, Mina C. ; Mngadi, Kathryn ; Giorgi, Elena E. ; Carpp, Lindsay N. ; Keefer, Michael ; Rerks-Ngarm, Supachai ; Dintwe, One ; Tomaras, Georgia D. ; Garrett, Nigel ; Williams, LaTonya D. ; Pantaleo, Giuseppe ; Corey, Lawrence ; Seaton, Kelly E. ; Sawant, Sheetal S. ; Baden, Lindsey R. ; Tomaka, Frank ; Chirenje, Mike ; Ake, Julie A. ; Bekker, Linda-Gail ; Nitayaphan, Sorachai ; Vasan, Sandhya ; McElrath, M. Juliana ; Frank, Ian ; Goepfert, Paul A. ; Gray, Glenda E. ; Pitisuttithum, Punnee ; Grunenberg, Nicole ; De Rosa, Stephen ; Moodie, Zoe ; Lu, Shan ; Chen, Shiyu ; Kublin, James G. ; Li, Shuying Sue ; Heptinstall, Jack

Developing an effective HIV vaccine is a momentous challenge. An exceptionally wide range of candidate HIV vaccines have been tested, yet many were poorly immunogenic, and of the select few that advanced into efficacy trials, only one demonstrated any efficacy. Here we report the results of the largest-scale cross-protocol immunogenicity comparison to date: 13 HIV vaccine trials (including 36 vaccine regimens) conducted across nine countries worldwide, strengthened by standardized trial designs, validated assays in centralized laboratories, and harmonized immunogenicity endpoints - providing an objective approach to identify the HIV vaccine candidate(s) with the best immunogenicity. A polyvalent DNA prime + protein boost regimen (HVTN 124) including Env immunogens of four subtypes, matched between prime and boost, achieved the best anti-V1V2 antibody responses by a large margin and also induced high CD4+ T-cell responses - two key immune responses implicated in HIV vaccine protection. Our results provide strong support to test this promising HIV vaccine design in more advanced phase clinical trials and will also guide the future design of additional HIV vaccines.Trial registration: ClinicalTrials.gov identifier: NCT01799954..Trial registration: ClinicalTrials.gov identifier: NCT02109354..Trial registration: ClinicalTrials.gov identifier: NCT02404311..Trial registration: ClinicalTrials.gov identifier: NCT02207920..Trial registration: ClinicalTrials.gov identifier: NCT02296541..Trial registration: ClinicalTrials.gov identifier: NCT03284710..Trial registration: ClinicalTrials.gov identifier: NCT02915016..Trial registration: ClinicalTrials.gov identifier: NCT02997969..Trial registration: ClinicalTrials.gov identifier: NCT03122223..Trial registration: ClinicalTrials.gov identifier: NCT03409276..Trial registration: ClinicalTrials.gov identifier: NCT02968849..Trial registration: ClinicalTrials.gov identifier: NCT03060629..Trial registration: ClinicalTrials.gov identifier: NCT00223080..

2025-12-31·ANNALS OF MEDICINE

Who feels safe calling 911: are prior experiences of anti-Black racial discrimination associated with hesitancy seeking emergency medical services in the event of accidental drug overdose? – a study protocol

Article

作者: Entrup, Parker ; Hall, O. Trent ; Garcia, Stephanie ; Jegede, Oluwole ; Martel, Jose Perez ; Jordan, Ayana ; Joseph, Lucy ; Trimble, Candice ; Grayson, Sydney ; Mathis, Myra ; Tetrault, Jeanette

BACKGROUND:

Racial discrimination is associated with health disparities among Black Americans, a group that has experienced an increase in rates of fatal drug overdose. Prior research has found that racial discrimination in the medical setting may be a barrier to addiction treatment. Nevertheless, it is unknown how experiences of racial discrimination might impact engagement with emergency medical services for accidental drug overdose. This study will psychometrically assess a new measure of hesitancy in seeking emergency medical services for accidental drug overdose and examine prior experiences of racial discrimination and group-based medical mistrust as potential corollaries of this hesitancy.

METHOD:

Cross-sectional survey of 200 Black adults seeking treatment for substance-use-related medical problems (i.e. substance use disorder, overdose, infectious complications of substance use, etc.). Participants will complete a survey including sociodemographic information, the Discrimination in Medical Settings Scale, Everyday Discrimination Scale, Group-Based Medical Mistrust Scale, and an original questionnaire measuring perceptions of and prior engagement with emergency services for accidental drug overdose. Analyses will include exploratory factor analysis, Cronbach's alpha, and non-parametric partial correlations controlling for age, gender, income, and education.

