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Last update 08 Apr 2026

Ganciclovir

Last update 08 Apr 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

2-(6-Amino-purin-9-ylmethoxy)-propane-1,3-diol, 2-amino-9-((1,3-dihydroxypropan-2-yloxy)methyl)-1H-purin-6(9H)-one, 2-amino-9-((1,3-dihydroxypropan-2-yloxy)methyl)-3H-purin-6(9H)-one

+ [24]

Target

DNA polymerase

Action

inhibitors

Mechanism

DNA polymerase inhibitors(DNA polymerase inhibitors)

Therapeutic Areas

Infectious DiseasesEye DiseasesSkin and Musculoskeletal Diseases

Active Indication

Keratitis, HerpeticCytomegalovirus RetinitisCytomegalovirus Infections

Inactive Indication

Acquired Immunodeficiency SyndromeColitisHematopoietic stem cell transplantation+ [4]

Originator Organization

Syntex Corp.

Active Organization

Tanabe Pharma Corp.

Chong Kun Dang Pharmaceutical Corp.

Hainan Poly Pharm. Co., Ltd.

+ [4]

Inactive Organization

Hoffmann-La Roche, Inc.Dr. Gerhard Mann Chem.-pharm. Fabrik GmbHRoche Palo Alto LLC

+ [4]

License Organization-

Drug Highest PhaseApproved

First Approval Date

United States (23 Jun 1989),

Cytomegalovirus InfectionsCytomegalovirus Retinitis

RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea)

Structure/Sequence

Molecular FormulaC9H13N5O4

InChIKeyIRSCQMHQWWYFCW-UHFFFAOYSA-N

CAS Registry82410-32-0

138

Clinical Trials associated with Ganciclovir

NCT07513623

/ Not yet recruitingPhase 2IIT

Systemic and Topical Antiviral Control of Cytomegalovirus Anterior Uveitis: Treatment Outcomes - Trials I and II

The goal of this clinical trial is to compare antiviral treatment strategies for cytomegalovirus (CMV) anterior uveitis - a viral infection causing inflammation inside the front of the eye - in immunocompetent adults aged 18 years and older. The main questions it aims to answer are:
Does oral valganciclovir reduce aqueous humor CMV viral load more effectively than topical ganciclovir 2% eye drops or placebo after 7 days of treatment (Trial I)? Does long-term suppressive antiviral therapy (oral valganciclovir or topical ganciclovir 2% eye drops) reduce the rate of CMV anterior uveitis recurrence over 12 months compared to placebo (Trial II)?
Researchers will compare oral valganciclovir, topical ganciclovir 2% eye drops, and placebo to see if either antiviral treatment reduces viral load and controls eye inflammation more effectively in the short term, and whether long-term antiviral suppression can prevent the disease from coming back after the inflammation has been controlled.
Participants will:
* Undergo anterior chamber paracentesis (removal of a small amount of fluid from the front of the eye) for PCR testing to confirm CMV as the cause of their eye inflammation before enrollment
* Be randomly assigned to receive oral valganciclovir 900 mg twice daily, topical ganciclovir 2% eye drops six times daily, or placebo for 7 days (Trial I), in addition to standard steroid eye drops
* Return for follow-up visits at Day 7 and Day 21 for eye examinations, laboratory blood tests, and a second anterior chamber paracentesis at Day 7 to measure viral load after treatment
* If eye inflammation is controlled after Trial I, be offered enrollment into Trial II, where they will be randomly assigned to long-term suppressive oral valganciclovir, topical ganciclovir 2% eye drops, or placebo for 12 months, with follow-up visits approximately every 2 months and additional visits if inflammation returns

Start Date01 Jul 2026

Sponsor / Collaborator

The University of California, San Francisco

[+2]

NCT07450365

/ Not yet recruitingPhase 4IIT

Evaluation of Long-term Efficacy of 4 to 6-month Course Antiviral Therapy for Neurodevelopmental Impairments Caused by Congenital Cytomegalovirus Infection: A Multicenter, Randomized, Controlled, Non-inferiority Clinical Trial

