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Top 38 Rare diseases treatment startups
Oct 10, 2024 | By Jason Kwon | 22 |
These startups develop new technologies to treat rare diseases, such as gene therapies, precision medicines.
1
Country: USA | Funding: $940.1M
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
2
Country: USA | Funding: $592.8M
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.
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.
3
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).
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: $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.
6
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.
7
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.
8
Country: USA | Funding: $1.5B
Zogenix engages in the development and commercialization of products for the treatment of central nervous system disorders and pain.
Zogenix engages in the development and commercialization of products for the treatment of central nervous system disorders and pain.
9
Country: USA | Funding: $442.6M
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.
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.
10
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.
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.
