Spiroplasma mirum

Mirum Pharmaceuticals Reports Third Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Monday, November 15, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Nausea, Stomach, Form, Pregnancy, Symptoms of COVID-19, Form 10-K, Cholestasis, Liver, Abdomen, Vitamin, Alagille syndrome, LDL, Breakthrough therapy, IBAT, Gastrointestinal bleeding, Food, European Medicines Agency, Vitamin A, Diarrhea, Spiroplasma mirum, Recycling, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Biliary atresia, Institute of Liver and Biliary Sciences, VISTAS, Primary sclerosing cholangitis, Vomiting, Pain, Marketing, Event, Primary biliary cholangitis, Volixibat, Cholestatic pruritus, PFIC, SEC, Liver injury, Pain scale, FSV, Webcast, Form 10-Q, Patient, Skin, FDA, Bone, Adult, Pharmaceutical industry, Medical imaging, LIVMARLI™ (maralixibat) oral solution, Volixibat, Mirum Pharmaceuticals, Inc., LIVMARLI™ (MARALIXIBAT) ORAL SOLUTION, VOLIXIBAT, MIRUM PHARMACEUTICALS, INC.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis and biliary atresia.

Key Points: 
  • LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis and biliary atresia.
  • LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia.
  • To learn more about ongoing clinical trials with LIVMARLI, please visit Mirums clinical trials section on the companys website.
  • Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases.

Mirum Pharmaceuticals to Report Third Quarter 2021 Financial Results and Host Conference Call on November 15, 2021

Retrieved on: 
Monday, November 8, 2021
Biotechnology, General Health, Health, Genetics, Pharmaceutical, Pregnancy, Cholestasis, Investor, Alagille syndrome, IBAT, European Medicines Agency, Spiroplasma mirum, Medication, Biliary atresia, Institute of Liver and Biliary Sciences, VISTAS, Primary sclerosing cholangitis, Marketing, Primary biliary cholangitis, Cholestatic pruritus, PFIC, Webcast, Nasdaq, Patient, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the company will report financial results for the quarter ended September 30, 2021 on Monday, November 15, 2021.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the company will report financial results for the quarter ended September 30, 2021 on Monday, November 15, 2021.
  • That same day, Mirum will host a conference call and audio webcast at 1:30 p.m. PT/4:30 p.m.
  • You may also access the call via webcast by visiting the Investors & Media section on Mirums corporate website.
  • Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases.

CANbridge Pharmaceuticals Enters into Duchenne Muscular Dystrophy Gene Therapy Research Agreement with University of Washington School of Medicine

Retrieved on: 
Monday, November 1, 2021
Research, Genetics, Biotechnology, Other Health, Health, University, Pharmaceutical, Science, Education, Death, Spiroplasma mirum, University, Weakness, Muscular dystrophy, Glioblastoma, Research, MPS, Degenerative disease, Goal, Glycogen storage disease, LSD, Neuromuscular disease, Patient, DMD, WuXi Biologics, Cardiomyopathy, MPS II, University of Massachusetts, Hunter syndrome, GBM, National Organization for Rare Disorders, Disease, University of Washington School of Medicine, CEO, University of Massachusetts Medical School, Male, Program, Haemophilia, University of Washington Department of Global Health, Neuromuscular Disorders, Duchenne muscular dystrophy, Incidence, Department of Neurology, Xuanwu hospital, Disorder, Lists of diseases, AAV, Department, Pharmaceutical industry, Vaccine, Dentistry, Fine chemical, Dystrophinopathies, the Chamberlain Laboratory, University of Washington Department of Neurology, the Odom Laboratory, University of Washington Department of Neurology, CANbridge Pharmaceuticals Inc., DYSTROPHINOPATHIES, THE CHAMBERLAIN LABORATORY, UNIVERSITY OF WASHINGTON DEPARTMENT OF NEUROLOGY, THE ODOM LABORATORY, UNIVERSITY OF WASHINGTON DEPARTMENT OF NEUROLOGY, CANBRIDGE PHARMACEUTICALS INC.

CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that it has entered into a two-year sponsored research agreement with the University of Washington School of Medicine, in Seattle, Washington, for gene therapy research in Duchenne muscular dystrophy (DMD), a rare neuromuscular disease.

Key Points: 
  • CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that it has entered into a two-year sponsored research agreement with the University of Washington School of Medicine, in Seattle, Washington, for gene therapy research in Duchenne muscular dystrophy (DMD), a rare neuromuscular disease.
  • The program will be under the direction of Jeffrey Chamberlain, Ph.D., professor in the Departments of Neurology, Medicine and Biochemistry, the McCaw Endowed Chair in Muscular Dystrophy at the University of Washington School of Medicine, and Director of the Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center of Seattle.
  • Guy Odom, Ph.D., Research Assistant Professor in the Department of Neurology at the University of Washington, will serve as the co-principal investigator.
  • His lab has been studying muscular dystrophy mechanisms, particularly dystrophin structure, and gene therapy approaches.

CANbridge Pharmaceuticals Enters into Exclusive Worldwide Research Collaboration and Licensing Agreement with Scriptr Global for Stitchr™ RNA Assembly Technology to Develop Gene Therapy Product Targeting Dystrophinopathies

Retrieved on: 
Monday, October 25, 2021
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Death, Spiroplasma mirum, University, Glioblastoma, Weakness, Research, Becker muscular dystrophy, Entrepreneurship, MPS, Glycogen storage disease, 2-Bromo-LSD, Heart, Patient, DMD, WuXi Biologics, Cardiomyopathy, RNA, MPS II, GBM, University of Massachusetts, Hunter syndrome, Inc., National Organization for Rare Disorders, Dystrophinopathy, Muscle tissue, Disease, Degenerative disease, CEO, University of Massachusetts Medical School, Male, BMD, Haemophilia, DCM, Prevalence, Duchenne muscular dystrophy, Incidence, Lists of diseases, Disorder, DNA, Pharmaceutical industry, 3D printing, Vaccine, Fine chemical, dystrophinopathies, SCRIPTR GLOBAL, CANbridge Pharmaceuticals Inc., DYSTROPHINOPATHIES, SCRIPTR GLOBAL, CANBRIDGE PHARMACEUTICALS INC.

CANbridge will gain exclusive worldwide rights to develop, manufacture and commercialize a gene therapy candidate for the treatment of dystrophinopathies, using Scriptr Globals Stitchr platform, a proprietary ribozyme-mediated RNA assembly technology.

Key Points: 
  • CANbridge will gain exclusive worldwide rights to develop, manufacture and commercialize a gene therapy candidate for the treatment of dystrophinopathies, using Scriptr Globals Stitchr platform, a proprietary ribozyme-mediated RNA assembly technology.
  • Scriptr Global will be responsible for research, while CANbridge will assume all responsibilities for development, manufacturing, regulatory, and commercialization.
  • We are pleased to be aligning with Scriptr Global and the Stitchr technology platform, which we believe has the potential to revolutionize the dystrophinopathy gene therapy field, said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc.
  • CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments.

CANbridge Pharmaceuticals CAN103 Investigational New Drug Application Approved in China for Treatment of Gaucher Disease

Retrieved on: 
Wednesday, October 20, 2021
Biotechnology, Pharmaceutical, Genetics, Health, ERT, Clinical trial, Spiroplasma mirum, Doctor of Philosophy, IND, CDE, Genetic distance, Degenerative disease, Cell, Investigational New Drug, Haemophilia, WuXi Biologics, Gaucher, Splenomegaly, Enzyme replacement therapy, University, Disease, Anemia, GBA, GBM, Research, Mutation, Bone pain, III, MPS II, Glioblastoma, Quality of life, CEO, Disorder, University of Massachusetts Medical School, LSS, Patient, Thrombocytopenia, LSD, Glycogen storage disease, Partnership, MPS, NMPA, Gaucher's disease, Frost & Sullivan, Hunter syndrome, Lists of diseases, University of Massachusetts, Hepatomegaly, Pharmaceutical industry, Fine chemical, Gaucher disease (GD), CANbridge Pharmaceuticals Inc., GAUCHER DISEASE (GD), CANBRIDGE PHARMACEUTICALS INC.

CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge as part of its rare disease partnership with WuXi Biologics (2269.HK) for the long-term treatment of adults and children with Gaucher disease, Types I and III.

Key Points: 
  • CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge as part of its rare disease partnership with WuXi Biologics (2269.HK) for the long-term treatment of adults and children with Gaucher disease, Types I and III.
  • Founder, Chairman and CEO of CANbridge Pharmaceuticals Inc. We are working diligently to commence our clinical trial program in CAN103, one of the candidates from our partnership with WuXi Biologics.
  • Despite the fact that Gaucher disease treatments have been available in China for decades, most patients do not have access, underscoring the need for China-based treatments and solutions.
  • CANbridge Pharmaceuticals Inc. is a China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies.

Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Friday, September 10, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials

The Compensation Committee of Mirums Board of Directors approved the awards as an inducement material to the new employees employment in accordance with Nasdaq Listing Rule 5635(c)(4).

Key Points: 
  • The Compensation Committee of Mirums Board of Directors approved the awards as an inducement material to the new employees employment in accordance with Nasdaq Listing Rule 5635(c)(4).
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.

Mirum Pharmaceuticals to Participate in September Investor Conferences

Retrieved on: 
Thursday, September 2, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, Patient, European Medicines Agency, PDUFA, Review, Pregnancy, Biliary atresia, Spiroplasma mirum, Primary biliary cholangitis, Primary sclerosing cholangitis, Cholestasis, Cholestatic pruritus, PFIC, Alagille syndrome, Investor, FDA, NDA, Pharmaceutical industry

Visit the Investors and Media section of Mirums corporate website for additional information.

Key Points: 
  • Visit the Investors and Media section of Mirums corporate website for additional information.
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.

Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Wednesday, August 11, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, European Medicines Agency, IND, Pregnancy, Therapy, Cholestatic pruritus, Cholestasis, Biliary atresia, Alagille syndrome, Patient, Primary biliary cholangitis, Primary sclerosing cholangitis, Financial compensation, Review, PFIC, Medication, Professional Football Compensation Committee, Employment, NDA, Nasdaq, FDA, Spiroplasma mirum, PDUFA, Pharmaceutical industry

The Compensation Committee of Mirums Board of Directors approved the awards as an inducement material to the new employees employment in accordance with Nasdaq Listing Rule 5635(c)(4).

Key Points: 
  • The Compensation Committee of Mirums Board of Directors approved the awards as an inducement material to the new employees employment in accordance with Nasdaq Listing Rule 5635(c)(4).
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.

Mirum Pharmaceuticals Reports Second Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Thursday, August 5, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, Investor, Primary sclerosing cholangitis, Webcast, Low-density lipoprotein, Abdominal pain, U.S. Securities and Exchange Commission, Prescription Drug User Fee Act, Cholestasis, Primary biliary cholangitis, Liver disease, Biliary atresia, Cholestatic pruritus, Feces, Security (finance), Private Securities Litigation Reform Act, IND, Review, Vistas, European Medicines Agency, Patient, PFIC, ABCB11, COVID-19, Growth, Database, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Volixibat, Spiroplasma mirum, Diarrhea, Food and Drug Administration, Itch, Alagille syndrome, Adult, Pregnancy, NDA, Liver, Recycling, Therapy, Food, Xanthoma, Pharmaceutical industry, Maralixibat, Volixibat, Mirum Pharmaceuticals, MARALIXIBAT, VOLIXIBAT, MIRUM PHARMACEUTICALS

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.

Key Points: 
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.
  • Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.