AI Drug Discovery for Pharma and Biotech

Drug discovery

1

drug

With orphan designation

Overview

Congenital herpes simplex virus (HSV) infection is a rare but severe neonatal condition caused by vertical transmission of HSV-1 or HSV-2 during pregnancy, delivery, or postnatally. Clinical manifestations range from localized skin, eye, or mouth (SEM) involvement to disseminated disease or central nervous system (CNS) infection, with risks of mortality and long-term neurodevelopmental impairment. Early diagnosis and prompt intravenous acyclovir therapy significantly improve outcomes.

Population

  • Incidence: ~10 cases per 100,000 births globally (~14,000 annual cases), with regional variability (higher in Africa due to HSV-2 prevalence; HSV-1 accounts for >60% of cases in the Americas and Europe) [6][10][16].

  • Highest risk: Infants born to mothers with primary HSV infection during the third trimester (33–50% transmission risk without suppressive therapy) [1][9].

Burden

  • Mortality: Up to 60% untreated; reduced to 4% (CNS) and 30% (disseminated) with early antiviral therapy [3][7].

  • Morbidity: Neurologic sequelae (e.g., seizures, developmental delay) in 15–30% of survivors; SEM disease rarely progresses if treated promptly [5][7][16].

  • Global Impact: Disproportionately affects low-resource regions due to delayed diagnosis and limited access to antivirals, contributing to long-term disability and healthcare costs [6][17].

  • Key interventions include maternal screening, clinician awareness of nonspecific neonatal symptoms (e.g., sepsis-like presentation), and rapid PCR testing of CSF/skin lesions [5][9].

Therapies

  • High-dose IV acyclovir (60 mg/kg/day for 14–21 days) based on disease severity (SEM: 14 days; CNS/disseminated: 21 days) [3][7][16].

  • Oral acyclovir suppressive therapy (6 months post-IV) to reduce recurrence and improve neurodevelopmental outcomes in CNS cases [7][14].

  • Cesarean delivery for active maternal genital lesions and maternal antiviral prophylaxis (e.g., valacyclovir) during late pregnancy to reduce viral shedding [7][9].

Categories: rare developmental anomalies during embryogenesis, rare infectious diseases, rare teratologic disorders

Research Papers

105 drug discovery papers about Congenital herpes simplex virus infection, with 1 first-in-class and 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

105 drug discovery papers about Congenital herpes simplex virus infection, with 1 first-in-class and 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-25 | HSV, HIV, and Pregnancy: Epidemiology, Viral Interactions, Management, and Prevention.

We summarize the epidemiology, biology, management and prevention of HSV-2 and HIV in pregnancy. The seroprevalence of HSV-2 is higher in people living with HIV than in HIV-negative people, and 75% of women with recurrent genital herpes may have a recurrence during pregnancy. Those with a history of recurrent genital HSV infection should be offered acyclovir/valacyclovir at 36 weeks gestation to decrease need for C-section. The most important intervention for reducing HIV vertical transmission is maternal antiretroviral therapy (ART) to suppress HIV replication. Maternal ART also reduces HSV-2 cervicovaginal shedding and thus, HSV-2 vertical transmission. HSV-2 viral shedding is incompletely suppressed with acyclovir/valacyclovir, and newer drugs such as the helicase-primase inhibitors show promise but have not been studied in pregnancy. Safe options also exist for preventing HIV acquisition in pregnancy but remain in development for HSV-2. Safe, effective HSV-2/HIV management strategies exist in pregnancy.

Open article ↗



2026-07-24 | Comparative Genomics of Herpes Simplex Virus 2 Isolated from a Maternal-Neonatal Dyad Reveals High Consensus Sequence Homology as Well as Minor Variant Diversity.

Neonatal herpes simplex virus disease most often occurs following mother-to-neonate transmission during childbirth. However, genomic differences between the maternal and neonatal viral populations are poorly understood. Here, we performed deep whole-genome sequencing of clinical HSV-2 samples obtained from a maternal-neonatal dyad to investigate viral population dynamics during transmission.

