Croda Pharma ’s Post

The phase 1/2 clinical trial with OCU400 gene therapy showed that >50% of patients with RHO mutation had an improvement level of 2 or more Lux after one year of treatment and is moving forward to the phase 3 study. OCU400 is also tested for RP due to mutations in NR2E3 and CEP 290. It is an emerging modifying gene therapy, not gene dependent, which provides copies of the NR2E3 gene to improve the regulation of multiple functions in the retina, including: maintenance and development of photoreceptors, metabolism, phototransduction, inflammation and cell survival. Ocugen is also testing other modifying gene therapies including OCU410 for dry AMD and OCU420ST for Stargard’s disease.

New Cell, Gene Therapy Hub In Lake Success Could Prove A 'Turning Point' For Cancer Treatment - Daily Voice >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #competitivemarketing #biotech #pharmaceutical #healthcare

In this blog, Form Bio focuses on critical quality attributes (CQAs) for adeno-associated virus (AAV)-based therapies and how we’re helping gene therapy developers assess them with innovative and proven technologies: - Prioritizing AAV Analytical Methods Early in R&D - Assessing Critical Quality Attributes for AAV Gene Therapies - How Form Bio is shaping the Future of AAV Gene Therapy Product Development Short and informative read. Want to get a sneak peek of what our viral construct comparison report looks like? Click through to the blog and request it at the bottom.

🌟 Visgenx Extends Collaboration with Charles River Laboratories for Gene Therapy Advancement Exciting developments are underway in the realm of gene therapies, particularly in the fight against degenerative retinal diseases. Visgenx, a pioneering biotechnology company, has announced a significant extension of its collaboration with Charles River Laboratories, a leading contract development and manufacturing organization (CDMO). The focus of this collaboration is VGX-0111, a promising gene therapy designed by Visgenx to address dry age-related macular degeneration (AMD). Gene therapy manufacturing is a nuanced process, where precision and quality are paramount. William Pedranti, #CEO of Visgenx, expresses confidence in Charles River's capabilities, highlighting their crucial role in manufacturing VGX-0111. With a commitment to advancing treatments for those affected by dry-AMD, this collaboration signifies a strategic step forward. The collaboration gains momentum following Visgenx's successful Type B pre-IND meeting with the #FDA in the summer of 2023. Charles River's role will involve manufacturing materials for VGX-0111, with a specific objective: initiating a clinical proof-of-concept in 2025. The materials produced will support a good laboratory practice (GLP) pre-clinical toxicology study, building on a prior collaboration involving adeno-associated virus (AAV) production. Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions at Charles River Laboratories, expresses enthusiasm about expanding the partnership with Visgenx. The collaboration leverages Charles River's premier gene therapy CDMO capabilities, showcasing their expertise in advancing VGX-0111 closer to clinical trials. As Visgenx and Charles River Laboratories unite their strengths, the gene therapy landscape for degenerative retinal diseases takes a significant stride forward. This collaboration underscores the shared commitment to delivering innovative solutions and brings hope to those grappling with the impact of dry age-related macular degeneration. #GeneTherapy #Biotechnology #InnovationInHealthcare 🧬💡

New Cell, Gene Therapy Hub In Lake Success Could Prove A 'Turning Point' For Cancer Treatment - Daily Voice >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #pharmaceutical #pharma #competitivemarketing #biotech

On January 24, 2024, I shall have the opportunity to attend the event ‘The State of Cell and Gene Therapy 2024’. This event represents a pivotal moment for updating on the biopharmaceutical realities that are revolutionising the landscape of disease treatment. Indeed, some of these are developing potentially curative therapies, leveraging the rapid and significant advances in the field of gene and cell therapy. During the event, a panel of experts from the industrial and academic world will convene to discuss the latest research discoveries, pioneering innovations, and game-changing technologies. These are not only influencing patients’ lives today, but are also predicted to elevate cell and gene therapies to unprecedented levels in the future. #CellTherapy #GeneTherapy #GEN2024 #Biopharmaceutical #MedicalInnovation #AdvancedResearch #DiseaseTreatment

Interesting read about how some Biotech stocks are experiencing a revival, benefiting some cell and gene therapy companies, but not all. The ability to generate revenue for approved cell and gene therapies is challenging due to many factors including regulatory hurdles, manufacturing challenges, limited eligible patients, and coverage barriers from payers. Enjoy the read...

What if we could treat the underlying genetic causes of devastating inherited diseases, rather than just managing their symptoms? This is the potential promise of gene therapies for primary mitochondrial diseases (PMDs). Kait Rouillard, PhD and Rachel Ward review recent developments in gene therapy for PMDs and provide insights into the regulatory benefits of developing these treatments and the incentives for potential pharmaceutical sponsors. https://lnkd.in/eP5tdXkY Next week we will link to the conclusion of this two-part series, where we further explore the opportunities for using regulatory strategy to assist with gene therapy development for PMDs. At Kymanox, Our expertise spans preclinical testing, manufacturing, development, and regulatory submissions for these transformative treatments. Let's collaborate to advance safe, effective gene therapy solutions to address the unmet needs of PMD patients and families...because patients deserve better. Reach out to connect with our team: https://lnkd.in/gy2UWAEj #cellandgene #lifescience #leadership #regulatory

Verve Therapeutics has paused its gene editing study for heart disease due to unexpected lab abnormalities in a participant. This development underlines the challenges of pioneering medical treatments: - The side effect led Verve to prioritize a backup therapy, aiming for a safer delivery method. - This pivot emphasizes the delicate balance between breakthrough innovation and patient safety. Key Considerations - 🔹 How do we navigate the risks and rewards of new gene therapies? 🔹 What implications does this have for the future of heart disease treatment and gene editing technologies? #GeneTherapy #HealthcareInnovation #PatientSafety

My latest piece for Citeline Regulatory - an interview with Orchard Therapeutics' SVP for market access, Francis Pang, in which he talks all thing #HTA, with a focus on Orchard's ex-vivo gene therapy #Libmeldy. It was really interesting to hear about the various ways in which gene therapies and their unique qualities have pushed reimbursement bodies and industry to implement new value assessment and data analysis methods. Most importantly, Orchard has succeeded in negotiating market access deals in a growing number of European countries, hopefully helping to pave the way for future gene therapies that will undergo HTAs. At the moment only a handful of companies have brought cell and gene therapy products to market, but plenty more have candidates in the pipeline which will require HTA bodies to be flexible without compromising on value and quality principles. #atmps #genetherapy #hta #exvivo #rarediseases