Methodology

All entries for: Rare Disease

January 15, 2026

Advaccine Biopharmaceuticals Suzhou Co., Ltd.

Orphan Drug Designation

treatment of recurrent respiratory papillomatosis

Disease Area: Rare Disease

Source

January 15, 2026

XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd

Orphan Drug Designation

treatment of malignant glioma

Disease Area: Rare Disease

Source

January 13, 2026

Shanghai Vitalgen BioPharma Co., Ltd.

Orphan Drug Designation

treatment of Gaucher disease

Disease Area: Rare Disease

January 13, 2026

BPGbio Inc.

Orphan Drug Designation

treatment of primary CoQ10 deficiency

Disease Area: Rare Disease

Source

January 13, 2026

Galderma Research and Development, LLC

Orphan Drug Designation

treatment of systemic sclerosis

Disease Area: Rare Disease

Source

January 13, 2026

Biotle Biotechnology (Hangzhou) Co., Ltd.

Orphan Drug Designation

treatment of multiple myeloma

Disease Area: Rare Disease

Source

January 13, 2026

Heronova Pharmaceuticals, LLC

Orphan Drug Designation

treatment of Stargardt disease

Disease Area: Rare Disease

Source

January 13, 2026

HemaNiche Biotech Co., Ltd

Orphan Drug Designation

treatment of post-hematopoietic stem cell transplantation thrombocytopenia

Disease Area: Rare Disease

Source

January 13, 2026

University of California, San Francisco

Orphan Drug Designation

treatment of acute myeloid leukemia

Disease Area: Rare Disease

Source

January 12, 2026

Juvena Therapeutics

Investment

Juvena Therapeutics raised $79.5 million to advance protein-based therapeutics that restore cellular function and promote tissue repair for chronic and rare diseases.

Disease Area: Rare Disease

Source