A new CDMO partnership aims to bring clinical-grade AAV gene therapies to patients with LGMD types 2C and 2D, with an IND filing targeted for the second half of 2026 < Back Andelyn Biosciences and Evolyra Therapeutics Join Forces to Advance Gene Therapies for Limb-Girdle Muscular Dystrophy A new CDMO partnership aims to bring clinical-grade AAV gene therapies to patients with LGMD types 2C and 2D, with an IND filing targeted for the second half of 2026 Andelyn Biosciences, a Columbus, Ohio-based cell and gene therapy contract development and manufacturing organisation (CDMO), has announced a manufacturing partnership with Evolyra Therapeutics to produce clinical-grade adeno-associated virus (AAV) gene therapies for two forms of limb-girdle muscular dystrophy (LGMD): types 2C and 2D, also designated LGMDR5 and LGMDR3. The deal brings together Evolyra's proprietary next-generation viral vector with Andelyn's established manufacturing infrastructure, and represents another step forward in a field that is steadily gaining momentum. Limb-girdle muscular dystrophies are a group of around 32 distinct genetic disorders characterised by progressive weakness and wasting of the muscles surrounding the hips and shoulders. There are no approved disease-modifying treatments for any LGMD subtype, and the conditions can significantly reduce quality of life, mobility, and life expectancy. With more than 580,000 patients affected worldwide and a global market estimated to exceed $100 billion, the commercial and clinical case for effective therapies is considerable. Evolyra's programmes target the LGMDR3 and LGMDR5 subtypes specifically, a segment representing roughly 20,000 patients and an $11 billion market opportunity. At the core of Evolyra's approach is AAVMYO2, a proprietary muscle-targeting AAV vector that the company says substantially improves skeletal muscle expression while reducing liver toxicity relative to first-generation vectors. Liver toxicity has been one of the more persistent concerns in systemic AAV gene therapy, and addressing it at the vector level rather than through post-treatment management is a meaningful engineering priority. Evolyra reports complete protein expression and muscle restoration in preclinical animal models, though as with all such results, the translation to human trials will be the real test. Evolyra's approach is designed to deliver a complete, functional gene rather than a partial replacement, which the company considers essential for effective treatment Evolyra Therapeutics of conditions like LGMDR3 and LGMDR5. The company is building what it describes as a scalable, modular gene therapy platform, drawing on a consortium of over 500 patients to provide biomarker data, disease progression tools, and trial readiness support Evolyra Therapeutics. Under the partnership, Andelyn will apply its AAV Curator® Platform to support IND-enabling studies and Good Manufacturing Practice (GMP) production for Evolyra's clinical trials. The platform is designed to support the full arc of rare disease therapy development, from preclinical stages through to clinical manufacturing, without requiring companies to rebuild their supply chain at each phase transition. "Evolyra exemplifies the kind of transformative science we are eager to support," said Matt Niloff, Chief Commercial Officer at Andelyn Biosciences. "Their innovative approach to treating LGMDs with an AAV gene therapy aligns with our mission to accelerate access to life-changing treatments for patients with rare and ultra-rare diseases. We are very honored to help bring this breakthrough therapy to the patients who need it most." Nicholas Johnson, MD, CEO of Evolyra Therapeutics, pointed to the manufacturing continuity as a particular advantage: "We appreciate the partnership and commitment from Andelyn Biosciences. Their platform will allow a seamless transition between the different phases of our therapeutic development, ultimately accelerating our ability to bring these therapies to individuals living with LGMD as quickly as possible." Evolyra is targeting an IND submission in the second half of 2026, with Phase I/II clinical trials to follow. The company recently closed a $5 million seed round and is currently in the process of raising a $20 million Series A to fund programme advancement. That funding trajectory is not unusual for a company at this stage, though completing the Series A in the current biotech financing environment will matter considerably for whether the 2026 timeline holds. The partnership arrives at a time of genuine, if cautious, progress in LGMD gene therapy more broadly. In March 2026, Atamyo Therapeutics shared the first clinical results for its ATA-200 gene therapy in LGMD-2C/R5, from a Phase Ib/II trial at the University of Florida's Powell Gene Therapy Center. Ddw-online Meanwhile, Sarepta Therapeutics has multiple LGMD programmes in clinical development, with its SRP-9003 therapy meeting the primary endpoint of its Phase 3 EMERGENE trial, demonstrating robust beta-sarcoglycan protein expression in treated patients. CGTLive® These parallel programmes in overlapping LGMD subtypes underscore both the scale of unmet need and the competitive dynamics emerging in this space. Evolyra will need to differentiate on both the clinical profile of AAVMYO2 and its speed to trials. The Andelyn partnership is designed to help on the latter count, and the combination of an experienced CDMO with a next-generation vector and a patient-connected clinical network gives Evolyra a credible, if still early-stage, development platform. For patients with LGMDR3 and LGMDR5, who currently have no therapeutic options beyond symptom management, the prospect of multiple well-resourced gene therapy programmes entering clinical testing is the most tangible reason for optimism. Whether any of them ultimately translate into approved treatments remains to be seen. Author BioFocus Newsroom Previous Next

