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- Immunocore Unveils Promising HIV Functional Cure Data at CROI 2025
Promising early data at CROI 2025 shows that HIV functional cure candidate, IMC-M113V, is well-tolerated and may offer prolonged viral suppression without the need for lifelong antiretroviral therapy. < Back Immunocore Unveils Promising HIV Functional Cure Data at CROI 2025 Promising early data at CROI 2025 shows that HIV functional cure candidate, IMC-M113V, is well-tolerated and may offer prolonged viral suppression without the need for lifelong antiretroviral therapy. Immunocore is a leading biotechnology company focused on developing cutting-edge therapies that harness the power of the immune system to treat a variety of cancers and infectious diseases. The company’s proprietary ImmTAC platform enables the development of novel immunotherapies that can target and destroy diseased cells with precision. Immunocore’s portfolio includes therapies for both oncology and infectious diseases, with a strong commitment to transforming the treatment landscape for patients worldwide. Immunocore Holdings plc (IMCR), a leader in immuno-oncology, has revealed groundbreaking early-stage data from its Phase 1/2 STRIVE trial of IMC-M113V, a novel candidate aimed at providing a functional cure for HIV. The data were presented in an oral session at the 2025 Conference on Retroviruses and Opportunistic Infections (CROI). The multiple ascending dose (MAD) phase of the trial showed promising results, indicating that IMC-M113V is well-tolerated and capable of inducing dose-dependent viral control in HIV patients. These findings come as a major step forward in the ongoing quest to develop a treatment that could eliminate the need for lifelong antiretroviral therapy (ART). Notably, some patients demonstrated viral suppression lasting for up to 12 weeks after ART interruption, providing early evidence of the potential for long-term control. IMC-M113V, Immunocore’s most advanced HIV candidate, targets the virus in a unique manner, leveraging the company’s proprietary ImmTAC technology to activate the immune system’s T cells to target and destroy HIV-infected cells. The STRIVE trial is designed to evaluate the safety, tolerability, and efficacy of IMC-M113V at escalating doses, and these initial findings mark an important milestone in the development of a functional cure for HIV. “We are excited to share the early results from the STRIVE trial, which represent an important step toward potentially transforming the treatment landscape for people living with HIV,” said Dr. Anna Taylor, Chief Medical Officer at Immunocore. “While these data are still in the early stages, the ability of IMC-M113V to provide prolonged viral suppression without the need for ART is encouraging, and we look forward to continuing to explore its potential in future trial stages.” Immunocore emphasized that while the data is still in its early phase, the results suggest the potential of IMC-M113V to be a game-changer in the fight against HIV, offering hope for a functional cure that could reduce or eliminate the dependency on daily ART regimens. The company is continuing to test higher doses of IMC-M113V in the ongoing trial, with further data expected in the coming months. Researchers and clinicians alike are watching closely to see how these findings progress, as the development of a functional cure for HIV remains one of the most sought-after goals in the field of infectious disease. Author BioFocus Newsroom Previous Next
- Advancing the Future of ADCs through Machine Learning: Lonza’s Synaffix and BigHat Biosciences Collaboration
The recent collaboration between Lonza’s Synaffix and BigHat Biosciences exemplifies the growing convergence of biotechnology with machine learning to produce next-generation cancer therapies. < Back Advancing the Future of ADCs through Machine Learning: Lonza’s Synaffix and BigHat Biosciences Collaboration The recent collaboration between Lonza’s Synaffix and BigHat Biosciences exemplifies the growing convergence of biotechnology with machine learning to produce next-generation cancer therapies. Antibody-drug conjugates (ADCs) are at the forefront of oncology treatment, offering a targeted therapeutic approach that delivers potent drugs directly to cancer cells, thereby minimizing systemic toxicity. This partnership combines BigHat’s ML (machine learning)-based antibody design platform, Milliner™ , with Synaffix’s proprietary ADC technology to develop a highly differentiated, effective, and safe ADC pipeline. Synaffix and BigHat Biosciences: Pioneers in ADC Innovation ADCs are designed to selectively deliver cytotoxic agents to cancer cells by harnessing the specificity of antibodies. However, achieving both selectivity and efficacy remains a challenge due to the inherent complexity of the antibody-drug conjugate structure. This is where Synaffix’s ADC technology platform becomes instrumental. Comprised of GlycoConnect™ , HydraSpace® , and toxSYN® technologies, the Synaffix platform provides a comprehensive toolkit that optimizes ADC drug-like properties and enhances efficacy and tolerability. Lonza's acquisition of Synaffix in June 2023 further strengthens this offering, creating an integrated, end-to-end service that accelerates the path from DNA to Investigational New Drug (IND) application. Synaffix's GlycoConnect™ technology provides site-specific payload attachment, utilizing antibody glycan structures to increase stability. This approach, alongside HydraSpace®, which includes a polar spacer to enhance therapeutic index, and toxSYN®, which features a suite of potent cytotoxic agents, positions Synaffix as a leading player in ADC development. These technologies collectively aim to maximize therapeutic index—a crucial parameter in ADC efficacy, denoting the balance between therapeutic effect and side effect profile.