Foundation Fighting Blindness的动态

Our portfolio company Belief BioMed announced that the Medical Products Administration (NMPA) of China has accepted the company's new drug application (NDA) for BBM-H901 for treatment of adults with hemophilia B. BBM-H901 is the first core product independently developed and manufactured by Belief BioMed; it's also the first gene therapy product indicated for inborn genetic diseases for which an NDA has been submitted in China. By achieving this milestone, BBM-H901 is expected to become the first approved recombinant AAV-based gene therapy product for hemophilia B in China and to reshape the landscape of AAV-based gene therapy worldwide. Professor Lei Zhang from the Hematology Hospital of the Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences) and the Leading PI of BBM-H901 registrational study said, “There are pressing unmet medical needs for hemophilia B patients in China. Currently, most patients are treated with prothrombin complex concentrate (PCC) as a replacement therapy, this treatment has infection risk and at the same time, it’s inconvenient for the patients. Furthermore, due to insufficient or non-standardized treatment, patients with moderate to severe hemophilia B often experience joint bleeding and deformities. With the latest BBM-H901 clinical data showing both good safety and efficacy, we look forward to bringing BBM-H901 to market and the clinical benefits it will bring to patients.” #HemophiliaB #GeneTherapy #BBMH901 #NDA #NMPA #BiotechInnovation #HealthcareAdvancement #AVVGeneTherapy #MedicalBreakthrough #ChinaBiotech #Pharmaceuticals #RareDiseaseTreatment #ClinicalResearch #HealthcareInnovation #QimingPortfolio #QimingHealthcare

Cell and gene therapy is an exciting new area in healthcare that offers a route towards tackling many illnesses and conditions that were previously untreatable. To give an idea of how quickly it’s growing, as of March 2024, the FDA had approved 36 gene therapies for use, with seven of those approvals coming last year. Research firm?Nova One?valued the market at $18.3 billion last year, and it forecasts compound annual growth of 18.3%, to be worth $58.7 billion by the end of this decade. To stay at the forefront of this sector, #Ireland is actively exploring ways to strengthen the ecosystem that helps the existing industry, together with emerging players, to tackle some of the challenges involved with C&GT. You can read more here: https://lnkd.in/eBxxtXYf #InvestInIreland #InvestInExtraordinary #Pharma

?? Exciting News! ?? ? The June Edition of BioProcess International has published a new article featuring Pamela Whalley, Associate Director CMC, on the topic presented at Advanced Therapies in Miami, earlier this year: Partnering for Gene Therapy Manufacturing - What is the Key to Success? ? In this insightful piece, Pam explores the crucial factors that gene therapy developers must consider when selecting a CDMO (Contract Development and Manufacturing Organization) and highlights the importance of demanding a pilot study to ensure success. ? Read the full article here: https://lnkd.in/gynu-mnA ? #GeneTherapy #Biotech #CDMO #Pharmaceuticals #BioProcess #ManufacturingSuccess #HealthcareInnovation #BPI #CMC #complement #conference

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.

The cell and gene therapy field is booming, but making these life-changing treatments accessible remains a challenge. How can we overcome cost barriers and manufacturing hurdles? Cell and gene therapy manufacturing relies heavily on pen and paper, hindering data sharing and slowing down processes. This can be life-threatening for patients who need timely treatment. We need to embrace digital solutions, both inside and outside the cleanroom, to improve efficiency and ensure patient safety. ?? Read Clinical Trials Arena's article for expert insights from Autolomous CEO Alexander Seyf, David Smith - BioCentriq, John W. Hadden II - ViroCell Biologics, and Romain du Hecquet de Rauville - EXO Biologics

The potential of cell and gene therapy is limitless. But to realize its full potential, we need to address key challenges. This Drug Discovery World article explores these challenges and Josh Ludwig highlights how ScaleReady is working to overcome them. Don't miss it! Read the full article: https://lnkd.in/eX-_e_rc #CGT #CellTherapy #Innovation