CONCLUSIONS:

This article describes a planned cross-sectional survey of Black patients seeking treatment for substance use related health problems. Currently, there is no validated instrument to measure hesitancy in seeking emergency medical services for accidental drug overdose or how experiences of racial discrimination might relate to such hesitancy. Results of this study may provide actionable insight into medical discrimination and the rising death toll of accidental drug overdose among Black Americans.

2025-12-01·JOURNAL OF MAMMARY GLAND BIOLOGY AND NEOPLASIA

Recurrent Nipple Duct Obstruction in Two Breastfeeding Patients: A Case Report and Discussion of the Underlying Pathophysiology

Article

作者: Rosen-Carole, Casey ; Young, Bridget ; Sadovnikova, Anna ; Greenman, Susan

Abstract:

Nipple pain is a common reason for premature breastfeeding cessation. There exists anecdotal evidence that one cause of lactational nipple pain is a ductal obstruction, but there is no published literature describing this phenomenon. Herein we present two case reports for two patients who experienced breast and nipple pain concurrent with milk flow reduction. Both patients removed a small stone-like obstructing object from their nipple; this action was painful for one of the patients, resulting in immediate release of milk and relief from breast pain. Both patients experienced recurrence of stone formation in their nipple ducts. We analyzed the mineral composition of the obstructing objects and breast milk using inductively coupled mass spectroscopy. We use literature on teat obstructions in dairy cows and dacryolith and sialolith formation to propose hypotheses as to how the formation of obstructing objects in milk ducts might occur. Future research directions for determining the pathophysiology, clinical presentation, and management of human nipple duct obstructions are discussed.

297

项与 University of Rochester 相关的新闻（医药）

2025-05-19

·微信

疗效持续24月！腓骨肌萎症AKR1B1抑制剂2/3期临床完整结果公布

5月18日（当地时间），Applied Therapeutics宣布其AKR1B1抑制剂(醛糖还原酶抑制剂)Govorestat治疗SORD缺乏症（山梨醇脱氢酶缺乏症，为腓骨肌萎缩症一种亚型）的2/3期临床研究（INSPIRE）12个月完整结果及18、24月顶线结果将在2025年周围神经学会（PNS）口头汇报。INSPIRE研究为旨在评估Govorestat治疗CMT-SORD患者有效性和安全性的随机、双盲、安慰剂对照的2/3期临床研究。56例CMT-SORD患者以2：1的比例随机接受每日一次Govorestat或安慰剂治疗。主要临床终点是12 个月时的10米步行跑步测试（10MWRT），该测试不再包含在CMT-FOM（腓骨肌萎缩症相关测评量表）综合评分中。其他关键试验目标包括血液山梨醇水平影响、山梨醇水平与临床结果的相关性以及CMT-健康指数（HI）。1、12月研究完整结果：对截止2024年2月的49例患者进行重新中期分析：山梨醇浓度绝对降低与10MWRT （p=0.031）和CMT-FOM下肢结构域（10MWRT、6MWT、爬楼梯、背屈、坐立）（p=0.037）的变化之间存在统计学显著相关性。主要终点10MWRT无统计学意义（p=0.457）。不过自INSPIRE研究开始以来，10MWRT已从CMT-FOM中删除，现在CMT-FOM仅由 11个部分组成。12个月时显示关键次要终点CMT-HI统计学显著改善（p=0.039）。12个月时山梨醇变化百分比与CMT-HI变化之间的总分以及活动度和平衡领域之间存在统计学上显著相关性。血液山梨醇水平具有统计学意义的降低（p<0.001）；总体上仍然安全且耐受性良好，组间的不良事件发生率相似。2、18和24月顶线结果：截至2024年10月11日，分别有24和15例名患者接受18个月和24个月评估。接受Govorestat持续治疗18个月和24个月顶线结果如下：24个月时MRI显示Govorestat治疗延缓疾病进展。结果改善维持24个月，包括在第12个月时CMT-HI的变化。18个月和 24个月山梨醇维持减少。24个月总体上保持安全且耐受性良好。CMT-SORD是一种罕见的进行性神经肌肉疾病，由山梨醇脱氢酶的基因突变引起，山梨醇脱氢酶负责山梨醇的代谢。在2020年之前，患者症状诊断为遗传原因不明的2型CMT或远端遗传性运动神经病变。在2020年进行全基因组测序计划后，CMT-SORD被确定为CMT（腓骨肌萎缩症）的一种亚型。Govorestat（AT-007，CAS：2170729-29-8）是一种具有中枢神经系统（CNS）渗透性的醛糖还原酶抑制剂，目前正被开发用于治疗几种罕见的神经系统疾病，包括经典型GAL、SORD缺乏症和磷酸甘露糖变位酶2-先天性糖基化障碍（PMM2-CDG）。FDA曾授予GAL孤儿药资格、儿科罕见病资格和快速通道资格，EMA授予GAL和SORD缺乏症两个适应症孤儿药资格。2024年11月，其治疗经典型半乳糖血症（GAL）的NDA被FDA拒绝，识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

