Latest news with #genetherapy
Yahoo
4 days ago
- Business
- Yahoo
Develop Robust In-Licensing Strategies in the Face of GI CGT Market Changes
Explore the evolving landscape of cell and gene therapies (CGTs) in gastroenterology. Key therapies include Anterogen's Cupistem and Takeda's Alofisel, the latter recently withdrawn by EMA. Understand market trends, develop licensing strategies, and identify opportunities in the CGT segment for GI diseases. Dublin, May 30, 2025 (GLOBE NEWSWIRE) -- The "Cell & Gene Therapies in Dermatology Disorders: Therapeutic Analysis" report has been added to there are only two cell and gene therapies (CGTs) on the market across all gastroenterology (GI) indications. Anterogen's Cupistem, indicated for the treatment of anal fistula in adult patients, was the first adipose tissue-derived mesenchymal stem cell (ASC) asset to receive approval in the GI market in Japan, 2012. This was followed by Takeda Pharmaceutical's Alofisel (darvadstrocel), which received approval from the European Medicines Agency (EMA) in 2018 and from Japan's Pharmaceuticals and Medical Devices Agency (PDMA) in 2021. However, on December 13th, 2024, the EMA announced the withdrawal of Alofisel (darvadstrocel) from the EU market citing lack of data that demonstrated the benefit of this therapy in Crohn's marketed therapies are prescribed when a patient has shown an inadequate response to at least one conventional or biologic to Buy Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Develop business strategies by understanding the trends shaping and driving the CGT in Gastrointestinal market. Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact Gastrointestinal disease targeting CGT therapeutics market in the future. Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors. Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage. Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships. Company Coverage: Krystal Biotech Inc RHEACELL GmbH Tego Science Inc Japan Tissue Engineering Bio Solution International Co Ltd Organogenesis Holdings Inc EHL Bio Co Ltd Castle Creek Biosciences Inc Abeona Therapeutics Inc Kangstem Biotech Co Ltd Key Topics Covered: 1. Preface2. Executive Summary3. Introduction and Scope4. Current Treatment Options4.1. What is Cell & Gene Therapy?4.2. History of the Development of CGT in Dermatology Disorders4.3. Marketed Product Profiles - Cell and Gene Therapy4.4. Challenges and Opportunities in CGT in Dermatology Disorders5. Pricing and Reimbursement Assessment5.1. High Cost of CGTs Requires Adaptation of Payment Models to Ensure Affordability and Patient Access5.2. Price of Cell Therapy6. Regulations6.1. Regulation of CGTs in the 8MM7. Future Market Assessment7.1. Top 20 Dermatology Disorders with CGT Development7.2. Top Five Dermatology Disorders with the Most CGT Pipeline Assets7.3. Top Five Dermatology Disorders Stratified by Molecule Type7.4. CGT in Dermatology Disorders - Pre-reg and Phase II/III7.5. Additional Players Expected to Join the Competition Within Five Years7.6. Industry Trends in the Application of CGTs in Dermatology Disorders7.7. Most of CGT Market Catalyst is Concentrated in 20258. Likelihood of Approval and Phase - Transition Success Rate Analysis8.1. CGT Candidates Have Higher LoA and PTSR vs. Indication Benchmarks9. Sales Forecast9.1. Sales are forecast to reach $1.5 billion by 2030For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900
ABC News
6 days ago
- Health
- ABC News
World-first CRISPR therapy could 'transform' treatment for rare genetic diseases, but key challenges lie ahead
It's been described as a revolutionary technology — and won its inventors a Nobel Prize. CRISPR gene-editing, often simply dubbed CRISPR, is a tool that allows scientists to precisely target and modify the human genome, making it possible to correct mutations and potentially treat genetic causes of disease. Earlier this month, scientists used CRISPR technology to achieve a significant milestone: re-write the DNA of a baby with a rare genetic disease. The patient, a now-10-month-old boy named KJ, is the first person in the world to successfully receive a personalised gene-editing therapy. The landmark case, led by scientists and doctors at the University of Pennsylvania and the Children's Hospital of Philadelphia, was published in the New England Journal of Medicine. Not long after he was born, KJ was diagnosed with a rare, life-threatening genetic disorder called CPS1 deficiency, which affects just one in 1.3 million babies. The disorder is caused by a mutation in a gene that affects a person's ability to properly metabolise protein, and results in toxic levels of ammonia to build up in the body. Unlike other CRISPR treatments, which were designed to be used in multiple people with the same disorder, KJ's therapy was customised to correct his specific disease-causing mutation. "This is a significant advance in our ability to modify human genes," said haematologist and gene therapy researcher John Rasko, who was not involved in the study. While it's too early to know whether the CRISPR treatment will work long-term, researchers say it could provide a blueprint for developing customised gene-editing therapies for others with rare diseases. "While KJ is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient's needs,' said lead researcher Rebecca Ahrens-Nicklas from the Children's Hospital of Philadelphia. The high levels of ammonia caused by KJ's CPS1 deficiency can cause severe damage to the brain and liver and even prove to be fatal. The best available treatment for the condition is a liver transplant, but only about half of babies with CPS1 deficiency live long enough to receive one. Scientists at the