Latest news with #rarediseases
Yahoo
22-07-2025
- Health
- Yahoo
Analysis Group Co-Authors Examine the Advancements in and Application of Health Economics and Outcomes Research to the Evaluation of Cell and Gene Therapies for Rare Diseases
BOSTON, July 22, 2025 /PRNewswire/ -- In a new article published in Value in Health, researchers from Analysis Group, a global leader in health economics and outcomes research (HEOR), collaborated with Novartis to examine how HEOR is transforming the evaluation and access pathways for cell and gene therapies (CGTs) for rare diseases. As CGTs move from experimental to increasingly approved treatments for rare diseases, health systems face urgent questions about how to evaluate, reimburse, and scale their access equitably. In their article, the authors spotlight five key HEOR innovation areas that are enhancing the generation of real-world evidence (RWE), supporting novel endpoints, and shaping future models of affordability and access. Because of small patient populations, heterogeneous disease presentations, a lack of effective alternative treatments, and hence, ethical concerns, gold standard randomized double-blind controlled clinical trials are often not suitable for establishing efficacy and safety for CGT treatments. Consequently, traditional economic models are not able to capture the holistic value of CGTs, given that the true benefits associated with such treatments are subject to a much longer time outside of the trial periods. For example, for patients with rare hereditary genetic disorders, timely access to CGTs can yield real differences in meaningful gains in life years. To address these challenges, researchers are increasingly relying on innovations in HEOR to inform proper clinical and economic evaluation of CGTs. An Analysis Group team led by Managing Principal Min Yang, Manager Su Zhang, and affiliate Lou Garrison collaborated with researchers from Novartis, led by Dr. Walter Toro, to examine the advancements in the evaluation of CGTs made possible by HEOR innovations, including the design and collection of long-term real-world data (RWD) for CGT efficacy extrapolation, justification for use of novel endpoints, integration of patient voices and preferences, and application of sophisticated statistical methodology, sometimes augmented with artificial intelligence (AI) or natural language processors. These advances are reflected in regulators' and health technology assessment (HTA) agencies' growing openness to the acceptance of RWE and patient-centered endpoints in their evaluations, marking a shift from traditional standards that may not suit CGTs. The authors note that emerging value assessment frameworks and novel reimbursement models offer solutions and adaptability to decision makers with enhanced ability to manage health equity and affordability.
Yahoo
21-07-2025
- Business
- Yahoo
Amicus Therapeutics to Announce Second Quarter 2025 Financial Results on July 31, 2025
PRINCETON, N.J., July 21, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the Company will host a conference call and live audio webcast on Thursday, July 31, 2025, at 8:30 a.m. ET to discuss financial results for the second quarter ended June 30, 2025. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN to access the event. A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at Web participants are encouraged to register on the website 15 minutes prior to the start of the call. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event. About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company's website at and follow on X and LinkedIn. CONTACT: Investors:Amicus TherapeuticsAndrew FaughnanVice President, Investor Relationsafaughnan@ (609) 662-3809 Media:Amicus TherapeuticsDiana MooreVice President, Corporate Communications dmoore@ (609) 662-5079 FOLD–GError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Associated Press
16-07-2025
- Business
- Associated Press
Quetzal Therapeutics Debuts with Expert Leadership, High-Impact Pipeline and $50 Million of Committed Capital
CHICAGO, July 16, 2025 (GLOBE NEWSWIRE) -- Quetzal Therapeutics, a newly formed biopharmaceutical company, announced its official launch today with $50 million of committed capital. Founded to address urgent unmet needs in rare and life-threatening diseases, Quetzal brings together a team of seasoned executives, scientific pioneers, and biotech & pharmaceutical veterans committed to delivering transformative therapies to patients around the world. At the core of Quetzal's mission is a belief that cutting-edge science, guided by proven leadership, can redefine what's possible in treatment for conditions like Acute Promyelocytic Leukemia ('APL') and invasive infections. The company has formed around a multi-asset pipeline, initially including: Built on Leadership Excellence Quetzal is led by an executive team with deep expertise in drug development, clinical strategy, and operational execution. 'Our team was brought together by a shared conviction: that patients with rare diseases deserve the same urgency and innovation as those with more common conditions,' said Usman Ahmed, CEO. 'Quetzal Therapeutics is built to deliver on that promise with the right science, the right strategy, and the right people.' Usman Ahmed, CEO & Chairman – A proven leader with over two decades of leadership in pharmaceuticals and start-up innovation. During his tenure at Nexus Pharmaceuticals, Usman led the execution of complex transactions, spearheaded operational improvements, and championed cost efficiencies in the highly specialized and challenging field of specialty and generic injectables. Dr. Sridhar Desikan, Co-Founder & Chief Scientific Officer – An industry veteran with 28 years of experience in NCE and generics development. Sridhar began his career at Novartis Pharmaceuticals and subsequently worked at DuPont Pharma, Bristol-Myers Squibb, Dr. Reddy's, and most recently at Nexus Pharmaceuticals as Chief Scientific Officer. Sridhar holds a Ph.D. in Chemical Engineering and has accreditations from Harvard Medical School's HMX Pro program. Sara