Title: Evaluation of Long-term Efficacy of 4-month versus 6-month Course Antiviral Therapy for Neurodevelopmental Impairment Caused by Congenital CMV Infection: A Multicenter, Randomized, Controlled, Non-inferiority Clinical Study
1. Background and Rationale：Congenital cytomegalovirus (cCMV) infection is a leading cause of childhood neurodevelopmental disability and sensorineural hearing loss (SNHL). International guidelines, based on evidence from high-income countries, recommend a 6-month antiviral course for symptomatic infection. However, clinical practice in China lags significantly, still adhering to a 3-4 week regimen due to a lack of high-quality domestic evidence. Preliminary data suggest a 4-month course may be non-inferior to the 6-month standard by potentially aligning with the transition from productive to latent infection around 4 months of age. This study aims to address this critical evidence gap.2. Study Objectives：Primary: To evaluate the impact of intermediate (4-month) versus long (6-month) course antiviral therapy on long-term neurodevelopmental outcomes in infants with moderate-to-severe cCMV infection, and to establish a novel diagnostic and therapeutic framework.Secondary: To identify high-risk factors associated with adverse long-term outcomes.3. Study Design and Methods：This is a prospective, multicenter, randomized, open-label, parallel-controlled, non-inferiority clinical trial.Participants: Newborns (≤30 days old) diagnosed with moderate-to-severe cCMV infection. Key exclusion criteria include gestational age <32 weeks, birth weight <1.8 kg, and coexisting genetic/metabolic diseases.Randomization & Intervention: Eligible subjects will be block-randomized 1:1 via a computer-generated sequence.Experimental Group: Receives 4 months of antiviral therapy.Control Group: Receives 6 months of antiviral therapy (current international standard).Both groups receive either intravenous ganciclovir (6 mg/kg, twice daily) or oral valganciclovir (16 mg/kg, twice daily).Sample Size: A total of 150 subjects will be enrolled (60 from the lead center), anticipating 126 evaluable cases at study completion (2 years of age) to demonstrate non-inferiority with a margin (Δ) of 20%, 80% power, and a one-sided alpha of 0.025.4. Evaluation and Endpoints：Primary Outcome: Composite poor neurodevelopmental outcome at 12 months of age, defined as (1) death, or (2) moderate/severe impairment, including cerebral palsy, epilepsy, moderate-to-severe SNHL (>40 dB threshold and/or requiring cochlear implantation), visual impairment, or a score <-2 SD on standardized developmental scales (Griffiths, BSID-II, or Bayley-III).Secondary Outcomes: Include mild neurodevelopmental impairment at 12 months, and poor/mild neurodevelopmental outcomes at 24 months.Assessments: Scheduled follow-ups include clinical, laboratory (hepatic/renal function, CMV DNA load), and instrumental evaluations (neuroimaging, audiology, ophthalmology) during treatment and at 6, 12, and 24 months of age.5. Data Management and Ethics：Data will be managed using a multicenter Electronic Data Capture (EDC) system with double data entry. The study protocol has been approved by the Medical Ethics Committee of Children's Hospital, Zhejiang University School of Medicine. Written informed consent will be obtained from all participants' guardians. The study is scheduled from January 2026 to December 2028.This study will provide crucial high-level evidence to inform optimal antiviral therapy duration for cCMV infection in China, with the goal of improving long-term child health outcomes and reducing disease burden.

Start Date15 Feb 2026

Sponsor / Collaborator

The Children's Hospital of Zhejiang University School of Medicine

[+4]

ChiCTR2600118780

/ Not yet recruitingNot ApplicableIIT

Start Date01 Jan 2026

Sponsor / Collaborator

The Children's Hospital of Zhejiang University School of Medicine

100 Clinical Results associated with Ganciclovir

100 Translational Medicine associated with Ganciclovir

100 Patents (Medical) associated with Ganciclovir

8,913

Literatures (Medical) associated with Ganciclovir

01 Jun 2026·DIAGNOSTIC MICROBIOLOGY AND INFECTIOUS DISEASE

Rapid diagnosis of a mixed pulmonary infection with Rhizopus microsporus, Aspergillus fumigatus, Pneumocystis jirovecii, and Cytomegalovirus in a Lymphoma patient using metagenomic next-generation sequencing: A case report

Article

Author: Qian, Jiao ; Zhu, Qiqi ; Zhang, Qinyi ; Wang, Jiajia ; Wang, Lisha

Immunocompromised patients are at high risk for life-threatening polymicrobial infections, often challenging to diagnose conventionally. We report a 60-year-old male with relapsed angioimmunoblastic T-cell lymphoma, fever, and pancytopenia post-chemotherapy. Chest CT showed scattered inflammation. Bronchoalveolar lavage fluid (BALF) culture grew only Klebsiella aerogenes, but fluorescent staining revealed aseptate hyphae. Metagenomic next-generation sequencing (mNGS) of BALF identified concurrent infections with Rhizopus microsporus, Aspergillus fumigatus, Pneumocystis jirovecii, cytomegalovirus, and SARS-CoV-2 within 48 hours. Targeted therapy with isavuconazole, sulfamethoxazole-trimethoprim, and ganciclovir was promptly initiated. Despite therapy, the patient deteriorated due to profound immunodeficiency and was discharged palliatively. This case highlights mNGS as a rapid diagnostic tool for mixed infections, though clinical correlation remains essential.