University of Pennsylvania had been investigating gene-editing therapies for similar genetic disorders and when KJ was diagnosed, they quickly mobilised to create a treatment to fix his specific mutation. To do this, they used a "genetic engineering trick" called CRISPR base editing, a second-generation CRISPR tool, said Marco Herold, CEO and head of the Blood Cancer and Immunotherapy Lab at the Olivia Newton-John Cancer Research Institute. "The researchers identified through [genome] sequencing that this mutation was the result of a change in DNA bases," Professor Herold, who was not involved in the study, told The Health Report. DNA sequences are made up of four different "letters" which represent different chemical bases. The order of these letters or bases determines the genetic information carried in the DNA. "[CRISPR technology] scans the DNA and runs over all the letters until it encounters the wrong letter — it can be programmed to find this," Professor Herold said. Unlike traditional CRISPR medicines, which bind to the target DNA, cut it, and silence or repair a problematic gene, base editors convert target DNA from one letter into another. "In this case, the letter was an A and it had to be changed into a C … and that leads to the repair," said Professor Herold, whose own research focuses on CRISPR screening and editing. KJ, who had been on a highly restrictive diet since birth, as well as medication to remove ammonia from his blood, was given a small first dose of the novel gene-editing therapy at seven months of age. Over the next two months, he received two more infusions at higher levels. Since the treatment, he's been able to eat a full protein diet and take just half his usual medication — a sign the therapy has, at least partially, reversed his disease. 'While KJ will need to be monitored carefully for the rest of his life, our initial findings are quite promising,' Dr Ahrens-Nicklas said. Professor Rasko, chair of the federal government's advisory committee on gene technology, said the speed at which the drug was developed was "extraordinary". But he stressed that longer follow up was needed to assess its safety and efficacy, and determine whether additional doses would be necessary. "These are very early days," Professor Rasko said. "Everything is looking great but let's wait a year and see what's going on." It's estimated there are more than 5,000 genetic diseases, which, while rare individually, affect hundreds of millions of people worldwide. In Australia, around two million people — or 8 per cent of the population — live with a rare disease, 80 per cent of which have a genetic cause. But the lack of economic incentive for pharmaceutical companies to develop drugs for extremely rare conditions has led to a scarcity of effective treatments, Professor Rasko said. "Of the 5,000 plus rare genetic diseases, we have a treatment that's specific for less than 5 per cent," he said. Peter Marks, who until recently was responsible for overseeing gene-therapy regulation at the US Food and Drug Administration, described KJ's therapy as potentially "transformational" for the treatment of rare genetic diseases. "Although not all rare diseases may be eligible for a gene-editing approach … there could be hundreds to thousands of diseases that could be treated through an approach similar to the one described," he wrote in an editorial for the New England Journal of Medicine. While KJ's treatment was targeted to his specific mutation, Dr Marks said the same technology could be adapted and "customised" to correct other rare genetic mutations, reducing the cost and complexity of developing new drugs. Professor Herold agreed the same approach could be taken to treat other illnesses caused by a single mutation, with only the CRISPR instructions needing to be changed. "But if you have multiple mutations … there are a lot of diseases that are made up of four, five, six different mutations, then it becomes difficult," he said. "We are not there yet, but we're working at this." Despite the promising results seen in KJ's case, there are several key challenges that need to be addressed before personalised gene-editing could be scaled up and expanded. For one, developing treatments that can successfully reach parts of the body other than the liver — where KJ's mutation occurred — is more difficult, and will require further research. "Because the liver is like a big sieve that processes poisons, toxins, and manufactures hormones and other proteins … the lipid nanoparticles [which encase the gene-editing products] get taken up there," Professor Rasko said. Even though KJ's treatment was a "breathtakingly impressive" proof of concept, replicating and adapting it for other patients would still be resource intensive, he added. "Every time we do this, we have to alter the guide DNA and the technology has to change. It has to be quality-controlled, it has to pass some form of regulatory approval … it's not just a one size fits all." A more established therapeutic approach called gene addition therapy, which involves introducing a working copy of a gene (rather than correcting one), had been a "scientific and medical success" but "an economic failure" to date, Professor Rasko said. "Companies that have been valued at billions of dollars have had to walk away because they can't recoup their costs without charging millions of dollars a pop for these genetic therapies," he said. But, he said, the rate of development and innovation in the field of gene editing — which may help to solve some of the challenges — was "awesome". "You just can't keep up." Listen to the full story on Radio National and subscribe to the Health Report podcast for more.
