Amicus Therapeutics Fighting Rare and Orphan Diseases with Relentless Research

Based in Cranbury, New Jersey, Amicus Therapeutics, is a biopharmaceutical company that develops therapeutics for rare and orphan diseases that are called lysosomal storage disorders.

Their platform, CHART, focuses on developing of enzyme replacement therapies with which to treat these lysosomal disorders. They are known in the biopharmaceutical industry as having an abundance of small molecule pharmacological chaperones.

As Amicus Therapeutics does not have the capability of manufacturing their products on their own they must rely on contracting it to other manufacturers, such as JCR Pharmaceutical and GlaxoSmithKline. They worked with them from 2010 to 2013 in investigating the coformulation and recombinant alpha-galactosidase for the advancement of migalastat monotherapy.

Amicus Therapeutics expanded its research facility from New Jersey, opening a second research site in San Diego, California in 2008 (Yahoo Finance). Then in 2010, they were able to collaborate with David Geffen at the School of Medicine,UCLA due to a grant received for $500,000 from the Michael J. Fox Foundation, the same year they received another grant for $210,300 from the Alzheimer’s Drug Discovery Foundation for pre-clinical research with Ichan School of Medicine, Mount Sinai, working with the Alzheimer’s Disease Research Center.

In 2013, a competitor of Amicus, Callidus Biopharma was acquired which gained them intellectual property and proprietary access for an enzyme replacement treatment for Pompe disease, an inherited Lysosomal Storage Disorder caused by the body not producing the enzyme acid alpha-glucosidase or GAA.

Their leading product, migalastat, which is used to treat persons with Fabry disease,a disease that can lead to progressive and irreversible organ damage. This disease will affect the nervous system as well as cardiac, renal and a multitude of other tissues. Migalastat is in the late stages of development.

Amicus has built a belief statement to cover their core values plus be a reminder for the reason they do every day what they do. Part of the belief is that they believe in the fight against these diseases, they strive to have the highest quality therapies for anyone with these diseases. They firmly believe in supporting the communities of these diseases and their families.
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Dr. Clay Siegall: A Study Reports Promising Results in Treatment of Rare Malignancies


Many clinical trials set up to try efficiency of new medications simply fail. They do not draw in enough patients, and they are not completed. This trend decelerates medical progress. However, NCI-MATCH is a study that has gone against that trend. The study has come up with a unique way of categorizing cancers for treatment. Contrary to the traditional method that classifies tumors based on the organ where they are first detected, this technique of sorting concentrate on specific mutations in the genetic makeups of cancer cells. According to oncologist Robert Comis, NCI-MATCH analyzes the distinct mutations that take place in the tumors.

NCI-MATCH in brief

NCI-MATCH enrolls patients who have consistently attained unsuccessful results from traditional cancer treatments. Nancy Nahmias is an example of a patient who reacted poorly to chemotherapy to the extent of developing a severe reaction known as sepsis, which led to her hospitalization for six weeks. Nahmias’ doctors told her that standard chemotherapy was not an ideal option for her. Her daughter, who serves as a physician, found out that the NCI-MATCH trial was ongoing and urged her mother to try it. After screening and analysis of the genetic pattern of malignancy, scientists discovered that her mutation might be responsive to a treatment rarely offered to people with liver cancer. In its first three months, the trial enrolled 800 patients.

Who is Dr. Clay Siegall?

Dr. Siegall opened up Seattle Genetics in 1998. He is the firm’s CEO, executive president, and Board of Directors’ member. Seattle Genetics is in the process of establishing a diverse portfolio of preclinical and clinical drug candidates targeting a broad range of autoimmune and cancer indications. Dr. Siegall has revolutionized the company into an authority in antibody technologies, particularly the antibody-drug conjugate technology.

Dr. Siegall has supervised numerous venture-funding campaigns that have allowed Seattle Genetics to generate over $330 million inclusive of the firm’s IPO back in 2001. The biotechnology champion has structured strategic partnerships, such as a global partnership with Genentech worth over $800 million. It has also entered several ADC partnerships with Genentech, Progenics, CuraGen, MedImmune, and Bayer. Since 2011, Seattle Genetics’ ADC collaborations have garnered over $65 million.