All entries for: Rare Disease

January 21, 2026

Mendra

Investment

Mendra raised $82 million to apply artificial intelligence to the development of rare-disease drugs, accelerating discovery and commercialization.

Disease Area: Rare Disease
January 20, 2026

University of Granada

Orphan Drug Designation

treatment of primary CoQ10 deficiency

Disease Area: Rare Disease
January 16, 2026

Gleeson Lab - UCSD

Orphan Drug Designation

treatment of primary CoQ10 deficiency

Disease Area: Rare Disease
January 15, 2026

Atossa Therapeutics, Inc.

Orphan Drug Designation

treatment of Duchenne muscular dystrophy

Disease Area: Rare Disease
January 15, 2026

Revio Therapeutics LLP

Orphan Drug Designation

treatment of Duchenne muscular dystrophy

Disease Area: Rare Disease
January 15, 2026

Lantern Pharma Inc.

Orphan Drug Designation

treatment of soft tissue sarcoma

Disease Area: Rare Disease
January 15, 2026

Ascentage Pharma Group, Inc.

Orphan Drug Designation

treatment of myelodysplastic syndrome

Disease Area: Rare Disease
January 15, 2026

Nanjing Reju Therapeutics Co., Ltd.

Orphan Drug Designation

treatment of idiopathic pulmonary fibrosis

Disease Area: Rare Disease
January 15, 2026

NexEos Diagnostics Inc. (dba NexEos Bio)

Orphan Drug Designation

treatment of vernal keratoconjunctivitis

Disease Area: Rare Disease
January 15, 2026

Daiichi Sankyo, Inc.

Orphan Drug Designation

treatment of esophageal cancer

Disease Area: Rare Disease
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