临床结果临床2期

2025-04-21

·Business Wire

United Therapeutics to Feature Clinical Data Across its Commercial and Development Portfolio at the American Thoracic Society 2025 International Conference

SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that recent research across its commercial and development portfolio will be presented at the American Thoracic Society (ATS) International Conference in San Francisco on May 16-21, 2025. At ATS, United Therapeutics is sponsoring several events including the Respiratory Innovation Summit and the ATS Research Program Benefit Networking Event, and the company is hosting the Jenesis Innovative Research Awards™ Ceremony. “Our series of posters and presentations at ATS will showcase the most recent findings on several products across our core portfolio and ongoing development pipeline,” said Andrew Nelsen, PharmD, Vice President, Global Medical Affairs at United Therapeutics. “We are pleased to present additional interim efficacy data from the ADVANCE EXTENSION open-label study evaluating ralinepag for pulmonary arterial hypertension, which has the potential, through once-daily dosing, to fundamentally change the positioning of prostacyclins in the treatment paradigm.” Posters, mini-symposia, and discussion sessions include: Thematic poster session. Sunday, May 18, 11:30 a.m. to 1:15 p.m. PT: A48/P1624 – TETON Phase 3 Clinical Trials of Inhaled Treprostinil in the Treatment of Idiopathic Pulmonary Fibrosis: Annual Update of Preliminary Baseline Data. Presented by Steven D. Nathan, M.D., Inova Fairfax Hospital. Thematic poster session. Sunday, May 18, 11:30 a.m. to 1:15 p.m. PT: A48/P1623 – TETON-PPF Clinical Trial of Inhaled Treprostinil for the Treatment of Progressive Pulmonary Fibrosis: Preliminary Baseline Demographics. Presented by Steven D. Nathan, M.D., Inova Fairfax Hospital. Poster discussion session. Sunday, May 18, 2:15 p.m. to 4:15 p.m. PT: A104/908 – Expert Nurse Insights on the Future of Parenteral Prostacyclin Pumps for Pulmonary Arterial Hypertension. Presented by Lori Reed, N.P., Piedmont Healthcare. Rapid abstract poster discussion session. Monday, May 19, 9:15 a.m. to 11:15 a.m. PT: B26/410 – The Minimal Important Difference in NT-proBNP in Various Forms of Pulmonary Hypertension. Presented by Stephen Mathai, M.D., MHS, Johns Hopkins School of Medicine. Mini Symposium. Monday, May 19, 3:15 p.m. to 3:27 p.m. PT: B96 – Ralinepag Treatment to Achieve and Maintain REVEAL Lite 2 Low Risk Status: Interim Results of ADVANCE EXTENSION. Presented by Ray Benza, M.D., Icahn School of Medicine at Mount Sinai Center. Thematic poster session. Tuesday, May 20, 11:30 a.m. to 1:15 p.m. PT: C58/P1412 – Hemodynamic Phenotypes of Interstitial Lung Disease Patients Enrolled in the PHINDER Study. Presented by Debabrata Bandyopadhyay, MBBS, M.D., FCCP, FRCP, FACP, University of South Florida. Thematic poster session. Tuesday, May 20, 11:30 a.m. to 1:15 p.m. PT: C58/P1403 – Screening of Pulmonary Hypertension in Interstitial Lung Disease in the PHINDER Study: A Comparison of Physician Gestalt with Hemodynamics. Presented by Sandeep Sahay, M.D., Houston Methodist Hospital. Thematic poster session. Tuesday, May 20, 11:30 to 1:15 p.m. PT: C57/P1390 – Efficacy and Safety of Oral Treprostinil in Intermediate-High Risk Pulmonary Arterial Hypertension: A FREEDOM-EV Subgroup Analysis. Presented by Jim White, M.D., Ph.D., University of Rochester. Thematic poster session. Tuesday, May 20, 11:30 a.m. to 1:15 p.m. PT: C57/P1393 – Thrombocytopenia Associated with Epoprostenol and Treprostinil Alone and in Combination with Sotatercept. Presented by Mardi Gomberg-Maitland, M.D., M.Sc., George Washington University. Hosted/Sponsored events include: The 2025 Respiratory Innovation Summit, Friday, May 16 and Saturday, May 17. Andrew Nelsen, PharmD, United Therapeutics, will deliver a presentation at 1:00 p.m. PT on Saturday. The ATS Research Program Benefit, Saturday, May 17, 6:00 p.m. to 7:30 p.m. PT. The benefit will be held at the San Francisco Marriot Marquis. The Jenesis Innovative Research Awards Ceremony, Sunday, May 18, 6:30 p.m. to 9:30 p.m. PT. The ceremony will be held at the InterContinental Mark Hopkins San Francisco. United Therapeutics: Enabling Inspiration At United Therapeutics, our vision and mission are one. We use our enthusiasm, creativity, and persistence to innovate for the unmet medical needs of our patients and to benefit our other stakeholders. We are bold and unconventional. We have fun, we do good. We are the first publicly-traded biotech or pharmaceutical company to take the form of a public benefit corporation (PBC). Our public benefit purpose is to provide a brighter future for patients through (a) the development of novel pharmaceutical therapies; and (b) technologies that expand the availability of transplantable organs. You can learn more about what it means to be a PBC here: unither.com/pbc. Forward-Looking Statements Statements included in this press release that are not historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, our efforts to innovate for the unmet medical needs of our patients, to benefit our other stakeholders, and to pursue our public benefit purpose of developing novel pharmaceutical therapies and technologies that expand the availability of transplantable organs. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language, and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of April 21, 2025, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason. JENESIS INNOVATIVE RESEARCH AWARDS is a trademark of United Therapeutics Corporation.