01 Jun 2026·EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

LeiCNS-PK3.2: A physiologically based pharmacokinetic model framework for predicting intracellular concentrations of acyclovir and ganciclovir active metabolites

Article

Author: Guo, Tingjie ; de Lange, Elizabeth C M ; Manson, Martijn L ; Sun, Ming

BACKGROUND:

Effective treatment of viral infections in the central nervous system (CNS) requires adequate nucleoside analogs exposure and conversion into phosphorylated metabolites (TP) in brain cells.

AIM:

To develop a CNS PBPK model (LeiCNS-PK3.2) that incorporates active brain cell membrane transport and brain intracellular fluid (brainICF) metabolism to predict and understand target site pharmacokinetics (PK), brainICF active triphosphate (TP) metabolites, and effects of antiviral drugs.

METHOD:

LeiCNS-PK3.0 was extended with a brain cell membrane asymmetry factor (AF) using rat-derived K_p,uu,cell values. Intracellular conversion of acyclovir and ganciclovir into TPs was modelled using in vitro kinetic parameters. LeiCNS-PK3.2 was validated on published rat and human plasma, brain extracellular fluid (brainECF), and cerebrospinal fluid (CSF) data. Simulated PK profiles for parent drug standard dosing regimens (acyclovir 10 mg/kg t.i.d.; ganciclovir 5 mg/kg b.i.d.) were compared to parent-drug profiles in brainECF, and TP metabolite in brainICF. Sensitivity analyses were performed on CNS physiological parameters and K_p,uu,cell values.

RESULTS:

LeiCNS-PK3.2 accurately recaptured the observed data (<2-fold error range). Simulated brainECF parent drug concentrations often remained below reported IC₅₀ values, whereas brainICF TPs had smaller AUCs and longer time to equilibrium compared to acyclovir/ganciclovir in brainECF. Simulated pathological variations during CNS infection showed minimal impact on brainICF TP exposure, with proportional shifts in brainICF TP concentrations when varying K_p,uu,cell. CONCLUSIONS: LeiCNS-PK3.2 can predict CNS target-site exposure of TPs and demonstrates that brainECF acyclovir/ganciclovir concentrations are distinct from brainICF TP levels. It may support intracellular PK/PD target determination to optimize dosing strategies for CNS viral infections.

01 Apr 2026·BIOMEDICAL CHROMATOGRAPHY

Development and Validation of a Liquid Chromatograph–Tandem Mass Spectrometry Method for Quantifying Ganciclovir in Dried Blood Spots for Therapeutic Drug Monitoring

Article

Author: Yano, Ikuko ; Kitahiro, Yumi ; Yamamoto, Kazuhiro ; Omura, Tomohiro ; Tamura, Naoki ; Sakane, Toshiyasu ; Kouyama, Suguru ; Saegusa, Jun ; Itohara, Kotaro

ABSTRACT:

Valganciclovir (VGCV) is the first‐line drug for preemptive therapy of cytomegalovirus (CMV) infection. However, even at standard doses, plasma concentrations of the active metabolite ganciclovir (GCV) show substantial inter‐individual variability. To ensure therapeutic efficacy and minimize adverse effects, therapeutic drug monitoring (TDM) based on the area under the concentration–time curve (AUC) is essential. Yet, conventional TDM via venous blood sampling is invasive and unsuitable for frequent monitoring. In this study, we aimed to develop a simple and minimally invasive method for GCV quantification using dried blood spots (DBS) combined with liquid chromatography–tandem mass spectrometry (LC–MS/MS). The method demonstrated a good linearity over a concentration range of 0.25–16 μg/mL and satisfied the validation criteria for accuracy and precision. It showed acceptable stability for up to 7 days under refrigerated conditions. Methanol was identified as the optimal extraction solvent, allowing for a simplified sample pretreatment without the need for ultrasonic processing. While hematocrit levels affected spot size and quantification accuracy, reliable measurements were obtained within the 30%–50% hematocrit range. The established DBS‐based LC–MS/MS method provides a promising, minimally invasive approach for TDM of GCV in the management of CMV infections.

