Top 10 Rare diseases treatment startups

Updated: March 03, 2024

Rare disease treatment startups are dedicated to addressing the unmet needs of individuals affected by rare diseases by developing innovative therapies and solutions. These startups leverage advanced technologies such as gene therapies, precision medicine approaches, and rare disease registries to revolutionize the field.
1
Country: USA | Funding: $3.2B
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
2
Country: USA | Funding: $3B
At Moderna, we are pioneering the development of a new class of drugs made of messenger RNA (mRNA). This novel drug platform builds on the discovery that modified mRNA can direct the body’s cellular machinery to produce nearly any protein of interest, from native proteins to antibodies and other entirely novel protein constructs that can have therapeutic activity inside and outside of cells.
3
Country: USA | Funding: $2.4B
Amicus Therapeutics is a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases.
4
Country: USA | Funding: $1.5B
Zogenix engages in the development and commercialization of products for the treatment of central nervous system disorders and pain.
5
Country: USA | Funding: $614.6M
Dyne Therapeutics is a new biotechnology company pioneering targeted therapies for patients with serious muscle diseases
6
Country: USA | Funding: $433.5M
Dicerna Pharmaceuticals develops therapeutic agents in various disease areas based on dicer substrate technology platform. The company offers RNA interference (RNAi)-targeted drugs and delivery systems for therapeutic areas of oncology and metabolic diseases. It also provides Dicer Substrate Technology, a second generation of gene silencing, which triggers RNAi and is used in therapeutic areas, including inflammation, immunology, and cardiovascular diseases.
7
Country: USA | Funding: $414.8M
Disc Medicine is a hematology company that hopes can become the first disease-modifying treatment for erythropoietic porphyrias, a family of rare and debilitating genetic disorders caused by dysregulated heme synthesis.
8
Country: USA | Funding: $379.5M
Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases.
9
Country: USA | Funding: $341.6M
Rezolute is a clinical-stage biopharma specializing in the development of drug therapies for patients with metabolic and orphan diseases.
10
Country: USA | Funding: $232M
Rallybio develops antibodies, small molecules and engineered proteins for rare and orphan diseases (across a wide range of settings including central nervous system disorders, metabolic disorders, musculoskeletal diseases, nephrology and hematology).
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11
Country: USA | Funding: $230M
Keros Therapeutics develops novel treatments for patients suffering from hematologic and musculoskeletal disorders with high unmet need
12
Country: USA | Funding: $227.6M
Translate Bio is a leading mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction.
13
Country: USA | Funding: $201.6M
Entrada Therapeutics is a developer of novel therapeutics intended to treat devastating diseases such as Duchenne muscular dystrophy (DMD).
14
Country: Denmark | Funding: $190M
Hemab is focused on the development of bispecific antibodies for the treatment of rare bleeding disorders.
15
Country: Denmark | Funding: $190M
Hemab is focused on the development of bispecific antibodies for the treatment of rare bleeding disorders.
16
Country: USA | Funding: $185M
LEXEO Therapeutics develops gene therapy for the world's most devastating diseases
17
Country: France | Funding: $178.5M
Inventiva is a clinical stage biopharmaceutical company with an expertise in fibrosis, oncology and orphan diseases. We are focussing on diseases with a high unmet medical need where either no treatments are available, such as NASH and Systemic Sclerosis, or the current standard of care leaves important manifestations of the diseases unaffected, as it is the case in many lysosomal storage diseases.
18
Country: USA | Funding: $154.9M
Casma Therapeutics is a preclinical-stage therapeutics company focused on modulating the autophagy pathway to address unmet medical needs and transform the lives of patients.
19
Country: USA | Funding: $144.8M
Imara is provider of novel therapeutics for the people living with sickle cell disease and hemoglobinopathies.
20
Country: Spain | Funding: $143.9M
Minoryx Therapeutics offers pharmacological chaperones, which are small molecule drugs used for the treatment of genetic diseases.
21
Country: Sweden | Funding: $108.2M
Calliditas Therapeutics, a specialty pharmaceutical company that develops pharmaceutical products in fields of unmet medical need. Its products include Nefecon, a treatment for IgA nephropathy and Busulipo, an alternative for myeloablation prior to bone marrow transplantation.
22
Country: Switzerland | Funding: $100M
Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases
23
Country: USA | Funding: $98M
MyoKardia is pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Our initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically-driven forms of heart failure
24
Country: USA | Funding: $98M
LAM Therapeutics develops drugs for rare diseases and cancer.
25
Country: USA | Funding: $85M
Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies.
26
Country: Sweden | Funding: $68.9M
Wilson Therapeutics is a biopharmaceutical company that committed to improving lives of patients with Wilson Disease through the development of new treatment options.
27
Country: UK | Funding: $67.9M
Biotechnology company integrating artificial intelligence with expert pharmacology to discover treatments for rare diseases
28
Country: USA | Funding: $67.1M
rdmd empower patients and communities to change the way that drug development happens in rare disease.
29
Country: USA | Funding: $67.1M
AllStripes' mission is to unlock new treatments for people with rare disease
30
Country: Canada | Funding: $64.6M
Congruence Therapeutics is building a team of 'drug hunters' to go after rare disease targets
31
Country: Ireland | Funding: €49M
Priothera is dedicated to improving the lives of patients suffering from hematological malignancies by delivering an innovative immune modulator to enhance the curative potential of allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in these patients.
32
Country: USA | Funding: $35M
Aceragen is a biopharmaceutical company with a focus on rare illnesses hopes to be the first to find a treatment for Farber disease.
33
Country: Netherlands | Funding: $27.5M
Azafaros B.V. aims at developing new therapeutic agents for the treatment of rare metabolic disorders.
34
Country: France | Funding: €20M
InnoSkel is developing novel genetherapies for rare skeletal diseases
35
Country: Japan | Funding: $7.6M
Shionogi supply the best possible medicine to protect the health and wellbeing of the patients they serve.
36
Country: Spain | Funding: €5.1M
Bionure Farma develops innovative therapies for the treatment of orphan ophthalmology diseases and multiple sclerosis.
37
Country: UK | Funding: £4.1M
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