CBS News
26-05-2025
- Health
- CBS News
New Jersey teen pain-free thanks to new sickle cell disease treatment
For the first time in his life, a young New Jersey man is pain-free thanks to a new gene treatment working to cure patients of sickle cell disease. Gerald Quartey, 18, is the first patient in the Garden State to be treated with the groundbreaking Lyfgenia treatment outside of a clinical trial and following FDA approval in 2023 for patients 12 and older. "Most of the time, I would just be inside because I was in pain" All his life, Quartey has suffered from the debilitating side effects of the rare and life-threatening inherited blood disorder. He needed frequent medical care and couldn't play sports. Even the cold triggered painful episodes. "It was really rough. I missed a lot school, a lot of things that just, like, normal kids would be doing," he said. "Most of the time, I would just be inside because I was in pain." It's also been incredibly painful for his mother, Evelyn Quartey, who lost her oldest son, Emmanuel, to sickle cell when he was 7. "It's been rough," she said. "And there was no treatment at that time." "We are essentially curing patients of their disease" Gerald Quartey was treated by Dr. Stacey Rifkin-Zenenberg at Hackensack Meridian Joseph M. Sanzari Children's Hospital. "The gene therapy treatment is a treatment where we are essentially curing patients of their disease," she said. She explains doctors collect a patient's blood stem cells, which are then genetically modified in a lab. The patient undergoes intense chemotherapy before the modified cells are infused back in the body to produce new, healthy red blood cells. "Just to endure just a few weeks of pain for a lifetime free of pain is, it's definitely worth it," Gerald Quartey said. Gerald completed his treatment at the hospital, ringing a bell and celebrating with the medical staff. "It just felt really great to know that that chapter of my life is over," he said. "So happy and so thankful that he's sickle cell-free," Evelyn Quartey said. Now pain-free, Gerald plans to head to Penn State to study nursing and psychiatry, hoping to help others, inspired by those who helped him.
Yahoo
23-05-2025
- Business
- Yahoo
Regenerative Medicine Strategic Market Intelligence Report 2025: From Rare to Common - The Expanding Horizon of Gene Therapies
Explore the transformative potential of regenerative medicine in healthcare, focusing on curing diseases, eliminating cancer, and restoring tissues through innovative gene and cell therapies. Discover key industry leaders, emerging trends, and applications across therapeutic areas. Unlock insights into reshaping medical devices. Dublin, May 23, 2025 (GLOBE NEWSWIRE) -- The "Strategic Intelligence: Regenerative Medicine (2025)" has been added to offering. The report offers insights into the fundamentals of regenerative medicine and its potential impact on the medical device industry. It highlights key innovators and emerging challengers in the field and provides an analysis of trends shaping the future of regenerative medicine. Regenerative medicine stands at the forefront of groundbreaking innovation in healthcare, revolutionizing the approach to curing diseases, eradicating cancer, and revitalizing damaged or aging tissues. Through advanced techniques in gene therapy, cell therapy, and tissue repair, this field aims to restore biological functions by correcting genetic defects, rebalancing cellular environments, and replacing compromised tissues. Core to this medical revolution is its capacity to reshape the landscape of the medical devices industry. By providing novel solutions and therapeutic strategies, regenerative medicine enhances patient care and outcomes, underscoring its vital role in the future of medicine. The integration of regenerative therapies not only addresses immediate medical challenges but also promises long-term healthcare stability by reducing the burden of chronic diseases and improving quality of life for patients worldwide. Regenerative medicine leaders are pioneering efforts to bring these therapies to clinical practice, concentrating on strategic expansions into more prevalent disease areas. Stakeholders investing in this dynamic theme anticipate a profound impact on healthcare delivery, setting new standards for therapeutic efficacy and patient well-being. As the field of regenerative medicine continues to evolve, it holds the promise of unprecedented advancements, fostering a healthcare environment where diseases are not merely treated but cured, and where age-related