News (Medical) associated with Ganciclovir

04 Nov 2025

·www.globenewswire.com

Kamada Announces First Patient Enrolled into an Investigator-Initiated Clinical Trial of CYTOGAM® to Prevent Cytomegalovirus in Kidney Transplantation

Post Marketing Study to Evaluate the Role of CYTOGAM in the Reduction of Risk of Late CMV Disease Following the Conclusion of Standardly Prescribed Antiviral Treatment in High-Risk Kidney Transplant RecipientsREHOVOT, Israel, and HOBOKEN, N.J. , Nov. 04, 2025 (GLOBE NEWSWIRE) -- Kamada Ltd. (NASDAQ: KMDA; TASE: KMDA.TA), a global biopharmaceutical company with a portfolio of marketed products indicated for rare and serious conditions and a leader in the specialty plasma-derived field, today announced that the first patient was enrolled into an investigator-initiated post-marketing clinical trial of CYTOGAM® (Cytomegalovirus Immune Globulin Intravenous [Human]) (CMV-IGIV) to prevent late Cytomegalovirus (“CMV”) infection, a common post-transplant infectious complication that remains an unaddressed medical need, despite recent advances in anti-viral drug therapies. The study, titled “Strategic Help with Immunoglobulin to Enhance protection against Late Disease (CMV)” or SHIELD, is a prospective, randomized, controlled multicenter investigator-initiated study in CMV high-risk kidney transplant recipients. CMV disease is an opportunistic infection that can occur in transplant recipients due to the use of anti-rejection medications that weaken the immune system. Anti-rejection medication is typically most intense in the first few months after transplant. During this period, transplant recipients are prescribed prophylactic antiviral medications. The SHIELD study will investigate the benefits of CYTOGAM administered at the conclusion of the anti-viral prophylaxis to reduce the risk of clinically significant late CMV in kidney transplant recipients who are CMV seronegative and have a CMV seropositive donor. These patients are at the highest risk of developing late-onset CMV infection, which is associated with worse transplant recipient health and outcomes. The study is being conducted by leading experts and key opinion leaders in CMV and organ transplantation, Camille Kotton, M.D., Infectious Disease Specialist and Clinical Director, Transplant and Immunocompromised Host Infectious Diseases at Massachusetts General Hospital, and David Wojciechowski D.O., Medical Director of the Kidney Transplantation Program at the University of Texas Southwestern Medical Center. Both investigators are recognized experts in transplant-related infections. Drs. Kotton and Wojciechowski said in a joint statement: “CMV disease is a leading cause for organ dysfunction and severe transplantation complications. There is a significant need in post-operative transplant care for rigorous scientific exploration of innovative prophylactic approaches for decreasing the risk of late CMV infection and improving transplant patient outcomes. We are grateful for Kamada’s support of this trial to explore the use of CYTOGAM dosed at the end of anti-viral prophylaxis therapy in patients with the highest risk of late CMV.” “Up to one third of kidney transplant patients may develop late CMV infection or disease after stopping initial anti-viral prophylaxis therapy,” said Amir London, Kamada’s Chief Executive Officer. “CYTOGAM is a critical life-saving therapeutic, and our support for this study further underscores Kamada’s commitment to serving the transplant community. We look forward to the results of this important post-marketing trial, led by two distinguished thought leaders in Drs. Kotton and Wojciechowski.” About CYTOGAMCytogam is an intravenous immunoglobulin containing standardized amount of antibody to cytomegalovirus. It is indicated for the prophylaxis of cytomegalovirus disease associated with transplantation of kidney, lung, liver, pancreas and heart. In transplants of these organs other than kidney from CMV seropositive donors into seronegative recipients, prophylactic CMV-IGIV should be considered in combination with ganciclovir. Important Safety Information for CYTOGAM:Cytogam is contraindicated in individuals with a history of a prior severe reaction associated with the administration of this or other human immunoglobulin preparations. Persons with selective immunoglobulin A deﬁciency have the potential for developing antibodies to immunoglobulin A and could have anaphylactic reactions to subsequent administration of blood products that contain immunoglobulin A, including Cytogam. Minor reactions, such as ﬂushing, chills, muscle cramps, back pain, fever, nausea, vomiting, arthralgia, and wheezing, were the most frequent adverse reactions observed during the clinical trials for Cytogam.  Immune Globulin Intravenous (Human) products have been reported to be associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. Thrombotic events have been reported in association with IGIV. Cytogam is derived from human plasma. As with all plasma-derived products, the risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated. Please see full Prescribing Information for full safety prescribing details.To report SUSPECTED ADVERSE REACTIONS, contact Kamada at pharmacovigilance@kamada.com or 1-(866)-916-0077 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. About Kamada Kamada Ltd. (the “Company”) is a global biopharmaceutical company with a portfolio of marketed products indicated for rare and serious conditions and a leader in the specialty plasma-derived therapies field. The Company’s strategy is focused on driving profitable growth through four primary growth pillars: First, organic growth from its commercial activities, including continued investment in the commercialization and life cycle management of its proprietary products, which include six FDA-approved specialty plasma-derived products: KEDRAB®, CYTOGAM®, GLASSIA®, WINRHO SDF®, VARIZIG® and HEPAGAM B®, as well as KAMRAB®, KAMRHO (D)® and two types of equine-based anti-snake venom products, and the products in the distribution segment portfolio, mainly through the launch of several biosimilar products in Israel. Second: the Company aims to secure significant new business development, in-licensing, collaboration and/or merger and acquisition opportunities, which are anticipated to enhance the Company’s marketed products portfolio and leverage its financial strength and existing commercial infrastructure to drive long-term growth. Third: the Company is expanding its plasma collection operations to support revenue growth through the sale of normal source plasma to other plasma-derived manufacturers, and to support its increasing demand for hyper-immune plasma. The Company currently owns three operating plasma collection centers in the United States, in Beaumont Texas, Houston Texas, and San Antonio, Texas. Lastly, the Company is leveraging its manufacturing, research and development expertise to advance the development and commercialization of additional product candidates, targeting areas of significant unmet medical need, with the lead product candidate Inhaled AAT, for which the Company is continuing to progress the InnovAATe clinical trial, a randomized, double-blind, placebo-controlled, pivotal Phase 3 trial. FIMI Opportunity Funds, the leading private equity firm in Israel, is the Company’s controlling shareholder, beneficially owning approximately 38% of the outstanding ordinary shares. CONTACTS:Chaime OrlevChief Financial OfficerIR@kamada.com Brian RitchieLifeSci Advisors, LLC212-915-2578britchie@LifeSciAdvisors.com

