ERT

David Herron Appointed as Calyx CEO

Retrieved on: 
Wednesday, April 19, 2023
Professional Services, Health, Technology, Finance, Software, Biotechnology, LLP, Executive, Herron, ERT, Acquisition, Patient, Partnership, Growth, CapVest Limited, Management, Calyx

GLO Healthcare, an expanding healthcare platform formed by CapVest Partners LLP, announces the appointment of David Herron as Calyx Chief Executive Officer (CEO).

Key Points: 
  • GLO Healthcare, an expanding healthcare platform formed by CapVest Partners LLP, announces the appointment of David Herron as Calyx Chief Executive Officer (CEO).
  • Prior to joining Calyx, Herron served as President and CEO of Bioclinica (now part of Clario).
  • Commenting on the appointment, Renaud Dehareng, Executive Chairman of Calyx said: “We are delighted to welcome David to the Calyx team.
  • Kate Briant, Senior Partner at CapVest, will remain a member of the Calyx Board, and as chair of GLO Healthcare.

ClinOne Closes Series A Funding Round to Scale its Site and Participant Engagement platform, Data Driven Insights and Interoperability

Retrieved on: 
Thursday, April 13, 2023
Kohlberg Kravis Roberts, Medicine, Partnership, Central Waqf Council, ARCA, ERT, Consent management, Health care, Clinical trial, Arbutus Biopharma, ICON, General partnership, Investment company, ICON PLC, Patient, Community, Genstar Capital, Growth, Health, Environment, Caregiver, Pharmaceutical industry

BOSTON and BOULDER, Colo., April 13, 2023 /PRNewswire-PRWeb/ -- ClinOne, the leading provider of Adaptive Experiences in clinical trials announced today it closed a $3 million Series A funding to accelerate the growth of its platform and broaden its reach to serve customers. This latest round, led by Boulder Ventures, with participation from existing investors CU Healthcare Innovation Fund and First Trust Capital Partners, will continue to advance ClinOne's mission to offer flexible engagement solutions for all participants to support their clinical trial journey.

Key Points: 
  • As part of this investment, Robert Conway will join the ClinOne Board as Executive Chair.
  • ClinOne sells an integrated platform that offers comprehensive support for sites, participants, and caregivers throughout the clinical trial journey.
  • ClinOne has built a platform which offers ease of use along with comprehensiveness and efficiency for all stakeholders to meet.
  • ClinOne makes their participation in clinical trials simple, reliable, and useful, and by offering flexibility, can reach a diverse group of participants.

Lokavant Hires Experienced Leaders to Accelerate Growth Trajectory

Retrieved on: 
Tuesday, April 4, 2023
Research, Apps, Applications, Technology, Clinical Trials, Professional Services, Software, Data Analytics, Pharmaceutical, Health, Science, Mills, GSK plc, IBM, Meredith, SOCOM U.S. Navy SEALs: Confrontation, Marketing, Clinical trial, ICON, Intelligence, Science 37, Roivant Sciences, ERT, Patient, News, Management, Pharmaceutical industry

Lokavant, the clinical trial intelligence platform company, announces strategic new hires to drive its mission to make clinical trials smarter.

Key Points: 
  • Lokavant, the clinical trial intelligence platform company, announces strategic new hires to drive its mission to make clinical trials smarter.
  • Mills worked previously as global vice president of sales for Clario, and in various commercial leadership roles at ICON.
  • “Having been in the industry for over 20 years, I have not seen a more exciting company than Lokavant come to market,” said Mills.
  • Weatherhead came to Lokavant from Castor, a provider of decentralized clinical trial technology, where he was vice president of marketing.

CANbridge Announces Financial Results and Corporate Updates for Twelve Months Ended December 31, 2022

Retrieved on: 
Friday, March 31, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, IPO, Glycogen storage disease type II, American Society of Gene and Cell Therapy, Congress, Investigational New Drug, Patient, MG, Cell, IBAT, Enzyme replacement therapy, Fabry disease, DSM-IV codes, University of Massachusetts, Gene, Food, GD, Glioblastoma, Paroxysmal nocturnal hemoglobinuria, ESGCT, Rare, FDA, IND, Safety, COVID-19, MPS II, Myasthenia gravis, MPS, AAV, PNH, Alexion, Alagille syndrome, Hunter syndrome, Gene therapy, SMA, Liver disease, Clinical trial, NDA, Part, Catholic Medical Mission Board, ERT, Mouse, European Society for Primary Immunodeficiencies, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Society, Humanitarian use licenses, European Society of Gene and Cell Therapy, Pharmaceutical industry, Cryptocurrency, Vaccine, Fabry, C5

CANbridge Pharmaceuticals, Inc. (“CANbridge,” stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced financial results for the twelve months ended 2022 and corporate update.