\ The Role of Machine Learning in ADC Development BigHat Biosciences brings a powerful AI/ML-driven platform, Milliner™ , to this collaboration, with the potential to redefine ADC design and production speed. Traditional antibody engineering processes can be time-consuming and resource-intensive, requiring extensive laboratory work to develop antibodies with optimal biophysical properties. Milliner™ accelerates this process by integrating synthetic biology with advanced ML models, allowing rapid iteration and optimization of antibody candidates. This platform streamlines antibody design, enabling the development of antibodies with complex functions tailored to withstand the demands of drug conjugation without compromising therapeutic efficacy. Through this collaboration, Synaffix’s GlycoConnect™ technology benefits from BigHat’s precisely engineered antibodies, optimized for stable payload attachment and high binding affinity to target antigens. This union of ML and ADC technology not only facilitates the rapid progression of BigHat’s ADCs but also enhances their therapeutic potential by creating more robust, stable, and effective antibody-drug conjugates. Toward a Fully Integrated ADC Development Pathway The collaboration enables BigHat to leverage Lonza's full suite of ADC services, from antibody production to clinical trial supply. This integration allows BigHat to develop ADC candidates efficiently, reducing the timelines traditionally required for IND-enabling studies. The pathway, facilitated by Lonza’s state-of-the-art facilities, includes bioconjugation, drug product filling, and clinical batch production, all under a single quality system. This seamless, vertically integrated approach underscores Lonza’s commitment to accelerating ADC pipelines and helping biotech partners bring transformative therapies to market more swiftly. The demand for ADCs in the oncology sector continues to rise, spurred by the pressing need for safer, targeted treatments. However, manufacturing and development bottlenecks remain, as the complexity of ADCs requires specialized knowledge and infrastructure at every step of production. The integration offered by Lonza through Synaffix’s platform is a significant advantage, providing companies like BigHat with a streamlined path from initial design to clinical readiness. With Lonza’s recent facility expansion to handle high-potency vial filling, the infrastructure is in place to meet increasing market demands while maintaining the highest quality standards. Potential Impact of the Collaboration on ADC Therapeutics This partnership between Synaffix and BigHat Biosciences has implications far beyond individual drug candidates, potentially influencing the future of ADCs across the oncology landscape. By combining Synaffix’s established technology with BigHat’s AI-driven antibody discovery, the collaboration can produce ADCs that deliver drugs more precisely to tumor cells, thus enhancing both safety and efficacy. This approach directly addresses the unmet medical needs in difficult-to-treat cancers, where traditional therapies often fail due to limited selectivity and high toxicity. With Milliner™ , BigHat can continuously refine and optimize antibodies, leveraging ML to minimize the attrition rate of ADC candidates during preclinical and clinical stages. As ADCs are notoriously challenging to develop, with only a limited number of approved ADCs on the market, such advances in antibody design can lead to higher success rates, faster development timelines, and more accessible treatments for patients. Conclusion The collaboration between Lonza’s Synaffix and BigHat Biosciences is a notable advancement in the ADC field, showcasing how the synergy of machine learning and cutting-edge bioconjugation technology can propel next-generation oncology therapies. Synaffix’s platform offers a streamlined solution for developing ADCs with high stability, selectivity, and safety, while BigHat’s ML-powered Milliner™ platform provides a faster, more effective pathway to antibody design and optimization. This partnership not only strengthens each company’s competitive edge but also highlights the transformative potential of integrating biotechnology with artificial intelligence in cancer treatment innovation. As ADC technology continues to evolve, collaborations such as this set the stage for a future where AI-enhanced biologics bring safer, more effective therapies to patients worldwide. Author BioFocus Newsroom Previous Next