Phase 3Immunotherapy

26 Jun 2025

·www.echemi.com

Merck’s New CMV Drug Approved in China Targets Nearly 40000 HSCT Patients

On June 23, 2025, Merck announced that its novel non-nucleoside cytomegalovirus (CMV) inhibitor, Letermovir, in both tablet and injectable forms, has been approved by the National Medical Products Administration (NMPA) for use in children aged 6 months and above, weighing ≥6kg. The newly approved indication covers the prevention of CMV infection and disease in allogeneic hematopoietic stem cell transplant (HSCT) recipients who are CMV-seropositive (R+), including adults and children. According to Merck, Letermovir is the first and only CMV prevention therapy in China available for both pediatric and adult HSCT patients. CMV, a prevalent herpes virus, poses a severe threat to transplant recipients, significantly increasing transplant-related mortality rates. In China, CMV infection is widespread, with 60%-80% of infants testing positive for antibodies. Children, who lack fully developed immune systems, face an increased risk of CMV reactivation after transplantation. Data shows that in 2022 and 2023, there were 39,918 HSCT recipients in China, of which 9,860 were children—accounting for 1 in every 4 transplant patients. The approval of Letermovir addresses a critical unmet need in CMV prevention for this vulnerable group. Previously approved CMV inhibitors in China include Roche’s ganciclovir and valganciclovir, while domestic companies such as Zhejiang Asia-Pacific Pharmaceutical and Shanghai Pharma have also entered the market with generic versions. However, Letermovir offers a novel mechanism of action, making it a game-changing option for CMV prevention in HSCT patients, particularly children.