Open article ↗



2026-04-28 | Cyclic GMP-AMP Synthase (cGAS) Activators for Congenital Herpes Prevention

UniProt identifies cGAS (Q8N884) as key innate immune receptor sensing viral nucleic acids and producing type I interferons. The protein catalyzes cyclic GMP-AMP formation from ATP and GTP, triggering antiviral responses. Enhancing cGAS activity could prevent vertical transmission of herpes simplex virus from mother to newborn, addressing the devastating congenital herpes infections described in Orphanet (ORDO:293).

Open article ↗



2026-04-14 | TORCH: Current state of knowledge as of 2025.

The acronym TORCH designates a group of pathogens that can lead to serious pregnancy complications, such as miscarriage, fetal growth restriction, and congenital infections. This review summarizes current insights into these infections, offering practical guidance primarily for obstetricians, infectious disease specialists, and general practitioners involved in prenatal care. The TORCH complex includes Toxoplasma gondii, Rubella virus, Cytomegalovirus (CMV), and Herpes simplex virus (HSV). The definition may be extended to encompass additional (other) pathogens such as Hepatitis B and C viruses (HBV, HCV), Human immunodeficiency virus (HIV), Varicella-zoster virus (VZV), Treponema pallidum (syphilis), Parvovirus B19, and Zika virus. Screening practices for TORCH infections during pregnancy vary significantly across countries. Despite widespread access to medical care and increasing awareness among women planning pregnancy, routine screening for TORCH pathogens is not universally implemented. In Poland, diagnostic procedures during pregnancy are defined by the Standard of Perinatal Care established by the Ministry of Health. This regulation- which replaced earlier recommendations of the Polish Society of Gynecologists and Obstetricians- does not distinguish between ,,mandatory" and ,,recommended" tests but specifies a unified set of investigations to be performed at defined stages of pregnancy. Screening conducted during pregnancy plays a crucial role in detecting previously unrecognized infections. In Poland , a substantial proportion of new diagnoses of HIV, HBV and HCV among young women are made during routine antenatal testing, underscoring the importance of standardized serological screening in prenatal care rather than relying on diagnosis before conception. However, due to their distinct epidemiological and clinical profiles, HIV, HBV, and HCV infections are not discussed in detail in this review.

Open article ↗



2026-04-01 | Chorioretinitis Scarring in a Neonate Secondary to Congenital Herpes Simplex Type 2.

Purpose: To describe a rare case of congenital herpes simplex virus type 2 (HSV-2) infection with bilateral retinal involvement in a neonate. Methods: A single case is presented along with a brief literature review. Results: A neonate born to a mother with unrecognized HSV-2 infection presented with skin lesions initially thought to be secondary to amniotic band syndrome. Subsequent evaluation revealed positive skin cultures for HSV-2, suggesting in utero transmission, most likely during the early second trimester. Fundus examination demonstrated large, bilateral hyperpigmented chorioretinal scars consistent with retinal involvement. Conclusions: Congenital HSV infection, although rare, should be considered in neonates presenting with skin lesions and chorioretinitis. Early recognition and prompt initiation of intravenous acyclovir therapy, even before culture confirmation when clinical suspicion is high, are critical to optimize outcomes.

Open article ↗



2026-07-25 | HSV, HIV, and Pregnancy: Epidemiology, Viral Interactions, Management, and Prevention.

We summarize the epidemiology, biology, management and prevention of HSV-2 and HIV in pregnancy. The seroprevalence of HSV-2 is higher in people living with HIV than in HIV-negative people, and 75% of women with recurrent genital herpes may have a recurrence during pregnancy. Those with a history of recurrent genital HSV infection should be offered acyclovir/valacyclovir at 36 weeks gestation to decrease need for C-section. The most important intervention for reducing HIV vertical transmission is maternal antiretroviral therapy (ART) to suppress HIV replication. Maternal ART also reduces HSV-2 cervicovaginal shedding and thus, HSV-2 vertical transmission. HSV-2 viral shedding is incompletely suppressed with acyclovir/valacyclovir, and newer drugs such as the helicase-primase inhibitors show promise but have not been studied in pregnancy. Safe options also exist for preventing HIV acquisition in pregnancy but remain in development for HSV-2. Safe, effective HSV-2/HIV management strategies exist in pregnancy.