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> #ADA24: Fractyl pitches potential of GLP-1 gene therapy, but pushes clinical trials back to 2025: A small biotech company is developing a one-time gene therapy that could turn the pancreas into its own GLP-1 drug factory and eliminate the need for frequent injections of medicines like Ozempic or Wegovy. Fractyl Health previously showed that an injection of its treatment, delivered in viral vectors injected directly into the liver, caused obese mice to lose nearly 25% of their body weight in just two weeks. At a scientific conference on Sunday, the company said that mice who got a GLP-1 drug for four weeks maintained their 22% weight loss four weeks later when they switched to the gene therapy. The updated data were presented at the American Diabetes Association’s scientific sessions in Florida. In an interview with Endpoints News ahead of the conference, Fractyl CEO Harith Rajagopalan said that the yo-yo-ing weight loss and gain seen in people who go on and off GLP-1 drugs emphasizes the need for one-and-done alternatives. “The question is shifting from how do you lose weight to how do you keep it off,” Rajagopalan said. “If the effects wear off, you are talking about limited benefit for society. But if you can make those effects permanent, you can imagine what a post-obesity society looks like.” Last year at the diabetes conference, Fractyl announced plans to begin testing its gene therapy in both diabetes and obesity patients in 2024. But that ambitious timeline has slipped — as has the company’s stock, which is down about 70% since its February IPO. Rajagopalan said the company is now planning a clinical trial in patients with diabetes for 2025. The results will inform a future trial testing a slightly different gene therapy design in people with obesity, but Rajagopalan wouldn’t say when that trial might be. Longer-term studies in animals are also underway, he said. While there’s been ongoing interest from drugmakers about how to decrease the frequency of dosing for weight loss drugs and to make their effects last longer, a permanent treatment would likely raise many of its own questions. The drugs have been associated with side effects such as muscle loss, which could become a particular issue as patients age and become more frail, for example. Novo Nordisk recently opened a research center outside of Boston focused on genetic medicines. Its leaders told Endpoints in February that it is working on genetic medicines that could complement its weight loss drugs but haven’t disclosed interest in making a gene therapy version of Wegovy. And Eli Lilly, which is also opening a genetics-focused research hub in Boston, has also remained mum on the prospects for a GLP-1 gene therapy. Rajagopalan says that the company’s endoscopic procedure for shuttling… #lucidquest #genetherapy #celltherapy

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Exsilio Therapeutics raises $82 million for mRNA-based gene therapy, led by Tal Zaks: Exsilio Therapeutics has raised $82 million in hopes of using mRNA to surmount the limitations of current gene therapies. mRNA’s a medium that the biotech’s chairman and interim CEO Tal Zaks is intimately familiar with. Zaks was Moderna’s chief medical officer from 2015 to 2021, leading the company’s development of its Covid-19 vaccine before becoming a partner at the healthcare investment group OrbiMed. Exsilio received an undisclosed amount of seed money in 2022 from OrbiMed. Its $82 million Series A raise, announced Tuesday morning, was co-led by Novartis’ venture arm and Delos Capital, followed by OrbiMed, Insight Partners, JP Morgan’s life science investment group, CRISPR Therapeutics and Innovation Endeavors, among others. “The [gene therapy] field has coined the ‘one-and-done’ idiom. But ‘one-and-done’ is really a bug, it’s not a feature,” Zaks told Endpoints News on Monday ahead of the announcement. Exsilio plans to use an mRNA-based therapy to insert whole genes, which could potentially be applied across a host of diseases caused by genetic mutations. The mRNA would encode a gene of interest and items to facilitate its insertion, including mobile genetic elements, which could be delivered via a fatty acid bubble called a lipid nanoparticle (LNP). The mobile genetic elements are where Exsilio derives its name, which is Latin for leap. Mobile genetic elements include a vast library of DNA segments that include plasmids, transposons and integrons, among others, which can move around the genome. The company’s platform uses both computer modeling and wet lab experiments to discover and engineer the genetic elements used to insert genes. With Exsilio’s technology, a gene would be inserted at a safe harbor site — places in the genome where a gene can be inserted and function, but won’t cause safety risks. Zaks left much of the company’s science in the dark. He declined to elaborate on the mobile genetic elements, noting that the company still has to “get to a point where we’ve got our IP filed and we can actually show some data that we can talk about.” He said that Exsilio has a small team in Israel and is building a team in Boston, but declined to share who the company’s founding scientists or scientific co-founders are. On the Israeli-Palestinian War, he said “These have been clearly difficult times for our team,” but noted the company has maintained “full productivity.” He also declined to name which exact diseases Exsilio is hoping to work on, but said it could go after “any disease that has been a target for a gene therapy.” The hope is that an mRNA therapy would come with fewer toxic side effects — akin to the Covid-19 vaccine. While current gene… #lucidquest #genetherapy #celltherapy

Gene Therapy And Accelerated Approval: US FDA Mulls Pooling Data To Shrink Postmarket Studies ? Who should read this? Clinical trials and compliance execs in pharma, biotech, and CROs. ? What is it? Details of FDA plans to be more flexible in requirements for postmarket studies of cell and gene therapies that receive accelerated approval. ? Why does it matter? This article raises awareness of possible changes that could save companies time and money in their postmarket follow-up studies, with firms encouraged to seek early advice from the agency. https://lnkd.in/eNW7zBCQ Citeline Citeline Clinical #cellgenetherapy

Upcoming regulatory events for pipeline AAV gene therapies, with a focus on neuromuscular and blood disorders, are setting high expectations for the space in 2024. Read the full article below in the current issue of Pharma Technology Focus magazine: #globaldata #pharmaceuticaltechnology #AAV #genetherapies #neuromuscular #pharmaceuticals #pharmanews https://lnkd.in/gWaXJsCu