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Neoplasms

Nervous System Diseases

Infectious Diseases

AAV based gene therapy

Oncolytic virus

CRISPR/Cas

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Neoplasms	4
Nervous System Diseases	2
Infectious Diseases	2
Endocrinology and Metabolic Disease	1
Immune System Diseases	1

Top 5 Drug Type	Count

AAV based gene therapy	5
Oncolytic virus	4
CRISPR/Cas	1
Recombinant polypeptide	1

Top 5 Target	Count

Envelope (E) protein	1
titin	1
HRG(histidine rich glycoprotein)	1
PKR(eukaryotic translation initiation factor 2 alpha kinase 2)	1
IGHMBP2(immunoglobulin mu DNA binding protein 2)	1

OBJECTIVEAlthough multidisciplinary clinics improve outcomes in chronic limb-threatening ischemia (CLTI), their role in addressing socioeconomic disparities is unknown. Our institution treats patients with CLTI at both traditional general vascular clinics and a multidisciplinary Limb Preservation Program (LPP). The LPP is in a minority community, providing expedited care at a single facility by a consistent team. We compared outcomes within the LPP with our institution's traditional clinics and explored patients' perspectives on barriers to care to evaluate if the LPP might address them.METHODSAll patients undergoing index revascularization for CLTI from 2014 to 2023 at our institution were stratified by clinic type (LPP or traditional). We collected clinical and socioeconomic variables, including Area Deprivation Index (ADI). Patient characteristics were compared using χ², Student t, or Mood median tests. Outcomes were compared using log-rank and multivariable Cox analysis. We also conducted semi-structured interviews to understand patient-perceived barriers.RESULTSFrom 2014 to 2023, 983 limbs from 871 patients were revascularized; 19.5% of limbs were treated within the LPP. Compared with traditional clinic patients, more LPP patients were non-White (43.75% vs 27.43%; P < .0001), diabetic (82.29% vs 61.19%; P < .0001), dialysis-dependent (29.17% vs 13.40%; P < .0001), had ADI in the most deprived decile (29.38% vs 19.54%; P = .0061), resided closer to clinic (median 6.73 vs 28.84 miles; P = .0120), and had worse Wound, Ischemia, and foot Infection (WIfI) stage (P < .001). There were no differences in freedom from death, major adverse limb event (MALE), or patency loss. Within the most deprived subgroup (ADI >90), traditional clinic patients had earlier patency loss (P = .0108) compared with LPP patients. Multivariable analysis of the entire cohort demonstrated that increasing age, heart failure, dialysis, chronic obstructive pulmonary disease, and increasing WIfI stage were independently associated with earlier death, and male sex was associated with earlier MALE. Ten traditional clinic patients were interviewed via convenience sampling. Emerging themes included difficulty understanding their disease, high visit frequency, transportation barriers, distrust of the health care system, and patient-physician racial discordance.CONCLUSIONSLPP patients had worse comorbidities and socioeconomic deprivation yet had similar outcomes to healthier, less deprived non-LPP patients. The multidisciplinary clinic's structure addresses several patient-perceived barriers. Its proximity to disadvantaged patients and ability to conduct multiple appointments at a single visit may address transportation and visit frequency barriers, and the consistent team may facilitate patient education and improve trust. Including these elements in a multidisciplinary clinic and locating it in an area of need may mitigate some negative impacts of socioeconomic deprivation on CLTI outcomes.

23 Sep 2024·Breastfeeding medicine : the official journal of the Academy of Breastfeeding Medicine

Factors Associated with Mother's Own Milk Feeding and Direct Breastfeeding at Discharge in Preterm Infants with Feeding Difficulties: Clinical and Research Implications.

Article

Author: Jadcherla, Sudarshan R ; Alshaikh, Enas ; Bala, Faith

Background: Prematurity presents numerous barriers to mother's own milk (MOM) feeding and direct breastfeeding (DBF). Aim: This study aimed to determine factors associated with MOM feeding and DBF at discharge from the neonatal intensive care unit (NICU) in preterm-born infants presenting with feeding difficulties. Methods: A retrospective study of data from 237 preterm-born infants referred for evaluation of feeding difficulties and discharged home on full oral feeds was examined. Maternal and infant characteristics and oral feeding milestones were examined for their association with MOM intake and DBF at discharge using bivariate and multivariate regression analyses. Results: MOM feeding at discharge occurred in 35.4% (n = 84) infants. The odds of any MOM feeding at discharge were higher with higher maternal age, absence of maternal substance use, and fewer days between full per oral (PO) and discharge (all, p < 0.05). Among the 84 MOM-fed infants, 4.76% (n = 4) were exclusively breastfed, whereas 39.3% (n = 33) were partially DBF at discharge. The DBF infants had higher birthweight, no incidence of being small for gestational age, lower incidence of respiratory support at birth and intraventricular hemorrhage, lower postmenstrual age (PMA) at full PO, shorter duration from first PO to full PO, and lower PMA at discharge (all, p < 0.05). Conclusion: We found reduced use of MOM and DBF among preterm-born infants with feeding difficulties at NICU discharge. Clinical management and research advocacy must focus on targeted interventions in this setting by recognizing significant modifiable factors applicable to prepregnancy, pregnancy, NICU, and postdischarge care.

01 Sep 2024·Molecular Therapy - Methods & Clinical Development

The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome

Article

Author: Likhite, Shibi ; Jeon, Shin ; Meyer, Kathrin C ; Lee, Soo-Kyung ; Park, Jaein ; Li, Liwen ; Moon, Ji Hwan ; Lee, Jae W ; Shin, Dongjun

Heterozygous mutations in the FOXG1 gene manifest as FOXG1 syndrome, a severe neurodevelopmental disorder characterized by structural brain anomalies, including agenesis of the corpus callosum, hippocampal reduction, and myelination delays. Despite the well-defined genetic basis of FOXG1 syndrome, therapeutic interventions targeting the underlying cause of the disorder are nonexistent. In this study, we explore the therapeutic potential of adeno-associated virus 9 (AAV9)-mediated delivery of the FOXG1 gene. Remarkably, intracerebroventricular injection of AAV9-FOXG1 to Foxg1 heterozygous mouse model at the postnatal stage rescues a wide range of brain pathologies. This includes the amelioration of corpus callosum deficiencies, the restoration of dentate gyrus morphology in the hippocampus, the normalization of oligodendrocyte lineage cell numbers, and the rectification of myelination anomalies. Our findings highlight the efficacy of AAV9-based gene therapy as a viable treatment strategy for FOXG1 syndrome and potentially other neurodevelopmental disorders with similar brain malformations, asserting its therapeutic relevance in postnatal stages.

100 Deals associated with Research Institute at Nationwide Childrens Hospital

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100 Translational Medicine associated with Research Institute at Nationwide Childrens Hospital

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Pipeline

Pipeline Snapshot as of 26 Nov 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Discovery

10

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Preclinical

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

rAAV.TCAP ( telethonin )	Muscular Dystrophy, Limb-Girdle, Type 2G More	Preclinical
US11617783 ( titin )	Heart Diseases More	Discovery
WO2023235377 ( IL-27 )	Neoplasms More	Discovery
RU2821999 ( Envelope (E) protein )	Neoplasms More	Discovery
WO2022164860 ( LIPA )	Endocrinology and Metabolic Disease More	Discovery