degenerations are effectively reversed or mitigated. In conclusion, the growth of regenerative medicine is a key driver of transformation within the medical industry, offering opportunities that extend far beyond current healthcare capabilities. Engage with this innovative field to remain at the cutting edge of medical technology and patient care solutions. Scope of Regenerative Medicine Regenerative medicine offers transformative potential across healthcare sectors. Its diverse applications extend into various therapeutic areas. Gene therapies primarily target rare diseases, with future progression into common and polygenic conditions. Key Topics Covered: Executive Summary Players Technology Briefing Trends Industry Analysis Value Chain Companies Sector Scorecard Glossary Further Reading Thematic Research Methodology About the Analyst Contact the Publisher For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900
Associated Press
19-05-2025
- Health
- Associated Press
PMC's FDA Report Shows Continued Progress for Personalized Medicine
Washington, D.C., May 19, 2025 (GLOBE NEWSWIRE) -- The Personalized Medicine Coalition (PMC) has released a new report documenting the U.S. Food and Drug Administration's approval last year of 18 new personalized medicines. The agency also approved six new gene and cell-based therapies for rare genetic diseases and some cancers and approved or cleared new or expanded indications for 11 diagnostic testing systems. 'The new personalized medicine approvals and policy developments at the FDA in 2024 helps demonstrate that scientific innovation continues to move the health system away from one-size-fits-all, trial-and-error medicine, toward the utilization of molecular information to improve patient outcomes and make clinical care more efficient,' says PMC President Daryl Pritchard. The annual report, Personalized Medicine at FDA: The Scope & Significance of Progress in 2024, also highlighted several regulatory developments in the field of personalized medicine. Other 2024 achievements include: • expanded indications for previously authorized personalized therapies, including the first ever for an approved gene therapy treatment • safety label changes for certain cancer treatments based on pharmacogenomic (PGx)-based biomarker testing • release of a draft guidance document on Diversity Action Plans to improve enrollment of participants from underrepresented populations in clinical studies • establishment of the Center for Drug Evaluation and Research (CDER) Artificial Intelligence (AI) Council to provide oversight, coordination and consolidation of CDER activities around AI use • creation of the CDER Center for Real-World Evidence Innovation to coordinate, advance and promote the use of real-world evidence in regulatory decision-making • establishment of the Rare Disease Innovation Hub to enhance collaboration across the FDA for rare disease product development 'The newly approved products, and these important regulatory milestones, will help innovators and clinicians provide safer and more efficacious treatments and prevention regimens based on the principles of patient-centered care,' the report reads. The scope of breakthroughs underscored the acceleration of the personalized medicine field. The 2024 approval of 18 personalized medicines – meaning the label references specific biological markers – represent approximately 38 percent of all newly approved therapeutic molecular entities. Treatment areas include: cancer, Alzheimer's disease, cystic fibrosis, Niemann-Pick disease and alopecia. Personalized medicines now account for at least a quarter of drug approvals for each of the last 10 years. That's a notable increase from a decade ago when such treatments made up less than 10 percent of new therapies that were approved. 'We can't take progress for granted, however,' says Pritchard. 'The FDA should continue to promote policies and practices that push forward the field so we can realize the true potential of personalized medicine.' ### About the Personalized Medicine Coalition: The Personalized Medicine Coalition is a nonprofit education and advocacy organization comprised of more than 200 institutions from across the health care spectrum, including innovators, scientists, patients, providers and payers. PMC works closely with business executives, clinicians, lawmakers and government agency officials to promote the understanding and adoption of personalized medicine concepts, services and products. For more information, please visit Kayla Brown Personalized Medicine Coalition [email protected]