- Verdiva Bio Launches with $411M Funding to Revolutionize Obesity and Cardiometabolic Care
Biotech Startup Unveils Next-Generation Therapies, Including a Promising Once-Weekly Oral GLP-1 Peptide, Backed by Leading Global Investors. < Back Verdiva Bio Launches with $411M Funding to Revolutionize Obesity and Cardiometabolic Care Biotech Startup Unveils Next-Generation Therapies, Including a Promising Once-Weekly Oral GLP-1 Peptide, Backed by Leading Global Investors. Verdiva Bio , a newly established clinical-stage biopharmaceutical company, has launched with an impressive $411 million in Series A financing. The funding round was co-led by Forbion and General Atlantic, with additional participation from RA Capital Management, OrbiMed, Logos Capital, Lilly Asia Ventures, and LYFE Capital. The company is dedicated to developing innovative therapies targeting obesity, cardiometabolic disorders, and related complications. Its pipeline includes both oral and injectable treatments with the potential to be first-in-class or best-in-class. Notably, Verdiva's lead asset is a once-weekly oral GLP-1 peptide, VRB-101, which has demonstrated compelling efficacy potential in a Phase 1 study, confirming the viability of once-weekly dosing. Khurem Farooq, formerly the CEO of Aiolos Bio and Gyroscope Therapeutics, will lead Verdiva Bio as Chief Executive Officer. He is joined by a team of experienced drug developers and biotech professionals, including Chief Scientific Officer Jane Hughes and Chief Medical Officer Dr. Mohamed Eid. "People living with obesity and its complications deserve better options at each stage of their treatment journey," said Farooq. "We created Verdiva Bio to accelerate the development of differentiated medicines that address these significant unmet medical needs." Verdiva Bio's proprietary oral delivery technology is designed to enable patient-friendly dosing, greater scalability, and broader patient access. The company's portfolio also includes multiple amylin molecules (oral and subcutaneous agonists) and other undisclosed programs aimed at enhancing efficacy, improving tolerability, and promoting healthier weight loss. With this substantial financial backing and a robust pipeline, Verdiva Bio is poised to make significant strides in the treatment of obesity and cardiometabolic disorders, offering new hope to millions affected by these conditions worldwide. Author BioFocus Newsroom Previous Next
- International Brain Health Conference 2026 | BioFocus
< Back 18th – 19th May, 2026 Edinburgh, UK International Brain Health Conference 2026 Inaugural international conference bringing together global leaders in neuroscience, clinical research, public health, and policy, with a central focus on protecting brain health before dementia develops. The inaugural International Brain Health Conference 2026 (IBHC 2026), hosted by Scottish Brain Sciences , will bring together researchers, clinicians, life sciences and pharmaceutical partners, policymakers and innovators to examine how advances in early detection and personalised medicine can be translated into practice. The conference will be held at the Sheraton Grand Hotel. Professor Craig Ritchie, CEO and Founder of Scottish Brain Sciences and Professor of Brain Health and Neurodegenerative Medicine at the University of St Andrews, said the field has shifted decisively towards identifying risk and disease processes well before dementia becomes clinically apparent. He emphasised that while the science now allows earlier identification, the more difficult task lies in embedding this knowledge within healthcare systems, public policy and routine clinical care, a gap the conference is intended to address. International faculty and cross-sector programme IBHC 2026 will feature an international faculty including Professor Jeff Cummings (University of Nevada), Professor Vanessa Raymont (University of Oxford), Professor Allan Young (Imperial College London), and Dr Francesca Farina (University of Chicago). The programme will span: Early detection, including imaging and fluid biomarkers Women’s brain health, menopause and sex-specific risk Brain health services and health economics Nutrition, sleep and mental health Comorbidities and infectious disease Global and population-level prevention strategies Professor Jeffrey Cummings will open the meeting with a keynote on therapeutic development. He noted that treatments for Alzheimer’s disease are progressing at pace, creating new opportunities to intervene in the earliest phases of illness. At the same time, he stressed the importance of maintaining momentum in prevention research, arguing that innovation in both treatment and prevention will be central to improving long-term brain health outcomes. A growing imperative for early intervention With dementia prevalence rising worldwide and health systems under sustained pressure, attention is increasingly turning to modifiable risk factors and earlier-stage intervention. IBHC 2026 aims to provide a forum for rigorous scientific exchange and collaboration across sectors at a point when the translation of research into service delivery is becoming ever more urgent. Early Bird registration, offering a 50% discount, is available until 31 March 2026. Further details and programme updates are available from Scottish Brain Sciences . Previous Register now Next