Open article ↗



2026-07-24 | Comparative Genomics of Herpes Simplex Virus 2 Isolated from a Maternal-Neonatal Dyad Reveals High Consensus Sequence Homology as Well as Minor Variant Diversity.

Neonatal herpes simplex virus disease most often occurs following mother-to-neonate transmission during childbirth. However, genomic differences between the maternal and neonatal viral populations are poorly understood. Here, we performed deep whole-genome sequencing of clinical HSV-2 samples obtained from a maternal-neonatal dyad to investigate viral population dynamics during transmission.

Open article ↗



2026-04-28 | Cyclic GMP-AMP Synthase (cGAS) Activators for Congenital Herpes Prevention

UniProt identifies cGAS (Q8N884) as key innate immune receptor sensing viral nucleic acids and producing type I interferons. The protein catalyzes cyclic GMP-AMP formation from ATP and GTP, triggering antiviral responses. Enhancing cGAS activity could prevent vertical transmission of herpes simplex virus from mother to newborn, addressing the devastating congenital herpes infections described in Orphanet (ORDO:293).

Open article ↗



2026-04-14 | TORCH: Current state of knowledge as of 2025.

The acronym TORCH designates a group of pathogens that can lead to serious pregnancy complications, such as miscarriage, fetal growth restriction, and congenital infections. This review summarizes current insights into these infections, offering practical guidance primarily for obstetricians, infectious disease specialists, and general practitioners involved in prenatal care. The TORCH complex includes Toxoplasma gondii, Rubella virus, Cytomegalovirus (CMV), and Herpes simplex virus (HSV). The definition may be extended to encompass additional (other) pathogens such as Hepatitis B and C viruses (HBV, HCV), Human immunodeficiency virus (HIV), Varicella-zoster virus (VZV), Treponema pallidum (syphilis), Parvovirus B19, and Zika virus. Screening practices for TORCH infections during pregnancy vary significantly across countries. Despite widespread access to medical care and increasing awareness among women planning pregnancy, routine screening for TORCH pathogens is not universally implemented. In Poland, diagnostic procedures during pregnancy are defined by the Standard of Perinatal Care established by the Ministry of Health. This regulation- which replaced earlier recommendations of the Polish Society of Gynecologists and Obstetricians- does not distinguish between ,,mandatory" and ,,recommended" tests but specifies a unified set of investigations to be performed at defined stages of pregnancy. Screening conducted during pregnancy plays a crucial role in detecting previously unrecognized infections. In Poland , a substantial proportion of new diagnoses of HIV, HBV and HCV among young women are made during routine antenatal testing, underscoring the importance of standardized serological screening in prenatal care rather than relying on diagnosis before conception. However, due to their distinct epidemiological and clinical profiles, HIV, HBV, and HCV infections are not discussed in detail in this review.

Open article ↗



2026-04-01 | Chorioretinitis Scarring in a Neonate Secondary to Congenital Herpes Simplex Type 2.

Purpose: To describe a rare case of congenital herpes simplex virus type 2 (HSV-2) infection with bilateral retinal involvement in a neonate. Methods: A single case is presented along with a brief literature review. Results: A neonate born to a mother with unrecognized HSV-2 infection presented with skin lesions initially thought to be secondary to amniotic band syndrome. Subsequent evaluation revealed positive skin cultures for HSV-2, suggesting in utero transmission, most likely during the early second trimester. Fundus examination demonstrated large, bilateral hyperpigmented chorioretinal scars consistent with retinal involvement. Conclusions: Congenital HSV infection, although rare, should be considered in neonates presenting with skin lesions and chorioretinitis. Early recognition and prompt initiation of intravenous acyclovir therapy, even before culture confirmation when clinical suspicion is high, are critical to optimize outcomes.

Open article ↗



Access all drug discovery papers and probability of success in trials forecasts:

Access all drug discovery papers and probability of success in trials forecasts:

Drug Discovery Landscape

1 orphan drug designation for Congenital herpes simplex virus infection.

1 orphan drug designation for Congenital herpes simplex virus infection.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

N-Methanocarbathymidine

small molecules

FDA

2014-10-15

N & N Pharmaceuticals, Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.