1
Country: USA | Funding: $253M
Diagonal Therapeutics develops agonist antibody-based drugs designed to correct abnormal signaling in severe genetic diseases such as hereditary hemorrhagic telangiectasia. The company is also exploring its antibody as a treatment for the underlying cause of pulmonary arterial hypertension. It has demonstrated significant disease-modifying activity in several preclinical models of NHT and PAH and restore normal signaling in patient-derived cells. The company claims its antibodies are highly specific, easy to manufacture and convenient for use in patients, with potential for modification during disease management.
Diagonal Therapeutics develops agonist antibody-based drugs designed to correct abnormal signaling in severe genetic diseases such as hereditary hemorrhagic telangiectasia. The company is also exploring its antibody as a treatment for the underlying cause of pulmonary arterial hypertension. It has demonstrated significant disease-modifying activity in several preclinical models of NHT and PAH and restore normal signaling in patient-derived cells. The company claims its antibodies are highly specific, easy to manufacture and convenient for use in patients, with potential for modification during disease management.
2
Country: USA | Funding: $100M
Addition Therapeutics (University of California spinoff) develops RNA-only therapeutics using PRINT technology for genetic medicine. PRINT stands for Precise RNA-Mediated Insertion of Transgenes and involves the creation of all-RNA, non-viral lipid nanoparticles. In drugs created using PRINT technology, only the genetic sequence of the messenger RNA is altered, all other components remain unchanged. The company claims this enables the creation of safer, long-lasting, single-dose treatments that overcome the limitations of existing genetic medicine. One of the company's primary goals is the treatment of HIV. With a grant from the Gates Foundation, Addition is developing PRINT therapy to stimulate endogenous antibody production with a single dose for "lifelong protection against HIV."
Addition Therapeutics (University of California spinoff) develops RNA-only therapeutics using PRINT technology for genetic medicine. PRINT stands for Precise RNA-Mediated Insertion of Transgenes and involves the creation of all-RNA, non-viral lipid nanoparticles. In drugs created using PRINT technology, only the genetic sequence of the messenger RNA is altered, all other components remain unchanged. The company claims this enables the creation of safer, long-lasting, single-dose treatments that overcome the limitations of existing genetic medicine. One of the company's primary goals is the treatment of HIV. With a grant from the Gates Foundation, Addition is developing PRINT therapy to stimulate endogenous antibody production with a single dose for "lifelong protection against HIV."
3
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.
4
Country: USA | Funding: $3.4B
Genetix Biotherapeutics (formerly Bluebird Bio) develops gene therapies for rare genetic diseases using patient's own blood stem cells. The cells are harvested, modified outside the body to add working copies of the affected gene and then reintroduced into the patient, restoring the function of the affected gene. The company specializes in the treatment of sickle cell anemia, beta thalassemia and cerebral adrenoleukodystrophy (CALD). Its unique, single-dose treatments are FDA approved and address the underlying cause of the disease and can provide significant, long-term benefits to patients. Acquired by The Carlyle Group
Genetix Biotherapeutics (formerly Bluebird Bio) develops gene therapies for rare genetic diseases using patient's own blood stem cells. The cells are harvested, modified outside the body to add working copies of the affected gene and then reintroduced into the patient, restoring the function of the affected gene. The company specializes in the treatment of sickle cell anemia, beta thalassemia and cerebral adrenoleukodystrophy (CALD). Its unique, single-dose treatments are FDA approved and address the underlying cause of the disease and can provide significant, long-term benefits to patients. Acquired by The Carlyle Group
5
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.
6
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
7
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.
8
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.
9
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.
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: 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.
14
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).
15
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.
16
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).
17
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.
18
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.
19
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.
20
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.
21
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.
22
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.
23
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.
24
Country: UK | Funding: $114.9M
Biotechnology company integrating artificial intelligence with expert pharmacology to discover treatments for rare diseases
Biotechnology company integrating artificial intelligence with expert pharmacology to discover treatments for rare diseases
25
Country: Canada | Funding: $101.6M
Congruence Therapeutics is building a team of 'drug hunters' to go after rare disease targets
Congruence Therapeutics is building a team of 'drug hunters' to go after rare disease targets
26
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
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
27
Country: USA | Funding: $85M
Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies.
Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies.
28
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.
29
Country: Ireland | Funding: €50.7M
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.
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.
30
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.
Aceragen is a biopharmaceutical company with a focus on rare illnesses hopes to be the first to find a treatment for Farber disease.
31
Country: USA | Funding: $14.5M
Citizen Health is a company that helps patients oversee rare and complex health conditions
Citizen Health is a company that helps patients oversee rare and complex health conditions
32
Country: Spain | Funding: €5.1M
Bionure Farma develops innovative therapies for the treatment of orphan ophthalmology diseases and multiple sclerosis.
Bionure Farma develops innovative therapies for the treatment of orphan ophthalmology diseases and multiple sclerosis.
33
Country: UK | Funding: £4.1M
Raremark is an online service which makes scientific and medical knowledge accessible and understandable for patients and carers.
Raremark is an online service which makes scientific and medical knowledge accessible and understandable for patients and carers.