< Back 5th – 7th February, 2025 Santa Clara, CA PMWC 2025 PMWC, the “Precision Medicine World Conference” is the largest & original annual conference dedicated to precision medicine. PMWC’s mission is to bring together recognized leaders, top global researchers and medical professionals, and innovators across healthcare and biotechnology sectors to showcase practical content that helps close the knowledge gap between different sectors, thereby catalyzing cross-functional fertilization & collaboration in an effort to accelerate the development and spread of precision medicine. Previous Register now Next

AI-powered 3D lung imaging to accelerate assessment of CAL101, a first-in-class antibody targeting a key driver of fibrosis. < Back Qureight AI to Power Calluna’s Phase 2 IPF Study AI-powered 3D lung imaging to accelerate assessment of CAL101, a first-in-class antibody targeting a key driver of fibrosis. Qureight, a leading techbio company transforming clinical imaging through AI, today announced it will provide its advanced 3D imaging platform to support Calluna Pharma’s Phase 2 AURORA trial of CAL101, a novel investigational therapy for idiopathic pulmonary fibrosis (IPF). The partnership brings together cutting-edge imaging analytics and innovative drug development to tackle one of the most challenging and life-limiting lung diseases. IPF is a progressive and currently incurable condition with limited treatment options. CAL101, Calluna’s lead monoclonal antibody, targets S100A4, a protein that acts as an upstream amplifier of multiple pro-fibrotic pathways involved in IPF. Qureight’s platform will be used throughout the trial to deliver high-resolution, quantitative assessments of lung anatomy, including fibrosis volume and other imaging biomarkers. These insights will inform both patient selection and ongoing efficacy evaluation. “Calluna’s decision to work with us on this pivotal trial reflects the power of our AI platform to deliver real-time, clinically meaningful data,” said Steven Bishop, Chief Data Officer at Qureight. “IPF is a disease where time matters, for both patients and drug developers. Our technology enables faster, more precise evaluation of how new therapies impact lung structure, helping accelerate progress in this high-need area.” The AURORA study is a randomised, double-blind, placebo-controlled trial enrolling 150 IPF patients across more than 50 global sites in the US, UK, EU, Turkey, and South Korea. Participants will receive seven monthly infusions of CAL101 or placebo, with forced vital capacity (FVC), a key measure of lung function, serving as the primary endpoint. Qureight’s AI tools will allow researchers to detect subtle changes in lung fibrosis that are often missed by conventional image interpretation. Unlike traditional methods, which are manual, slow, and susceptible to human variability, Qureight’s platform delivers standardised, high-fidelity results that can be acted on quickly. “Dosing the first patient in AURORA marked a major milestone for Calluna and our mission to transform outcomes for people living with IPF,” said Dr. Jonas Hallén, Co-Founder and Chief Medical Officer at Calluna Pharma. “Qureight’s imaging technology is an integral part of the study, giving us critical, data-driven insight into how CAL101 may halt or slow lung function decline.” The collaboration underscores a growing trend in clinical research: integrating AI and digital health tools to de-risk development and accelerate timelines. Qureight’s role in AURORA highlights how advanced imaging analytics can enhance both the scientific rigor and efficiency of trials in complex diseases like IPF. About Qureight Qureight is a Cambridge-based techbio company using AI and cloud-based platforms to curate, analyze, and interpret imaging and clinical data in complex lung and heart diseases. Its technology enables real-time insights to support faster, more accurate clinical decision-making and drug development. About Calluna Pharma Calluna Pharma is a clinical-stage biopharmaceutical company developing novel therapies for fibrotic and inflammatory diseases. Its lead candidate, CAL101, is a first-in-class monoclonal antibody targeting S100A4, a key molecular driver of fibrosis. Author BioFocus Newsroom Previous Next

In a landmark advancement for genetic medicine, the NHS is set to offer a revolutionary gene therapy, Casgevy, to patients with transfusion-dependent beta thalassaemia. < Back NHS Introduces Groundbreaking Gene Therapy for Beta Thalassaemia In a landmark advancement for genetic medicine, the NHS is set to offer a revolutionary gene therapy, Casgevy, to patients with transfusion-dependent beta thalassaemia. The new therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics , marks a significant milestone in the treatment of this severe genetic blood disorder. Casgevy has been approved by the National Institute for Health and Care Excellence (NICE) and will be available from August 7. It is anticipated to benefit around 460 patients in England. Beta thalassaemia, a condition where the body produces insufficient haemoglobin, affects approximately 2,300 people in the UK, predominantly those of Mediterranean, Asian, or Middle Eastern descent. The disorder often leads to severe anaemia, necessitating lifelong blood transfusions and reducing life expectancy to around 50 years. Casgevy offers a potential cure by employing CRISPR gene-editing technology. The treatment involves extracting stem cells from a patient's bone marrow, modifying the genes in a lab to produce functioning haemoglobin, and reinfusing the corrected cells back into the patient. This process reprograms the cells to produce foetal haemoglobin, bypassing the genetic defect that hampers adult haemoglobin production. In clinical trials, 93% of patients did not require blood transfusions for at least a year post-treatment. "This is a historic moment for people living with beta