Methodology

All entries for: Rare Disease

February 23, 2026

William S. Brusilow

Orphan Drug Designation

Grosse Pointe
1-50 employees

treatment of amyotrophic lateral sclerosis

Disease Area: Rare Disease
Source
February 23, 2026

Sanegene Bio USA Inc.

Orphan Drug Designation

treatment of immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN )

Disease Area: Rare Disease
Source
February 23, 2026

Onconic Therapeutics

Orphan Drug Designation

Seoul
1-50 employees

treatment of small cell lung cancer

Disease Area: Rare Disease
Source
February 20, 2026

Doma Biopharmaceutical (Suzhou) Co., Ltd

Orphan Drug Designation

Suzhou Industrial Park
1-50 employees

treatment of small cell lung cancer

Disease Area: Rare Disease
Source
February 20, 2026

AstraZeneca Pharmaceuticals LP

Orphan Drug Designation

treatment of follicular lymphoma

Disease Area: Rare Disease
Source
February 19, 2026

Agios Pharmaceuticals, Inc.

Orphan Drug Designation

Cambridge
1-50 employees

treatment of sickle cell disease

Disease Area: Rare Disease
Source
February 19, 2026

Otsuka Pharmaceutical Development & Commercialization, Inc

Orphan Drug Designation

Rockville
1-50 employees

treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis

Disease Area: Rare Diseases
Source
February 19, 2026

PureTech LYT 100, Inc., a subsidiary of PureTech Health plc

Orphan Drug Designation

Boston
1-50 employees

treatment of idiopathic pulmonary fibrosis

Disease Area: Rare Disease
Source
February 17, 2026

NexEos Bio

Investment

NexEos Bio received a $3 million investment to advance NTX-1024, an ophthalmic solution for the treatment of the rare disease vernal keratoconjunctivitis (VKC)

Disease Area: Rare Disease
Source
Source
February 15, 2026

New York University, on behalf of NYU Grossman School of Medicine

Orphan Drug Designation

treatment of primary CoQ10 deficiency

Disease Area: Rare Disease
Source
Source
Scroll to Top