Tracking Rare Disease Drug Development Challenges Amid a Shifting Policy Environment

Rare diseases are defined as those which affect fewer than 200,000 people in the United States. Despite advancements in rare disease research and development due to incentives created in the Orphan Drug Act more than 40 years ago, many rare diseases still lack effective treatments due to the complex science and limited market incentives involved in their development. Of the 10,000+ known rare diseases, less than 10% have an FDA-approved treatment. The Inflation Reduction Act's healthcare provisions are an example of policies that discourage investment in rare disease treatments, leading to discontinued research, layoffs, and ultimately delayed treatments for patients. Incubate's Rare Disease Investment Tracker measures the impacts of a challenging policy environment on rare disease R&D.

Date
Date
Drug Type
Announcement Type
Company Size
March 18, 2025

Pyxis Oncology

Layoffs

Boston, MA
1-50 employees

20% reduction in workforce.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
March 13, 2025

Sutro Biopharma

Layoffs

South San Francisco, CA
201-500 employees

Reducing headcount by nearly 50 percent.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
March 11, 2025

Kiromic Biopharma

Layoffs

Houston, TX
1-50 employees

Furloughed substantially all of its workforce.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
March 6, 2025

Atea Pharmaceuticals

Layoffs

Boston, MA
51-200 employees

25% reduction in workforce.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
March 5, 2025

ALX Oncology

Layoffs

San Francisco, CA
51-200 employees

ALX Oncology, a Bay Area biotech firm currently running clinical trials for its anticancer therapy, announced Wednesday that it’s laying off about 30% of its staff.

Disease Area: Multiple, Oncology, Rare Diseases
Drug Type: Small Molecule
February 28, 2025

CRISPR Therapeutics

Layoffs

Switzerland
51-200 employees

Laying off an undisclosed number of employees.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
February 25, 2025

Lava Therapeutics

Layoffs

Netherlands
51-200 employees

Laying off 30% of its staff.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
February 13, 2025

Encoded Therapeutics

Layoffs

South San Francisco, CA
51-200 employees

29% reduction in workforce, mostly in technology and early-stage R&D functions.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
February 5, 2025

X4 Pharmaceuticals

Layoffs

Boston, MA
51-200 employees

43 employees laid off. All pre-clinical programs paused.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
February 4, 2025

Bristol Myers Squibb

Layoffs

Princeton, NJ
10,001-50,000 employees

67 employees laid off in New Jersey.

Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
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