Mathews, Vice President Program Leadership – With nearly 15 years of experience in the biotech industry, Sara is a seasoned asset leader with a proven track record of bringing cross-functional teams together and leading novel oncology therapeutics through global clinical programs from IND filing through late-stage development, filing preparation, and commercial launch planning. Christina Liwski, General Counsel & CHRO – With over a decade of diverse and sophisticated legal experience, Christina brings deep legal acumen to the team. Christina's experience spans multiple practice areas, including transactions and litigation. She most recently served as Assistant General Counsel at Nexus Pharmaceuticals, LLC and is admitted to practice in Illinois, Massachusetts, and the United States District Court for the District of Massachusetts. Guided by an Accomplished Board of Directors The company's Board of Directors features industry leaders with a track record of building, funding, and guiding successful companies: Aman Ahmed – With nearly a decade of experience in the pharmaceutical industry, Aman brings deep expertise in sales operations, DEA and FDA regulatory compliance, international sales, and therapeutic portfolio management. Dr. Jonathan Cheng – Dr. Jonathan Cheng is a physician-scientist and oncology drug development expert with a distinguished career across both industry and academia. As Chief Medical Officer at Systimmune, he leads the clinical development of a diverse pipeline of novel therapeutics, including innovative antibody-drug conjugates (ADCs) and the company's first-in-class EGFR-HER3 bispecific ADC currently in Phase 3 trials. Todd Chermak, Ph.D – Todd Chermak is a life sciences executive with 30 years of experience spanning the biopharmaceutical, diagnostics, and nutrition industries. He currently serves as Senior Vice President and Global Business Head of Immunology and Proteomics at CellCarta. Mike Warmuth – Mike Warmuth is currently working on various Boards: for profit, academic, and not-for-profit. From 2018 to 2023 Mr. Warmuth worked for EW Healthcare as an Operating Partner in their New York office. Prior to EW, Mr. Warmuth spent 30 years at Abbott in various senior executive capacities, where he led multiple divisions and business units. Strategic Talent Acquisition Partnership Quetzal Therapeutics proudly acknowledges the role of Heidrick & Struggles, a premier global leadership advisory firm, in identifying and recruiting Dr. Jonathan Cheng to the company's Board of Directors. Their expertise and commitment to excellence were instrumental in securing a highly respected thought leader whose insight will help shape Quetzal's long-term scientific and clinical strategy. For more information, please visit: Media Contact: [email protected]
Bloomberg
11-07-2025
- Health
- Bloomberg
Making Personalized Crispr Cures to Save More Babies
Hi, it's Gerry in New York. Researchers are trying to build on the success of 'baby KJ' and make more personalized treatments for kids with rare diseases. More on that in a moment, but first ... In May, baby KJ Muldoon made history as the first person to get a custom-made treatment using the gene-editing tool Crispr. Now a push is underway to do the same thing for eight more kids.
Yahoo
08-07-2025
- Business
- Yahoo
Hansa Biopharma appoints Dr Richard Philipson as Chief Medical Officer
LUND, Sweden, July 8, 2025 /PRNewswire/ -- Hansa Biopharma AB, "Hansa" (NASDAQ Stockholm: HNSA) today announced that Dr. Richard Philipson has been appointed Chief Medical Officer (CMO) effective 14 July. Dr Philipson will report to CEO Renée Aguiar-Lucander and be a member of the Executive Committee. Dr Philipson has over 25 years of industry experience and a successful track record in drug development, providing clinical leadership resulting in four product approvals, including in rare disease and gene therapy, and comes with expertise and success in building high-functioning teams, building pipelines and executing clinical development programs across all phases of development. He also brings in-depth knowledge of regulatory strategy in drug development. Dr Philipson most recently was CMO of Calliditas Therapeutics and previously spent 16 years at GlaxoSmithKline (GSK), including four years as Therapeutic Area Head in the Rare Diseases Unit. He also has experience from Takeda, and a 4-year period as CMO at Trizell. "We are very pleased to welcome Dr Philipson to the executive management team as CMO. He brings invaluable experience in the area of drug development, from first-in-human to Phase 3, and post-approval studies; with expertise across rare diseases, inflammation and oncology, which will be critical as we prepare for the next phase of our development," said CEO Renée Aguiar-Lucander. "I am excited to have the opportunity to join Hansa at this stage of the company's development. I look forward to working with the Hansa team and leveraging my experience from prior roles to support upcoming regulatory submissions and help build a strategic development plan for future indications," said Dr Philipson. Contacts for more information:Evan Ballantyne, Chief Financial OfficerIR@ Kenney, VP Global Corporate Affairsmedia@ Notes to editors About Hansa Biopharma Hansa Biopharma AB is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. The company has a rich and expanding research and development program based on its proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in autoimmune diseases, gene therapy and transplantation. The company's portfolio includes imlifidase, a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients and HNSA-5487, a next-generation IgG cleaving molecule with redosing potential. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at and follow us on LinkedIn. ©2025 Hansa Biopharma AB. Hansa Biopharma, the beacon logo, IDEFIRIX, and IDEFIRIX flower logo are trademarks of Hansa Biopharma AB, Lund, Sweden. All rights reserved. This information was brought to you by Cision The following files are available for download: 20250708-CMO_ENG_FINAL View original content:
