All entries for: Rare Diseases

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
January 8, 2026

Tessera Therapeutics

Layoffs

Somerville, MA
201-500 employees

A month after announcing a partnership with Regeneron to advance its lead gene editor, Tessera Therapeutics has disclosed it will lay off 90 employees. Endpoints News first reported the cuts. Several employees told Endpoints the layoffs will affect about 35% of the workforce. The cuts follow a 17% reduction in 2025 and 13% downsizing in 2024.

Disease Area: Chronic Disease, Multiple, Rare Diseases, Respiratory Diseases
Drug Type: Biologic
January 5, 2026

Delnaggar Biotech Group

Orphan Drug Designation

FDA orphan drug designation: treatment of high-grade anal intraepithelial neoplasia (HGAIN)

Disease Area: Rare Diseases
January 5, 2026

Nanjing RegeneCore Biotech Co.

Orphan Drug Designation

FDA orphan drug designation: treatment of acute myeloid leukemia

Disease Area: Rare Diseases
January 3, 2026

GSK

Layoffs

San Francisco, CA
50,001+ employees

Up to 350 people across GSK’s U.S. and U.K. workforces could lose their jobs as the pharma reorients its investments with its priorities. The layoffs are expected to affect fewer than 70 individuals in the US, and fewer than 50 in the UK, according to Fierce.

Disease Area: Immune Diseases, Multiple, Oncology, Rare Diseases, Respiratory Diseases
Drug Type: Biologic, Small Molecule
January 1, 2026

mAbTree Biologics AG

Orphan Drug Designation

FDA orphan drug designation: treatment of essential thrombocythemia

Disease Area: Rare Diseases
December 31, 2025

Pharmascience Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of pericarditis

Disease Area: Rare Diseases
December 31, 2025

Haichang Biotech Co.

Orphan Drug Designation

FDA orphan drug designation: treatment of osteosarcoma

Disease Area: Rare Diseases
December 23, 2025

Veloxis Pharmaceuticals

Orphan Drug Designation

FDA orphan drug designation: prevention of organ rejection in patients receiving a liver transplant

Disease Area: Rare Diseases
December 19, 2025

HemaCell Biotechnology Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of Bernard-Soulier syndrome

Disease Area: Rare Diseases
Scroll to Top