1
Country: USA | Funding: $3.9B
Moderna is a pioneer in developing a new class of drugs based on messenger RNA (mRNA). This new drug platform is based on the discovery that in-vitro engeneered and injected mRNA can direct the body's cellular machinery to produce virtually any protein of interest, from native proteins to antibodies and other entirely new protein constructs with therapeutic activity both inside and outside cells. In other words, mRNA can guide the body to produce its own drugs. Moderna used mRNA to create the first COVID-19 vaccine and is now applying this technology to create vaccines for infectious diseases (influenza, RSV, HSV, etc.), immuno-oncology (melanoma, lung cancer, liver cancer, etc.), rare diseases and autoimmune diseases.
Moderna is a pioneer in developing a new class of drugs based on messenger RNA (mRNA). This new drug platform is based on the discovery that in-vitro engeneered and injected mRNA can direct the body's cellular machinery to produce virtually any protein of interest, from native proteins to antibodies and other entirely new protein constructs with therapeutic activity both inside and outside cells. In other words, mRNA can guide the body to produce its own drugs. Moderna used mRNA to create the first COVID-19 vaccine and is now applying this technology to create vaccines for infectious diseases (influenza, RSV, HSV, etc.), immuno-oncology (melanoma, lung cancer, liver cancer, etc.), rare diseases and autoimmune diseases.
2
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.
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.
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: $1.3B
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
5
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.
6
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.
7
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.
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.
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: $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
10
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
11
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.
12
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).
13
Country: USA | Funding: $283M
Electra Therapeutics is a clinical stage biotechnology company developing therapies for cancer and other immunological diseases.
Electra Therapeutics is a clinical stage biotechnology company developing therapies for cancer and other immunological diseases.
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: Spain | Funding: €154.2M
Minoryx Therapeutics offers pharmacological chaperones, which are small molecule drugs used for the treatment of genetic diseases.
Minoryx Therapeutics offers pharmacological chaperones, which are small molecule drugs used for the treatment of genetic diseases.
