The fibroblast growth factor receptor protein is impacted by the genetic condition achondroplasia in the body. In achondroplasia, this protein starts to behave improperly, delaying bone formation in the cartilage of the growth plate. Achondroplasia causes shorter, improperly formed, and shorter bones as a result; individuals with the condition can be 42 to 56 inches tall. A parent or kid may pass on the genetic flaw. Nevertheless, achondroplasia is caused by a spontaneous mutation (a sudden genetic error) that develops in the developing embryo in around 80% of cases.
In 2022, the market for Achondroplasia is anticipated to reach a value of US$ 93.82 million. display a CAGR of 36.0% during the course of the projected (2022-2030).
Leading Companies in the Achondroplasia Industry:
1. BioMarin.
Achondroplasia, Duchenne muscular dystrophy, phenylketonuria, PKU, late infantile neuronal ceroid lipofuscinosis (CLN2), and haemophilia A are only a few of the uncommon genetic disorders and medical problems that are treated by BioMarin Pharmaceutical Inc. (BioMarin), which was founded in March 1997. The firm focuses on creating ground-breaking medicines for fatal and crippling illnesses. Many clinical and preclinical products are included in this product category, including Naglazyme-Mucopolysaccharidosis Type VI (MPS VI), Aldurazyme-MPS I, and others.The company operates through its manufacturing facilities in Novato, California. It conducts business operations in the US, Europe, Latin America, the Middle East, and Asia-Pacific. BioMarin is headquartered in San Rafael, California, the US.
2. Ribomic, Inc.
Ribomic, Inc. was established on August 1st, 2003, and produces aptamers, which are ribonucleic acids (RNA). The business carries out research and creates fresh pharmaceuticals based on the Ribomic Aptamer Refined Therapeutics System. The business also provides product licencing advice to pharmaceutical firms. RIBOMIC Inc. provides an update on the RBM-007 programme in wet age-related macular degeneration on March 23, 2022. Based on the Ribomic Aptamer Refined Therapies System platform and aptamer drug discovery technology, RIBOMIC Inc. is in the drug development industry. Self-discovery and co-discovery divisions make up its drug discovery business. The division responsible for self-drug discovery creates aptamers and licences the outcomes to pharmaceutical firms.In exchange for sharing in the co-discovery business with alliance pharmaceutical businesses developing aptamer pharmaceutical, the alliance partner pays the research expenditures to the Co-Discovery division.
3. BridgeBio Pharma Inc..
BridgeBio Pharma Inc (BridgeBio), a clinical-stage biotechnology business founded in 2015, creates cutting-edge medications for genetic illnesses. Acoramidis, fosdenopterin, patidegib topical gel, low-dose infigratnib, BBP-589, BBP-551, BBP-418, BBP-681, BBP-671, and other mendelian disease medications are included in the company's pipeline product portfolio. Precision oncology medications such as high-dose infigratinib, BBP-398, BBP-454, and BBP-954 are also included. Moreover, BridgeBio conducts pre-clinical, phase I, and phase II investigations as part of clinical trials. Academic institutions that work with the corporation include, among others, Medical University of South Carolina, University of Pittsburgh, Oregon Health, and Science University. Among its business partners are Helsinn, Alexion, and Bristol-Myers Squibb. US-based BridgeBio is based in Palo Alto, California.
4. Ascendis Pharma A/S.
Ascendis Pharma A/S is a manufacturer of pharmaceutical goods that was established in 2006. The company makes medications to treat conditions like diabetes, endocrinology, central nervous system problems, viral infections, and low levels of growth hormone. A biopharmaceutical business called Ascendis Pharma AS (Ascendis Pharma) specialises on creating medications for cancer and endocrinology diseases. TransCon hGH, a long-acting human growth hormone prodrug that targets GHD (growth hormone deficiency), TransCon PTH, a drug that treats hypoparathyroidism by reestablishing physiological levels of PTH (parathyroid hormone), and TransCon CNP, a CNP (C-type natriuretic peptide) drug against achondroplasia are all being studied (ACH).Moreover, TransCon IL-2 /, a form of interleukin-2 that stimulates the IL-2 receptor beta/gamma (IL-2R/), and TransCon TLR (toll-like receptor) 7/8 agonist are being studied for the treatment of cancer. To create its medications, Ascendis Pharma makes use of its exclusive TransCon technology platform. The US, Germany, and Denmark are where the corporation conducts business. The headquarters of Ascendis Pharma are in Copenhagen, Denmark.