Drug ApprovalImmunotherapyCell Therapy

20 Jun 2025

·www.prnewswire.com

Stem Cell-Derived Islet Therapies Shown to Reduce the Need for Injectable Insulin

Breakthrough Studies Demonstrate Stem Cell-Based Technologies as a Potential Alternative Treatment for Type 1 Diabetes CHICAGO, June 20, 2025 /PRNewswire/ -- Findings from two studies reveal advancements in stem cell-derived treatment options to treat type 1 diabetes. Results from the FORWARD study, also simultaneously published in the New England Journal of Medicine (NEJM), evaluating the first and only allogeneic, stem-cell derived, islet cell therapy and a study of genetically modified stem cells were unveiled as an oral presentation and late-breaking poster, respectively, at the 85th Scientific Sessions of the American Diabetes Association® (ADA) in Chicago. Since the 1920s, the standard of care for type 1 diabetes was insulin replacement therapy—requiring millions of Americans to administer insulin via a pump or injection multiple times a day. There is a growing body of research for alternative treatment solutions including allogenic therapies, where stem cells are used to regenerate damaged or impaired tissues as well as insulin production. "Stem cell therapy is showing tremendous promise in transforming type 1 diabetes care, offering real hope for insulin independence," said Marlon Pragnell, the ADA's vice president of research and science. "With advances in genetic engineering, these therapies may one day also evade immune attack—eliminating the need for immunosuppressive drugs." First-of-Its-Kind Allogenic, Stem Cell Treatment Reduces Injectable Insulin Use in All Participants Findings from the Phase 1/2 FORWARD clinical study—evaluating the safety and efficacy of the stem cell-derived islet product VX-880 for improving glycemic control and reducing the need for exogenous insulin in adults with type 1 diabetes—were presented as an oral presentation at this year's Scientific Sessions and simultaneously published in the NEJM. The Phase 1/2, open-label three-part study enrolled 12 adult participants with type 1 diabetes and impaired awareness of hypoglycemia complications to assess the implementation of VX-880, the first and only allogeneic, stem-cell derived, fully differentiated insulin-producing islet cell therapy on glycemic control in pivotal development. Participants received a full dose of VX-880 islets, transplanted into the liver via a portal vein infusion under a standard immunosuppression drug regime. All 12 participants demonstrated restoration of endogenous insulin secretion (measured as C-peptide), elimination of severe hypoglycemia events, and achievement of recommended glycemic control targets (A1C <7% and time in range >70%). The treatment reduced exogenous insulin use in all patients (mean reduction of 92%) and eliminated exogenous insulin use in 10 patients. Adverse events related to the use of VX-880 were consistent with typical islet infusion procedures and existing immunosuppressive drug regimens, and no additional adverse symptoms were observed in any of the participants. "Hypoglycemia remains a dangerous risk for individuals with type 1 diabetes who are dependent on exogenous insulin administration," shared Michael Rickels, MD, MS, Willard and Rhoda Ware professor in diabetes and metabolic diseases, Division of Endocrinology, Diabetes & Metabolism, University of Pennsylvania Perelman School of Medicine and presenting investigator. "These findings indicate the potential for a novel cellular therapy that restores endogenous insulin secretion to improve outcomes for type 1 diabetes patients who have been struggling to achieve glycemic control." The FORWARD study is now in phase 3 which aims to complete enrollment and dosing of approximately 50 participants throughout 2025. Additionally, they plan to launch a VX-880 islet-after-kidney study involving around 10 adults with type 1 diabetes who are already receiving immunosuppressive therapy following a prior kidney transplant. Stem Cell-Based Treatment with Safety Switch Shows Potential for Patients with Type 1 Diabetes Findings demonstrating the early-stage success of genetically modified stem cell-derived islet (SC-islet) replacement therapies for the treatment of type 1 diabetes, leveraging a novel safety feature, were presented as a late-breaking poster. Researchers used genetically engineered human embryonic stem cell (hESC) lines to develop specialized cells designed to evade immune system attacks with the integration of eight added protective genes to the SC-islets. The treatment included an inducible kill switch as a safety feature designed to eliminate unwanted or fast-growing cells when activated, using a common, Food and Drug Administration (FDA)-approved antiviral drug (Ganciclovir). Results showed the specialized cells effectively produced insulin and showed strong and consistent immune evasion while in the cell culture phase of testing. The genetically engineered hESC lines efficiently differentiated into insulin-secreting SC-islets in vitro. When co-cultured with various immune cell types, the SC-islets suppressed immune cell activation and were resistant to immune cell-mediated killing, meaning they successfully evaded immune system attacks. The added safety switch effectively destroyed unwanted cell growth. "Lab-grown insulin-producing cells show great potential for treatment of type 1 diabetes, but immune system attacks to implanted cells pose an ongoing challenge," said Jia Zhao, PhD, postdoctoral researcher, University of British Columbia and presenting investigator of the study. "These early results show potential for safer, longer term cell therapy for diabetes as we look to provide patients with solutions without the need for harmful immune-suppressing drugs." Researchers are now testing these cells in animals to see if they can avoid immune attacks and if the safety switch works as expected in the body. Research presentation details: Dr. Rickels will present the findings as an oral presentation during the regular abstract session: Innovation and Progress in Stem Cell-Derived Islet-Cell Replacement Therapy Durable Glycemic Control and Elimination of Exogenous Insulin Use with VX-880 in Patients with Type 1 Diabetes (T1D)—VX-880-101 (FORWARD) Presented on Friday, June 20 at 6:15 p.m. CT Dr. Zhao will present the findings at the late-breaking poster session presentation sessions: Immune-Shielded Islets from Engineered Human Pluripotent Stem Cells for Potential Allogeneic Therapy Presented on Sunday, June 22 at 12:30 p.m. CT About the ADA's Scientific Sessions The ADA's 85th Scientific Sessions, the world's largest scientific meeting focused on diabetes research, prevention, and care, will be held in Chicago, IL, on June 20–23. Thousands of leading physicians, scientists, and health care professionals from around the world are expected to convene both in person and virtually to unveil cutting-edge research, treatment recommendations, and advances toward a cure for diabetes. Attendees will receive exclusive access to thousands of original research presentations and take part in provocative and engaging exchanges with leading diabetes experts. Join the Scientific Sessions conversation on social media using #ADASciSessions. About the American Diabetes Association The American Diabetes Association (ADA) is the nation's leading voluntary health organization fighting to end diabetes and helping people thrive. This year, the ADA celebrates 85 years of driving discovery and research to prevent, manage, treat, and ultimately cure—and we're not stopping. There are 136 million Americans living with diabetes or prediabetes. Through advocacy, program development, and education, we're fighting for them all. To learn more or to get involved, visit us at diabetes.org or call 1-800-DIABETES (800-342-2383). Join us in the fight on Facebook (American Diabetes Association), Spanish Facebook (Asociación Americana de la Diabetes), LinkedIn (American Diabetes Association), and Instagram (@AmDiabetesAssn). To learn more about how we are advocating for everyone affected by diabetes, visit us on X (@AmDiabetesAssn). Media Contact: Mimi Carmody, [email protected] SOURCE American Diabetes Association WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyPhase 3Clinical Result

