Delving into the Latest Updates on H4 Orphan Pharma SAS with Synapse

Overview

Tags

Immune System Diseases

Respiratory Diseases

Eye Diseases

Small molecule drug

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

Immune System Diseases	1

Top 5 Drug Type	Count

Small molecule drug	2

Top 5 Target	Count

CFTR(Cystic fibrosis transmembrane conductance regulator)	2

We generated human meibomian gland and conjunctival epithelial cells producing lipids and mucins. We identified ion channels including ENaC, CFTR, TMEM16a, and KCNQ1, as well as NKCC. We found that electrolyte and water flux are regulated by signaling pathways mediated by purinergic and VIP receptors. Our findings provide valuable insights into epithelial ion and water transport in the human conjunctiva and meibomian gland, enhancing understanding of these processes in both physiological and disease states.

100 Deals associated with H4 Orphan Pharma SAS

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100 Translational Medicine associated with H4 Orphan Pharma SAS

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Pipeline

Pipeline Snapshot as of 29 Jun 2025

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

Preclinical

2

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

Compounds 554-001(H4 Orphan Pharma) ( CFTR )	Idiopathic Pulmonary Fibrosis More	Preclinical
Compounds 554-002(H4 Orphan Pharma) ( CFTR )	Sjogren's Syndrome More	Preclinical