Hot Investor Mandate 2: Pharma Firm Looks Worldwide for Treatments for Ultra Rare Diseases

9 Mar

A pharma firm based in the US specializes in treatments for diseases with under 20,000 patients in the USA or EU, and is seeking to in-license novel therapeutics and platforms in this space.

The firm is interested in therapeutic assets that treat severe, rare to ultra-rare diseases with high levels of mortality/morbidity. The firm is interested in assets at any stage, from discovery and early preclinical (i.e. where there may only be an animal model of disease that exists but no therapeutic yet) to marketed products. The firm requires that a treatment be transformative for patient care. While the firm’s primary experience is in antibodies and protein-based drugs, the firm is open to exploring opportunities that utilize other therapeutic modalities such as small molecules, gene therapy and nucleic acids. In addition, the firm is looking for new therapeutic platform technologies, especially those that can enhance the blood brain barrier penetrance of biologics as well as new methods to deliver nucleic acids to cells and target organs.

The firm is open to working with companies anywhere in the world, and has no set requirements for partners.

If you are interested in more information about this investor and other investors tracked by LSN, please email

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

%d bloggers like this: