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Top 41 Rare diseases treatment startups
Oct 02, 2025 | By Jason Kwon | 22 |
These startups develop new technologies to treat rare diseases, such as gene therapies, precision medicines.
1
Country: USA | Funding: $64.3M
Arnatar Therapeutics is a biopharmaceutical company that specializes in the development of novel nucleic acid medications.
Arnatar Therapeutics is a biopharmaceutical company that specializes in the development of novel nucleic acid medications.
2
Country: France | Funding: $315.9M
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.
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.
3
Country: Japan | Funding: $382.6M
Shionogi supply the best possible medicine to protect the health and wellbeing of the patients they serve.
Shionogi supply the best possible medicine to protect the health and wellbeing of the patients they serve.
4
Country: USA | Funding: $3.9B
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.
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.
5
Country: USA | Funding: $3.4B
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
6
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.
Amicus Therapeutics is a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases.
7
Country: USA | Funding: $1.3B
Dyne Therapeutics is a new biotechnology company pioneering targeted therapies for patients with serious muscle diseases
Dyne Therapeutics is a new biotechnology company pioneering targeted therapies for patients with serious muscle diseases
8
Country: USA | Funding: $1B
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.
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.
9
Country: USA | Funding: $398M
Rezolute is a clinical-stage biopharma specializing in the development of drug therapies for patients with metabolic and orphan diseases.
Rezolute is a clinical-stage biopharma specializing in the development of drug therapies for patients with metabolic and orphan diseases.
10
Country: USA | Funding: $379.5M
Keros Therapeutics develops novel treatments for patients suffering from hematologic and musculoskeletal disorders with high unmet need
Keros Therapeutics develops novel treatments for patients suffering from hematologic and musculoskeletal disorders with high unmet need
11
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.
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.
12
Country: USA | Funding: $360M
LEXEO Therapeutics develops gene therapy for the world's most devastating diseases
LEXEO Therapeutics develops gene therapy for the world's most devastating diseases
13
Country: USA | Funding: $301.6M
Entrada Therapeutics is a developer of novel therapeutics intended to treat devastating diseases such as Duchenne muscular dystrophy (DMD).
Entrada Therapeutics is a developer of novel therapeutics intended to treat devastating diseases such as Duchenne muscular dystrophy (DMD).
14
Country: USA | Funding: $238.6M
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).
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).
15
Country: Sweden | Funding: $210.4M
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.
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.
16
Country: USA | Funding: $195M
Glycomine is an early-stage biotech working on replacement therapies for rare diseases.
Glycomine is an early-stage biotech working on replacement therapies for rare diseases.
17
Country: Denmark | Funding: $190M
Hemab is focused on the development of bispecific antibodies for the treatment of rare bleeding disorders.
Hemab is focused on the development of bispecific antibodies for the treatment of rare bleeding disorders.
18
Country: Denmark | Funding: $190M
Hemab is focused on the development of bispecific antibodies for the treatment of rare bleeding disorders.
Hemab is focused on the development of bispecific antibodies for the treatment of rare bleeding disorders.
19
Country: Netherlands | Funding: €157M
Azafaros B.V. aims at developing new therapeutic agents for the treatment of rare metabolic disorders.
Azafaros B.V. aims at developing new therapeutic agents for the treatment of rare metabolic disorders.
20
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.
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.