- ThermoFisher Gibco Cell Therapy Speed Form | BioFocus
Streamline your workflow to set the pace. Accelerating cell therapy development and manufacturing. In a landscape of rapid market growth with a high pace of innovation, you need to keep up to realize your therapy’s full potential. Speed is therefore one of the most pressing challenges for cell therapy development and manufacturing. Discover how to streamline your workflow across cell isolation, activation, and expansion to stay ahead and help accelerate your treatments to patients. Learn more on how you can achieve your cell therapy quality goals and drive your performance forward, download now. First name Last name Country Email Institution City Would you like to have a Thermo Fisher Scientific bioprocessing specialist contact you for a quote, demo, sample, or to provide technical support? Choose an option I acknowledge and agree to the use of my contact information to receive messages about offerings by BioFocus, its brands, affiliates and/or third-party partners, consistent with the BioFocus Privacy Policy Read article
- 15th Advancing Women’s Leadership in Pharma & Healthcare | BioFocus
< Back LIVX 2026 8th – 10th December, 2026 Dubai, UAE Hosting the most powerful global ecosystem for longevity, biotech, and investment. ! Widget Didn’t Load Check your internet and refresh this page. If that doesn’t work, contact us. Previous Next
- 5th Chief Patient Officer Summit | BioFocus
< Back 22nd – 23rd July, 2025 Boston, MA 5th Chief Patient Officer Summit The 5th Chief Patient Officer Summit is THE annual meeting place to share industry best practices and cutting-edge , innovative case studies in patient advocacy and engagement. This popular, “go to” event meaningfully connects peers who are building transparent, integral relationships with patient communities and cultivating a corporate culture dedicated to providing the best possible patient experience. In the heart of Boston, you will experience a community of fellowship and valuable content focused on listening and co-creating with patient communities and actionizing on patient feedback. Through best practice sharing and a deep exploration of timely topics, you’ll not only expand your valuable connections but emerge from the meeting ready for leadership and next steps to ensure the patient voice is heard and reflected in R&D, clinical and post approval. Previous Register now Next
- Global Pharma and Biotech Summit | BioFocus
< Back 5th – 7th November, 2024 London, United Kingdom Global Pharma and Biotech Summit A Financial Times Live event, the Global Pharma and Biotech Summit is the essential annual event for you to stay on top of the latest trends and innovations in life sciences. Industry leaders and experts will discuss what’s new in areas such as drug discovery, clinical trials, market access and patient engagement. Previous Register now Next
- Pfizer Halts Development of Oral GLP-1 Agonist Danuglipron Amid Liver Safety Signal
Pfizer halts once-daily oral GLP-1 program danuglipron after liver safety concerns, shifting focus to alternative obesity targets including a GIPR antagonist in Phase 2. < Back Pfizer Halts Development of Oral GLP-1 Agonist Danuglipron Amid Liver Safety Signal Pfizer halts once-daily oral GLP-1 program danuglipron after liver safety concerns, shifting focus to alternative obesity targets including a GIPR antagonist in Phase 2. Pfizer Inc. (NYSE: PFE) has discontinued development of its oral GLP-1 receptor agonist, danuglipron (PF-06882961), following a clinical finding of potential drug-induced liver injury during Phase 2 dose-optimization studies. The decision marks a strategic retreat from one of the few oral GLP-1 assets positioned to challenge the current dominance of injectable incretin therapies in the obesity market. Danuglipron, a small-molecule GLP-1R agonist administered once daily, had been in development as an oral alternative to GLP-1 peptide injectables such as semaglutide (Novo Nordisk’s Wegovy) and tirzepatide (Eli Lilly’s Zepbound). Pfizer reported that while the pharmacokinetic and tolerability profiles in its latest once-daily formulation study met key objectives, a single case of elevated liver enzymes consistent with potential drug-induced liver injury prompted an internal risk-benefit reassessment. “Although the formulation achieved desired PK exposure and early indicators of efficacy, we observed an adverse hepatic event that, while reversible upon discontinuation, raised enough concern to halt development,” said Mikael Dolsten, MD, PhD, Chief Scientific Officer and President, Pfizer R&D. This decision follows an earlier discontinuation in 2023 of danuglipron’s twice-daily formulation, which suffered from dose-limiting GI tolerability issues including nausea and vomiting. The once-daily version was expected to mitigate these adverse effects while preserving weight loss efficacy. Implications for GLP-1 Drug Development and Competitive Landscape Pfizer’s exit from danuglipron development underscores the complexities of formulating safe, orally bioavailable GLP-1R agonists. Despite its promising preclinical and early clinical data, danuglipron faced increasing safety scrutiny given the class-wide regulatory sensitivity to hepatotoxicity and long-term tolerability in a chronic disease context. With obesity positioned as a multi-billion-dollar therapeutic area projected to exceed $100B globally by 2030, Pfizer’s withdrawal leaves a narrower field of oral GLP-1 contenders. Eli Lilly’s orforglipron and Viking Therapeutics’ VK2735, among others, continue to progress in clinical development, each aiming to deliver oral or small-molecule alternatives to peptide-based therapies. Market reaction to Pfizer’s move was swift. Shares of Novo Nordisk and Eli Lilly rose modestly on Monday, with investors viewing the decision as a de-risking of their market share in obesity and type 2 diabetes. Meanwhile, Pfizer reaffirmed its commitment to the space through other mechanisms. Strategic Refocus: GIPR Antagonist in the Pipeline While terminating danuglipron, Pfizer highlighted ongoing development of an oral glucose-dependent insulinotropic polypeptide receptor (GIPR) antagonist candidate, currently in Phase 2. GIP modulation is being explored both as monotherapy and in combination with GLP-1 agents for synergistic metabolic effects. “We remain committed to advancing next-generation metabolic therapies and are reallocating our internal resources toward novel mechanisms, including oral GIPR antagonists and other first-in-class approaches,” Dolsten said. Pfizer did not disclose timelines for GIPR candidate readouts but indicated continued focus on cardiometabolic disease as a core strategic priority. The company is expected to provide pipeline updates in upcoming earnings calls and at scientific congresses later this year. Outlook Pfizer’s withdrawal from danuglipron reflects both the regulatory and scientific headwinds facing oral GLP-1 drug development. The hepatic safety signal, even in a single subject, proved incompatible with chronic use in a preventive population — a reminder of the high safety bar required for obesity pharmacotherapies. With major pharma consolidating around either peptide injectables or next-generation dual/triple agonist platforms, the search for safe, effective, and convenient oral agents continues — and remains a highly competitive frontier for innovation. Author BioFocus Newsroom Previous Next
- ESHRE 40th Annual Meeting | BioFocus
< Back LIVX 2026 8th – 10th December, 2026 Dubai, UAE Hosting the most powerful global ecosystem for longevity, biotech, and investment. ! Widget Didn’t Load Check your internet and refresh this page. If that doesn’t work, contact us. Previous Next
- NHS Delivers First CAR-T Treatment for Adult B-cell ALL Patient | BioFocus
< Back NHS Delivers First CAR-T Treatment for Adult B-cell ALL Patient Pioneering ‘living drug’ offers new hope for adults with aggressive leukaemia. The first patient to receive a pioneering form of CAR-T therapy for aggressive leukaemia on the NHS has described the experience as both “fantastic” and “very sci-fi”. Oscar Murphy, 28, from Bury, became the inaugural recipient of the treatment for B-cell acute lymphoblastic leukaemia (B-cell ALL) at Manchester Royal Infirmary on 2 January, when he began the first of two infusions of his genetically modified immune cells. The immunotherapy, often referred to as a “living drug”, is designed to recognise and kill cancerous cells and will now be funded across several NHS centres in England. Around 50 patients a year are expected to qualify, though clinicians believe the number could rise. Murphy was diagnosed with B-cell ALL in March 2025 and initially underwent chemotherapy followed by a donor stem cell transplant in July. His cancer relapsed just months later. “The leukaemia I've got is so fast-acting,” he said. “It needs an even quicker response to stop it. And we've now got an answer for that.” Clinical trial data underpinning the NHS rollout showed 77% of patients entered remission after treatment, with half displaying no detectable cancer three and a half years later. On average, patients gained an additional 15.6 months of life. According to Murphy’s haematologist, Dr Eleni Tholouli, the new iteration of CAR-T therapy is both safer and more effective than existing options. “Usually, this type of leukaemia is very aggressive and adult patients don't live beyond six to eight months. With this therapy, we are able to offer them years and potentially a cure. It's very significant and is revolutionising the way we tackle this cancer.” CAR-T therapy has been available on the NHS for several years for certain leukaemias and lymphomas, but this marks the first time adults with B-cell ALL have had access to the treatment. The breakthrough also comes amid ongoing conversations about who can access CAR-T therapies and when. In our earlier feature on Myeloma UK’s campaigning efforts, stakeholders warned that while the science is advancing quickly, equitable access across the NHS is yet to catch up. Murphy’s personalised therapy required T-cells to be extracted and sent to a manufacturing site in Stevenage, where they were engineered