100 Deals associated with Ganciclovir

External Link

KEGG	Wiki	ATC	Drug Bank
D00333	Ganciclovir	J05AB06 S01AD09	DB01004

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Keratitis, Herpetic	United States	Bausch & Lomb, Inc.	15 Sep 2009
Cytomegalovirus Infections	United States	CHEPLAPHARM Arzneimittel GmbH	23 Jun 1989
Cytomegalovirus Retinitis	United States	CHEPLAPHARM Arzneimittel GmbH	23 Jun 1989

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hematopoietic stem cell transplantation	Phase 3	Austria	Roche Pharma AG	01 Sep 2010
Hematopoietic stem cell transplantation	Phase 3	Germany	Roche Pharma AG	01 Sep 2010
Hematopoietic stem cell transplantation	Phase 3	Spain	Roche Pharma AG	01 Sep 2010
Acquired Immunodeficiency Syndrome	Phase 3	United States	-	31 Aug 2001
HIV Infections	Phase 3	United States	Hoffmann-La Roche, Inc.	01 Jan 1997
HIV Infections	Phase 3	Canada	Hoffmann-La Roche, Inc.	01 Jan 1997
Keratoconjunctivitis, Infectious	Phase 2	France	Laboratoires Théa SAS	01 Mar 2009
Immunosuppression	Phase 2	United States	Hoffmann-La Roche, Inc.	31 Aug 2001
Stomach Diseases	Phase 2	United States	Hoffmann-La Roche, Inc.	31 Aug 2001
Colitis	Phase 1	United States	Hoffmann-La Roche, Inc.	31 Aug 2001