Key Points: 
  • CANbridge Pharmaceuticals, Inc. (“CANbridge,” stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced financial results for the twelve months ended 2022 and corporate update.
  • The adjusted loss for the year decreased by RMB124.6 million, or 21.4%, from RMB581.3 million, for the year ended December 31, 2021, to RMB456.7 million for the year ended December 31, 2022.
  • R&D expense excluding milestone payments increased by RMB37.8 million, from RMB213.9 million for the year ended December 31, 2021 to RMB251.7 million for the year ended December 31, 2022.
  • CANbridge completed the full technology transfer of these gene therapy products in late 2022.

U.S. Department of State, Bezos Earth Fund, and The Rockefeller Foundation Announce Winrock International as Crediting Body for Energy Transition Accelerator

Retrieved on: 
Tuesday, April 11, 2023
Carbon, ACR, Science, Private sector, Science Based Targets initiative, Rockefeller Foundation, EDF, U.S. Department of Defense Strategy for Operating in Cyberspace, CPI, Climate Policy Initiative, Agriculture, International Energy Agency, Disclosure, Jeff Bezos, HLCG, ART, GHG, GFANZ, Architecture, Accounting, Government, Energy transition, COP, Organization, ETA, United States Department of State, National Integration Council, Retirement, ERT, Environmental Defense Fund, 2013 United Nations Climate Change Conference, Developed country, Investment, Winrock International, Glasgow, United, Awards of the United States Department of State, Environment, Conference, EnergySolutions, Renewable energy

WASHINGTON, April 11, 2023 /PRNewswire/ -- The U.S. Department of State, the Bezos Earth Fund, and The Rockefeller Foundation today announced that Winrock International will develop and administer the carbon crediting standard to be employed by the Energy Transition Accelerator (ETA).

Key Points: 
  • WASHINGTON, April 11, 2023 /PRNewswire/ -- The U.S. Department of State, the Bezos Earth Fund, and The Rockefeller Foundation today announced that Winrock International will develop and administer the carbon crediting standard to be employed by the Energy Transition Accelerator (ETA).
  • The ETA is a joint initiative to catalyze private capital to accelerate the transition from fossil fuel to clean power in developing countries.
  • Annual clean energy investment must triple to $4.2 trillion by 2030 to keep a 1.5°C limit on warming within reach, according to the International Energy Agency.
  • The Department of State, the Bezos Earth Fund, and The Rockefeller Foundation are collaborating for the purpose of developing the ETA as an independent initiative.

YPrime Appoints Jim Corrigan as Chief Executive Officer

Retrieved on: 
Monday, April 3, 2023
Partnership, Clinical data management, GE HealthCare, ERT, Patient, Investment, Growth, Surgery, Shawn, Management

MALVERN, Pa., April 3, 2023 /PRNewswire/ -- YPrime (the "Company"), a global provider of cloud-based eClinical solutions, today announced that Jim Corrigan has joined the Company as CEO. Jim succeeds Shawn Blackburn who has served as CEO of the Company since he founded the business in 2006. As part of this transition, Shawn will continue with the Company leading a new team called YPrime Labs, focusing on future technology solutions that will enhance the patient experience and be integrated into YPrime's solution set.

Key Points: 
  • MALVERN, Pa., April 3, 2023 /PRNewswire/ -- YPrime (the "Company"), a global provider of cloud-based eClinical solutions, today announced that Jim Corrigan has joined the Company as CEO.
  • Jim succeeds Shawn Blackburn who has served as CEO of the Company since he founded the business in 2006.
  • Jim is a seasoned business leader with over 30 years of experience in the technology, life sciences, and healthcare industries.
  • "I have long admired YPrime and the phenomenal job Shawn and his team have done to build a strong platform poised for continued growth," said Jim Corrigan, CEO of YPrime.

Amicus Therapeutics Announces European Commission Approval for Pombiliti™ in Patients with Late-Onset Pompe Disease

Retrieved on: 
Monday, March 27, 2023
Civil service commission, Advanced Therapy Medicinal Product, Glycogen storage disease type II, European Commission, Committee, Miglustat, ERT, Patient, Physician, Committee for Medicinal Products for Human Use, Rare, Amicus Therapeutics, Enzyme replacement therapy, Therapy, EC, PROPEL, Caregiver, CHMP, Pharmaceutical industry, EU, Cipaglucosidase alfa

PHILADELPHIA, March 27, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the European Commission (EC) has granted approval for Pombiliti™ (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The Company has submitted the previously requested analytical testing for miglustat, the enzyme stabilizer component of AT-GAA. The Committee for Medicinal Products for Human Use (CHMP) opinion for miglustat is expected in the second quarter of 2023.

Key Points: 
  • The Committee for Medicinal Products for Human Use (CHMP) opinion for miglustat is expected in the second quarter of 2023.
  • “Late-onset Pompe disease is a rare, neuromuscular disorder that can have devastating consequences for patients and their families.
  • The European Commission approval for Pombiliti is another major step towards bringing this much needed, new treatment for all adults living in the EU with late-onset Pompe disease.
  • The EC approval of Pombiliti follows the positive opinion previously granted by the Committee for Medicinal Products for Human Use (CHMP).