临床3期

2025-04-21

·ir.unither.com

United Therapeutics to Feature Clinical Data Across its Commercial and Development Portfolio at the American Thoracic Society 2025 International Conference

Interim long-term data from the ongoing ralinepag phase 3 ADVANCE EXTENSION open-label study to be presented at a mini symposium An annual update of the preliminary baseline data from the TETON phase 3 studies of inhaled treprostinil in idiopathic pulmonary fibrosis will be presented at a poster session SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that recent research across its commercial and development portfolio will be presented at the American Thoracic Society (ATS) International Conference in San Francisco on May 16-21, 2025. At ATS, United Therapeutics is sponsoring several events including the Respiratory Innovation Summit and the ATS Research Program Benefit Networking Event, and the company is hosting the Jenesis Innovative Research Awards™ Ceremony. “Our series of posters and presentations at ATS will showcase the most recent findings on several products across our core portfolio and ongoing development pipeline,” said Andrew Nelsen, PharmD,Vice President, Global Medical Affairs at United Therapeutics. “We are pleased to present additional interim efficacy data from the ADVANCE EXTENSION open-label study evaluating ralinepag for pulmonary arterial hypertension, which has the potential, through once-daily dosing, to fundamentally change the positioning of prostacyclins in the treatment paradigm.” Posters, mini-symposia, and discussion sessions include: Thematic poster session. Sunday, May 18, 11:30 a.m. to 1:15 p.m. PT: A48/P1624 – TETON Phase 3 Clinical Trials of Inhaled Treprostinil in the Treatment of Idiopathic Pulmonary Fibrosis: Annual Update of Preliminary Baseline Data. Presented by Steven D. Nathan, M.D., Inova Fairfax Hospital. Thematic poster session. Sunday, May 18, 11:30 a.m. to 1:15 p.m. PT: A48/P1623 – TETON-PPF Clinical Trial of Inhaled Treprostinil for the Treatment of Progressive Pulmonary Fibrosis: Preliminary Baseline Demographics. Presented by Steven D. Nathan, M.D., Inova Fairfax Hospital. Poster discussion session. Sunday, May 18, 2:15 p.m. to 4:15 p.m. PT: A104/908 – Expert Nurse Insights on the Future of Parenteral Prostacyclin Pumps for Pulmonary Arterial Hypertension. Presented by Lori Reed, N.P., Piedmont Healthcare. Rapid abstract poster discussion session. Monday, May 19, 9:15 a.m. to 11:15 a.m. PT: B26/410 – The Minimal Important Difference in NT-proBNP in Various Forms of Pulmonary Hypertension. Presented by Stephen Mathai, M.D., MHS, Johns Hopkins School of Medicine. Mini Symposium. Monday, May 19, 3:15 p.m. to 3:27 p.m. PT: B96 – Ralinepag Treatment to Achieve and Maintain REVEAL Lite 2 Low Risk Status: Interim Results of ADVANCE EXTENSION. Presented by Ray Benza, M.D., Icahn School of Medicine at Mount Sinai Center. Thematic poster session. Tuesday, May 20, 11:30 a.m. to 1:15 p.m. PT: C58/P1412 – Hemodynamic Phenotypes of Interstitial Lung Disease Patients Enrolled in the PHINDER Study. Presented by Debabrata Bandyopadhyay, MBBS, M.D., FCCP, FRCP, FACP, University of South Florida. Thematic poster session. Tuesday, May 20, 11:30 a.m. to 1:15 p.m. PT: C58/P1403 – Screening of Pulmonary Hypertension in Interstitial Lung Disease in the PHINDER Study: A Comparison of Physician Gestalt with Hemodynamics. Presented by Sandeep Sahay, M.D., Houston Methodist Hospital. Thematic poster session. Tuesday, May 20, 11:30 to 1:15 p.m. PT: C57/P1390 – Efficacy and Safety of Oral Treprostinil in Intermediate-High Risk Pulmonary Arterial Hypertension: A FREEDOM-EV Subgroup Analysis. Presented by Jim White, M.D., Ph.D., University of Rochester. Thematic poster session. Tuesday, May 20, 11:30 a.m. to 1:15 p.m. PT: C57/P1393 – Thrombocytopenia Associated with Epoprostenol and Treprostinil Alone and in Combination with Sotatercept. Presented by Mardi Gomberg-Maitland, M.D., M.Sc., George Washington University. Hosted/Sponsored events include: The 2025 Respiratory Innovation Summit, Friday, May 16 and Saturday, May 17. Andrew Nelsen, PharmD, United Therapeutics, will deliver a presentation at 1:00 p.m. PT on Saturday. The ATS Research Program Benefit, Saturday, May 17, 6:00 p.m. to 7:30 p.m. PT. The benefit will be held at the San Francisco Marriot Marquis. The Jenesis Innovative Research Awards Ceremony, Sunday, May 18, 6:30 p.m. to 9:30 p.m. PT. The ceremony will be held at the InterContinental Mark Hopkins San Francisco. United Therapeutics: Enabling Inspiration At United Therapeutics, our vision and mission are one. We use our enthusiasm, creativity, and persistence to innovate for the unmet medical needs of our patients and to benefit our other stakeholders. We are bold and unconventional. We have fun, we do good. We are the first publicly-traded biotech or pharmaceutical company to take the form of a public benefit corporation (PBC). Our public benefit purpose is to provide a brighter future for patients through (a) the development of novel pharmaceutical therapies; and (b) technologies that expand the availability of transplantable organs. You can learn more about what it means to be a PBC here: unither.com/pbc. Forward-Looking Statements Statements included in this press release that are not historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, our efforts to innovate for the unmet medical needs of our patients, to benefit our other stakeholders, and to pursue our public benefit purpose of developing novel pharmaceutical therapies and technologies that expand the availability of transplantable organs. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language, and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of April 21, 2025, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason. JENESIS INNOVATIVE RESEARCH AWARDS is a trademark of United Therapeutics Corporation. View source version on businesswire.com: https://www.businesswire.com/news/home/20250421355717/en/ Dewey Steadman at (202) 919-4097 (media/investors) Harry Silvers at (301) 578-1401 (investors) https://ir.unither.com/contact-uthr/ Source: United Therapeutics Corporation

临床3期

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药物(靶点)	适应症	全球最高研发状态

碳酸氢钠	慢性肾病更多	临床2/3期
Encapsulated pancreatic cancer vaccine (Therapyx) ( IL-12R )	胰腺腺癌更多	临床1期
UR241-2 ( IRAK1 x IRAK4 )	成人急性髓性白血病更多	临床前
FPS-ZM1 ( AGER )	阿尔茨海默症更多	临床前
Delphinidin chloride ( EGFR x Hyaluronidase x VEGFR2 )	肿瘤更多	临床前