5. PhaseBio Pharmaceuticals, Inc.
PhaseBio Pharmaceuticals, Inc. is a biopharmaceutical business that was established in 2002. The company creates elastin-like polypeptides to lengthen the half-life, boost therapeutic medication effectiveness, and make administration simpler. PhaseBio Pharmaceuticals develops new medications to address metabolic, cardiovascular, and endocrine conditions. A clinical-stage biopharmaceutical firm called PhaseBio Pharmaceuticals, Inc. is dedicated to the creation and marketing of cutting-edge treatments for cardiovascular and cardiopulmonary illnesses. Bentracimab (PB2452), a brand-new ticagrelor-reversal agent, pemziviptadil (PB1046), a once-weekly vasoactive intestinal peptide receptor agonist for the treatment of pulmonary arterial hypertension, and PB6440, an oral agent for the management of resistant hypertension, are all in the company's pipeline.The creation of medicines with the potential for less-frequent dosage and enhanced pharmacokinetics, such as pemziviptadil, is made possible by PhaseBio's patented elastin-like polypeptide technology platform, which also spurs options for internal and joint drug research. Malvern, Pennsylvania in the United States serves as its headquarters.
6. Pfizer Inc.
Charles Pfizer and his relative Charles F. Erhart, two German businessmen, founded the corporation in New York in 1849. Biopharmaceuticals are discovered, developed, produced, and sold by Pfizer Inc. The business sells goods to cure a range of ailments, including cancer, inflammation, rare illnesses, metabolic, cardiovascular, and metabolic and pain problems, as well as women's health issues. Moreover, it offers contract manufacturing services, sterile injectable drugs, biosimilars, active pharmaceutical ingredients (APIs), and medicines. Pfizer offers its goods through distributors, merchants, pharmacies, hospitals, private physician offices, clinics, and public institutions. India, China, Japan, Ireland, Italy, Belgium, Germany, Singapore, and the US are all home to significant production facilities for it. The firm ships its goods to Europe, Africa, the Middle East, Asia-Pacific, Australia, and North and South America. The headquarters of Pfizer are in New York.
7. F. Hoffmann-La Roche Ltd .
F. Hoffmann-La Roche Ltd. (Roche) is a biotechnology business that creates medications and diagnostics to treat serious diseases. The company was founded in 1896. It offers medications for the treatment of respiratory illnesses, cancer, various auto-immune diseases, neurological disorders, ophthalmological disorders, and central nervous system problems. The business also provides diabetes management programmes, tissue-based cancer diagnostics, and in vitro diagnostics. Roche carries out research to find cutting-edge ways to treat, diagnose, and prevent illnesses. Hospitals, commercial laboratories, healthcare professionals, researchers, and pharmacists are among the groups that the business serves with its goods and services. The business operates in several nations along with its affiliates and partners. The headquarters of Roche are in Basel, Switzerland.
8. JCR Pharmaceuticals Co Ltd (JCR)
JCR Pharmaceuticals Co Ltd (JCR), a company established on September 13, 1975, produces, sells, purchases, imports, and exports pharmaceutical goods, regenerative medicines, and drug ingredients. For the treatment of uncommon disorders, it offers biopharmaceuticals in the field of regenerative medicine. In addition to a regenerative medical product for acute graft-versus-host disease and other disorders, the company offers pharmaceuticals to treat growth hormone deficiency, Turner syndrome, adult growth hormone deficiency, small for gestational age, renal anaemia in dialysis patients, and anaemia of prematurity. JCR is also involved in the buying, selling, importing, and exporting of lab equipment and medical products. It has subsidiaries in the US, Switzerland, Japan, and Brazil. Ashiya, Hyogo, Japan is home to JCR's headquarters.
*Definition: The fibroblast growth factor receptor protein is impacted by the genetic condition achondroplasia in the body.