using a viral vector to introduce a genetic sequence that enables them to recognise malignant cells. Newly expressed surface receptors act like a lock-and-key system to identify the cancer, transforming the cells into chimeric antigen receptor T-cells, or CAR-T cells, which are expanded into the millions before being cryopreserved. The first infusion delivered around 100 million CAR-T cells contained in just a few teaspoons of fluid. A second infusion of 300 million cells followed. As a living therapy, the reprogrammed T-cells are expected to persist in the body, continue dividing and maintain anti-cancer activity over time. Murphy admitted he was astonished that such a small volume could deliver such a potent intervention. “It's very sci-fi, but if it means it gets rid of the cancer permanently and my own cells can do it it's just fantastic.” The treatment is manufactured by Autolus , a University College London spin-out . During earlier clinical studies, patient cells had to be shipped to laboratories in the US, underscoring the significance of onshore manufacturing capacity. The therapy carries a list price of £372,000 per infusion, though the NHS has negotiated confidential discounts. Eligible patients over 26 whose B-cell ALL has relapsed or failed to respond to treatment will be able to access the therapy at centres in Manchester, Cambridge, Newcastle, Sheffield, Plymouth and London. Patients from Wales and Northern Ireland will need to travel to England, and the treatment is not yet approved in Scotland. NHS England estimates that around 50 patients a year may benefit, but Tholouli told the BBC she believed more candidates will emerge, and predicted the therapy could ultimately replace stem cell transplantation as a first-line approach. Prof Peter Johnson, NHS National Clinical Director for Cancer, described the development as a “landmark moment” for people with aggressive blood cancers, adding that it was “remarkable” that a treatment conceived through UK research was now being delivered across the health service. “It will help more people like Oscar live longer and healthier lives.” The potential of the therapy is already evident in earlier patients. Chris Williams, 29, from Belfast, who was treated in Manchester while the therapy was still experimental, has now been in remission for nearly three years. “A few years ago I was very unwell and now I'm able to live a full life. I was able to go back to work. I also met Chloe and now we're engaged. I have fantastic support from my family and they are over the moon.” Murphy married his fiancée Lauren at Manchester Royal Infirmary last month, conscious of the uncertainties surrounding his condition, though the couple have planned a second ceremony for October. Looking ahead, his ambitions are disarmingly ordinary. “I want children and the white picket fence with my amazing wife, I just want that normality. This is my gateway to doing it and I can't wait.” Author BioFocus Newsroom Previous Next
- Astellas Pharma Invests $50 million in AviadoBio to Bolster Gene Therapy Offering
The pharmaceutical giant invests in AviadoBio, giving them an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). < Back Astellas Pharma Invests $50 million in AviadoBio to Bolster Gene Therapy Offering The pharmaceutical giant invests in AviadoBio, giving them an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). Astellas Pharma has invested $50 million in AviadoBio, giving the pharmaceutical giant an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). AviadoBio's innovative approach targets a specific protein deficiency associated with the disease, aiming to deliver long-term therapeutic benefits. Specifically, Astellas is interested in AviadoBio's single dose AAV-based gene therapy, AVB-101, currently in phase 1/2 clinical trials. The partnership also includes potential milestone payments if development progresses successfully, showcasing Astellas' commitment to gene therapy as a major focus for treating neurodegenerative diseases. The deal is part of Astellas’ broader strategy to expand its gene therapy pipeline. AviadoBio's CEO, Lisa Deschamps , commented “As we complete dosing of the first cohort of patients in our phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia". Of the $50 million total sum being paid by Astellas Pharma, $30 million constitutes an upfront payment while the remaining $20 million comes in the form of an equity investment. In return, Astellas will be granted the option of being the therapy's exclusive global license holder. Astellas Pharma is a Japanese multinational pharmaceutical company focused on developing innovative therapies in various areas, including oncology, immunology, and gene therapy. It is heavily investing in cutting-edge medical technologies, such as gene therapies, to address complex diseases. AviadoBio , a London-based biotech, specializes in gene therapies for neurodegenerative diseases. The company is working on treatments that target specific genetic causes of these conditions, with a particular focus on frontotemporal dementia (FTD), aiming to deliver durable therapeutic benefits. Author BioFocus Newsroom Previous Next











