5 Top Orphan Drug Startups Impacting The Pharma Industry StartUs Insights

5 Top Orphan Drug Startups Impacting The Pharma Industry

We analyzed 231 orphan drug startups impacting pharma. Tiburio Therapeutics, Versantis, MeiraGTX, Dynacure, and Belite Bio 5 develop 5 top solutions. Learn more in our Global Startup Heat Map!

Our Innovation Analysts recently looked into emerging technologies and up-and-coming startups working on solutions for the pharma sector. As there are many startups working on a wide variety of solutions, we want to share our insights with you. This time, we take a look at 5 promising orphan drug startups.

Heat Map: 5 Top Orphan Drug Startups

Using our StartUs Insights Platform, covering 1.116.000+ startups & emerging companies, we looked at innovation in the drug discovery field. For this research, we identified 231 relevant solutions and picked 5 to showcase below. These companies were chosen based on a data-driven startup scouting approach, taking into account factors such as location, founding year, and technology among others. Depending on your specific criteria, the top picks might look entirely different.

The Global Startup Heat Map below highlights 5 startups & emerging companies developing orphan drug solutions. Moreover, the Heat Map reveals regions that observe a high startup activity and illustrates the geographic distribution of all 231 companies we analyzed for this specific topic.


Click to enlarge

Which startups develop the other 226 solutions?


Tiburio Therapeutics – Rare Endocrine Diseases

It is economically infeasible for conventional pharma companies to develop drugs for rare diseases as they have a small market size. Despite this, orphan drugs meet the needs of patients suffering from rare diseases. These include hundreds of rare endocrine diseases, each of which only affects a small subset of the population. Unlike widely common endocrine diseases such as diabetes, these receive little to no attention.

Tiburio Therapeutics is a US-based orphan drug startup. The company develops dopamine-somatostatin chimeric molecules that offer therapeutic potential for endocrine diseases with unmet needs. TBR-065, a compound for the treatment of rare endocrine diseases, is in Phase 1 trials. A similar compound, TBR-760, is in Phase 2 trials and treats non-functioning pituitary adenoma (NFPA).

Versantis – Acute Liver Diseases

Rare liver conditions include acute diseases and congenital metabolic disorders with startling mortality rates. These affect a varying number of people, with acute liver failure and acute-on-chronic liver failure affecting anywhere between a few thousand to a hundred thousand patients yearly in the US and Europe. With the push for orphan drug development in recent years, many drugs targeting these conditions are now being researched.

Swiss startup Versantis produces orphan drugs for patients with liver and rare pediatric diseases. VS-01 is a liposomal-based intra-peritoneal fluid that clears a large spectrum of toxic metabolites from the liver. It is undergoing clinical investigations for the treatment of acute-on-chronic liver failure, decompensated cirrhosis, and urea cycle disorders. The startup also develops drugs for chronic liver diseases and drug intoxications.

MeiraGTx – Achromatopsia

Achromatopsia, partial or complete absence of color vision, is a rare, inherited condition that is present in only 1 in 30,000 people. In most cases, it is due to mutations in two of the six genes that encode components of the light-sensing machinery of photoreceptors in the eye. Advances in gene therapy now enable the correction of inherited mutations to treat congenital conditions.

MeiraGTx is a UK-based gene therapy company that develops treatments for serious diseases with unmet medical needs. The company uses adeno-associated viral (AAV) vectors to offer gene therapies for ocular, neurodegenerative, and salivary gland diseases. AAV-CNGB3 and AAV-CNGA3 are two candidates for the treatment of achromatopsia that are delivered via subretinal injections and are undergoing clinical trials.

Dynacure – Centronuclear Myopathies (CNM)

Centronuclear Myopathies include a group of rare genetic muscular diseases. They cause muscle weakness and may lead patients to need wheelchairs or ventilators in extreme cases. However, since these diseases affect only a few thousand patients, there isn’t sufficient motivation for the pharma industry to invest in them. However, incentives for orphan drug development and advances in gene editing now enable drug development for such rare conditions.

France-based startup Dynacure works on drugs for rare and orphan diseases. The company uses antisense oligonucleotide technology to inhibit the production of the disease-causing protein. DYN101, an investigational drug from Dynacure, modulates the expression of dynamin 2 to treat CNM. The drug has received orphan drug designation by the Food and Drug Administration (FDA).

Belite Bio – Age-Related Macular Degeneration (AMD)

Unlike the other diseases mentioned above, age-related macular degeneration is a common disease. In fact, it is one of the leading causes of vision loss and mainly affects the elderly. If not diagnosed early, it is incurable in later stages. With the advances in nucleotide synthesis and delivery mechanisms, antisense drugs that block the action of a particular protein now offer a cure for erstwhile incurable diseases.

Belite Bio is a US-based startup that develops treatment for age-related metabolic diseases. Their Anti-RBP4 platform targets a vitamin A transporter protein linked to the development of aging metabolic diseases. LBS-008 is an oral drug that targets dry AMD and is currently undergoing clinical trials. The drug has obtained orphan drug designation in both the US and Europe.

What About The Other 226 Solutions?

While we believe data is key to creating insights it can be easy to be overwhelmed by it. Our ambition is to create a comprehensive overview and provide actionable innovation intelligence so you can achieve your goals faster. The 5 orphan drug startups showcased above are promising examples out of 231 we analyzed for this article. To identify the most relevant solutions based on your specific criteria, get in touch.

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