treatment of neuroblastoma
All entries for: Rare Diseases
August 13, 2026
SEED Therapeutics Inc.
Orphan Drug Designation
King of Prussia
1-50 employees
Disease Area: Rare Diseases
August 13, 2026
Scholar Rock, Inc.
Orphan Drug Designation
Cambridge
1-50 employees
Treatment of facioscapulohumeral muscular dystrophy
Disease Area: Rare Diseases
August 10, 2026
Epicrispr Biotechnologies
Later Stage VC
Epicrispr Biotechnologies raised $90 million to advance its pipeline of first-in-class epigenetic therapies for the treatment of neuromuscular and ophthalmic diseases, including facioscapulohumeral muscular dystrophy and Duchenne muscular dystrophy, both rare disorders.
Investment Amount (In Millions): 90
Disease Area: Rare Diseases
August 10, 2026
HepaRegeniX GmbH
Orphan Drug Designation
Tubingen
1-50 employees
treatment of frontotemporal dementia
Disease Area: Rare Diseases
August 10, 2026
HepaRegeniX GmbH
Orphan Drug Designation
Tubingen
1-50 employees
prevention of post-hepatectomy liver failure
Disease Area: Rare Diseases
August 5, 2026
Xadcera Biopharmaceutical (Suzhou) Co., Ltd.
Orphan Drug Designation
treatment of small cell lung cancer
Disease Area: Rare Diseases
August 5, 2026
United Therapeutics Corp
Neutral Outlook
Silver Spring, MD
1,001-5,000 employees
While the impact of the IRA on the pharmaceutical industry cannot yet be fully determined, it is likely to be significant.
Disease Area: Cardiology, Oncology, Rare Diseases, Respiratory Diseases
Drug Type: Biologic, Small Molecule
August 5, 2026
Sarepta Therapeutics, Inc.
Neutral Outlook
Cambridge, MA
501-1,000 employees
The U.S. government and individual states continue to aggressively pursue healthcare reform, which includes ongoing attempts to manage utilization as well as control and/or lower the cost of prescription drugs and biologics. Recent years have seen a number of reform initiatives focused on drug pricing and payment. For example, the Inflation Reduction Act (“IRA”), passed in 2022, has had and will likely continue to have a significant impact on the pharmaceutical industry.
Disease Area: Multiple, Neurological Diseases, Rare Diseases
Drug Type: Biologic, Small Molecule
August 5, 2026
Tenaya Therapeutics, Inc.
Neutral Outlook
South San Francisco, CA
51-200 employees
For example, the IRA passed by Congress authorizes the Secretary of the Department of HHS to negotiate prices directly with participating manufacturers for selected medicines covered by Medicare even if these medicines are protected by an existing patent. While we do not believe that the IRA or its effects will impact our ability to obtain patents in the near future, we cannot be certain whether it will affect our patent strategy in the long run.
Disease Area: Cardiology, Multiple, Rare Diseases
Drug Type: Small Molecule
August 5, 2026
Akebia Therapeutics, Inc.
Neutral Outlook
Cambridge, MA
201-500 employees
On August 16, 2022, the Inflation Reduction Act of 2022, or IRA, was signed into law by President Biden. Among other things, the IRA imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation (first due in 2023); and replaces the Part D coverage gap discount program with a new discounting program (beginning in 2025).
Disease Area: Hematology, Multiple, Rare Diseases
Drug Type: Small Molecule