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03586284 (STACCATO, CTgov)	Phase 2/3	Uveitis, Anterior	51	Topical placebo+Valganciclovir Hydrochloride (Oral Valganciclovir)	eeilgzdvph(dtejqmqhpy) = ibhxoixxas wcmnpudfal (kjciwlljog, 0.87) View more	-	10 Feb 2026
				Placebo Oral Tablet+Ganciclovir Sodium (Topical Ganciclovir 2%)	eeilgzdvph(dtejqmqhpy) = lvtmsqedwb wcmnpudfal (kjciwlljog, 0.59) View more
NCT04706507 (GRAIL^3, CTgov)	Phase 3	Respiratory Failure \| Sepsis	205	IV Ganciclovir (IV Ganciclovir)	pwlkpwktrp(bwxrzbbdgh) = dxyzndbhdn ncqvkekzjq (lrxqnvtllc, 11.1) View more	-	12 Dec 2025
				Normal saline (Placebo)	pwlkpwktrp(bwxrzbbdgh) = uvbnmcyoww ncqvkekzjq (lrxqnvtllc, 10.5) View more
EURETINA2025	Not Applicable	Cytomegalovirus Retinitis	1	Intravitreal Foscarnet + Systemic Ganciclovir/Valganciclovir + Anti-VEGF (Aflibercept) + Topical Corticosteroids	wsjknfvodj(nuatlxsszg) = Transient ocular hypertension (IOP: 25 mmHg) post- injection, managed with topical timolol. Cystoid macular edema in OS (GMC: 242 μm) resolved after 3 monthly aflibercept injections. unvdfhkoir (fyazjxcgmt ) View more	Positive	04 Sep 2025
Tandem Meetings ASTCT and CIBMTR2023	Not Applicable	Cytomegalovirus Infections	-	Ganciclovir/Valganciclovir	zuvbpagcgn(dpmuasckqz) = cgiixvnumu unclbhucsr (ankkzttwhg )	Positive	01 Feb 2023
				Letermovir	zuvbpagcgn(dpmuasckqz) = rjbsbpilud unclbhucsr (ankkzttwhg )
Tandem Meetings ASTCT and CIBMTR2022	Not Applicable	Hematopoietic stem cell transplantation	-	Ganciclovir prophylaxis through day +100	aasqahtiyy(blqdoscmci) = thfcblltwz vpqngnfeba (rwuyeaxeng, 0 - 71.3) View more	Negative	01 Mar 2022
				Alternative CMV prophylaxis prior to day +100	aasqahtiyy(blqdoscmci) = ithcvlffeh vpqngnfeba (rwuyeaxeng, 19.4 - 66.9) View more
NCT02382588 (CTgov)	Phase 2	Keratitis, Herpetic	8	gancyclovir gel (Gancyclovir Gel)	aecqyunuli(ircupyasff) = bcjadaktzi dnlypgyzlq (ejrjdyusbi, seyiezbmkz - mffvvavhlf) View more	-	08 Jul 2021
				Hypromellose gel (Hypromellose Gel)	aecqyunuli(ircupyasff) = mytyhthznf dnlypgyzlq (ejrjdyusbi, vnslzcjgds - ibglzexxsr) View more
Tandem Meetings ASTCT and CIBMTR2021	Not Applicable	Hematopoietic stem cell transplantation	-	Ganciclovir prophylaxis through day +100	cwrsdbfnng(xhyrgbvlbt) = ygkpyoxfaa ogvdovpmkb (cegluqllao, 13.0 - 59.4) View more	-	01 Mar 2021
				Ganciclovir (No CMV viremia/disease)	cwrsdbfnng(xhyrgbvlbt) = qltxgkfbmf ogvdovpmkb (cegluqllao, 62.3 - 82.9) View more
Tandem Meetings ASTCT and CIBMTR2019	Not Applicable	-	-	Ganciclovir pretransplant	lpxxrofemm(vamammfhxv) = noppjaswmz iowgnurhdy (twbfjdksrs, 18% - 37%)	-	01 Mar 2019
EBMT2018	Not Applicable	-	30	Ganciclovir and Foscarnet combination therapy	xhdplgnpek(dtkrgmjbfo) = there seems to be a relation between the duration of combination therapy and nephrotoxicity, which should be monitored carefully qaohuxzoeg (uxupmrefka )	-	19 Nov 2018
NCT01647529 (R733/17/2010, Pubmed \| PLoS One)	Not Applicable	Cytomegalovirus Infections	29	Ganciclovir gel, 0.15%	ebhljmtqyx(bvaqftrnme) = bnlggydwqm mnpszhgehy (mryuaazgqn )	-	01 Jan 2018

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