AVROBIO Reports Fourth Quarter and Fiscal Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 23, 2023
Health, Other Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, GMP, Tissue, VCN, University of California, San Diego Performance-Based Skills Assessment, Gaucher's disease, Enteropathy, University, Safety, Health care, Food, CMC, Aes, Clinical trial, MPS II, Skin, Glycogen storage disease, Cerebrospinal fluid, MPS, CNS, IDS, Cystinosis, AVRO, ERT, HSPC, Patient, Physician, Risk, Medicines and Healthcare products Regulatory Agency, Liver, CSF, HSC, UOM, FDA, Syndrome, Enzyme replacement therapy, Lymphadenopathy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Hunter syndrome, GI, Cystine, DSM-IV codes, MHRA, Disease, Pharmaceutical industry, Vaccine, Gaucher

(Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the fourth quarter and year ended Dec. 31, 2022 and provided a business update.

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the fourth quarter and year ended Dec. 31, 2022 and provided a business update.
  • “2022 was a transformative year for AVROBIO, topped in December with a robust clinical and regulatory update for our Gaucher disease program.
  • Safety data to date from GD1 and GD3 patients indicate no adverse events (AEs) related to drug product.
  • No major chemistry, manufacturing and controls (CMC) changes are anticipated for AVROBIO’s plato® gene therapy platform as the company prepares to enter a registrational trial for GD3.

Life Science and Healthcare Industry Executive Adam Deutsch Joins uMotif Board as Company Brings the Next-Generation of eCOA/ePRO to Expanding Global Market

Retrieved on: 
Wednesday, March 15, 2023
Medtech, Research, Political science, Communication, Transformation, London Stock Exchange, IPO, London, FTSE, Biomet, GM, Corporation, Motif, Quality, Rutgers University, Health, General counsel, Convatec, Integration, Critical care, University School, Associate, The Conference Board, JD, Corporate development, Growth, Customer service, New York University School of Law, Clinical trial, Marketing, High, ERT, Management, Deutsch, Clario Tech

BOSTON, March 15, 2023 /PRNewswire-PRWeb/ -- uMotif – one of the clinical trial technology market's fastest growing companies – bolstered its status as the leader in next-generation eCOA/ePRO with the announcement that life science and healthcare executive Adam Deutsch has joined its Board of Directors.

Key Points: 
  • In bringing strong business acumen and a thoughtful approach to transforming companies, Deutsch is focused on how to better serve the research participant.
  • uMotif CEO Steve Rosenberg ( LinkedIn ) said, "Adam has an impeccable track record of guiding the growth of private and public companies in our industry.
  • uMotif Chairman of the Board Steve Powell ( LinkedIn ) said, "we're delighted to welcome Adam Deutsch to the uMotif Board.
  • His expertise in creating healthcare and life science market leaders will greatly benefit uMotif as it pursues its ambitious growth plans.

Amicus Therapeutics Announces Positive Long-Term Data from Phase 3 Open-label Extension Study of AT-GAA in Late-Onset Pompe Disease at the 19th Annual WORLDSymposium™ 2023

Retrieved on: 
Wednesday, February 22, 2023
Biomarker, Vital capacity, Glycogen storage disease type II, Safety, Therapy, Principal, Rare, OLE, Amicus Therapeutics, PROPEL, Myopathy, Extension, Glycogen, Cell, ERT, CK, Patient, Durability, Glycogen storage disease, Department of Neurology, Xuanwu hospital, Serum, FVC, Creatine kinase, Ludwig Maximilian University of Munich, Transport, Pharmaceutical industry, Dietary supplement, Television, MD

PHILADELPHIA, Feb. 22, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced positive results from the global Phase 3 open-label extension (OLE) study (ATB200-07) to investigate the long-term efficacy and safety of AT-GAA in adult patients with late-onset Pompe disease, an inherited lysosomal disorder caused by an enzyme deficiency that leads to the accumulation of glycogen in cells. Study participants treated with AT-GAA for up to 104 weeks showed persistent and durable effects on six-minute walk distance (6MWD), stability in forced vital capacity (FVC), and continued reductions in biomarkers of muscle damage and disease substrate.

Key Points: 
  • These clinical results are being featured at the 19th Annual WORLDSymposium™ 2023 in a poster presentation and an oral platform presentation scheduled for Sunday, February 26, 2023, at 9:00 a.m.
  • The presentation will be given by Benedikt Schoser, MD, Department of Neurology, Ludwig-Maximilians-University of Munich and Principal Investigator in the PROPEL study.
  • Consistent with the Phase 1/2 study and the Phase 3 PROPEL study results, these first long-term results suggest that AT-GAA treatment for up to two years was associated with a durable effect.
  • No new safety signals were identified during the open label extension study.