thalassaemia," said Amanda Pritchard, NHS Chief Executive. "This therapy offers a life free from regular transfusions and the debilitating symptoms of the disorder, promising a longer and healthier life." The gene-editing tool CRISPR, which won the Nobel Prize for Chemistry in 2020, is integral to this treatment. It precisely targets and edits the DNA responsible for the haemoglobin switch from foetal to adult forms. This innovative approach not only addresses the symptoms but also targets the root cause of beta thalassaemia. The therapy's introduction follows a thorough evaluation by NICE, which considered both its costs and benefits. While the listed price of Casgevy is £1.65 million per patient, NHS England has negotiated a lower price to make it accessible. The treatment will be available at seven specialist centres across the UK, ensuring it reaches those in need efficiently. "This transformative treatment offers patients a life-changing opportunity, enabling them to repair their own cells and embrace a future free from the challenges of their condition," stated Romaine Maharaj, Executive Director of the UK Thalassaemia Society. This gene therapy not only represents a breakthrough for beta thalassaemia but also paves the way for future treatments of other genetic disorders, such as sickle cell anaemia, which is currently under negotiation for NHS approval. The rollout of Casgevy by the NHS signifies a new era in the treatment of genetic blood disorders, providing hope and improved quality of life for many patients. This advancement underscores the potential of gene therapy to address and potentially cure debilitating conditions, marking a significant achievement in medical science. Author BioFocus Newsroom Previous Next

A licensing deal with Apertura Gene Therapy gives the London-based biotech a clinically validated delivery system to pair with its gene-silencing platform, but the hard work is still ahead. < Back AviadoBio Bets on Blood-Brain Barrier Technology in Push for One-Time Alzheimer's Treatment A licensing deal with Apertura Gene Therapy gives the London-based biotech a clinically validated delivery system to pair with its gene-silencing platform, but the hard work is still ahead. For a long time - decades - the blood-brain barrier has been one of the most stubborn obstacles in neurology. Protective by design, it blocks the vast majority of therapeutics from reaching the central nervous system, a problem that has frustrated drug developers targeting everything from brain tumours to Alzheimer's disease. A new licensing agreement between Aviado Bio and Apertura Gene Therapy represents the latest attempt to crack this problem, this time by exploiting a well-characterised biological transport mechanism to ferry gene therapy directly into the brain. Under the deal , London-based AviadoBio has licensed TfR1 CapX™, a next-generation adeno-associated virus (AAV) capsid developed by New York-based Apertura. The capsid is engineered to bind human transferrin receptor 1 (hTfR1), a protein expressed on the surface of brain endothelial cells that naturally shuttles iron-bound transferrin across the blood-brain barrier. By hitching a ride on this established pathway, TfR1 CapX is designed to enable intravenous delivery of genetic payloads to neurons and astrocytes throughout the brain and spinal cord, without the need for invasive surgical administration. AviadoBio intends to combine TfR1 CapX with its proprietary vMiX™ platform, a vectorised RNA interference system that uses AAV delivery to silence disease-causing genes. Unlike conventional RNAi therapies requiring repeated dosing, vMiX is designed to provide durable gene knockdown from a single administration. The company is applying the platform across a pipeline targeting neurodegenerative diseases, with its most advanced CNS programme being AVB-406, an investigational gene therapy for Alzheimer's disease and related tauopathies, which works by silencing MAPT, the gene encoding tau protein. Tau accumulation is a defining pathological feature of Alzheimer's and a range of other neurodegenerative conditions collectively known as tauopathies. Reducing tau expression at the genetic level, rather than attempting to clear aggregated protein after the fact, is a mechanistically distinct approach from many of the antibody-based strategies that have dominated the Alzheimer's field in recent years. Lisa Deschamps, Chief Executive Officer of AviadoBio, said the agreement "strengthens a key component of our strategy to develop transformative genetic medicines for neurodegenerative diseases" and that it: "enhances our ability to advance AVB-406 and broadens the potential of our vMiX platform in Alzheimer's disease, other tauopathies, and additional high-need indications." The choice of TfR1 as a brain shuttle target is not arbitrary. It is among the most extensively characterised CNS delivery receptors in the field, with a track record in clinical contexts spanning paediatric and geriatric populations. Alex Bloom, Chief Technology Officer at AviadoBio, described it as "the most extensively studied, clinically validated brain shuttle receptor," adding that the licence "enhances our ability to advance our vMiX gene-silencing platform to enable one-time, IV-delivered gene silencing in rare and common disorders with high unmet medical need." Preclinical data shared by both AviadoBio and independent groups suggest TfR1 CapX achieves widespread neuronal and astrocytic transduction across the brain and spinal cord, a meaningful distribution profile for diseases like Alzheimer's that affect broad neural networks rather than discrete anatomical regions. Apertura itself describes TfR1 CapX as a second-generation capsid, demonstrating superior CNS delivery over its predecessor, BI-hTFR1, the first-generation version of which was published in the journal Science. Dave Greenwald, Managing Director at Deerfield Management and Executive Chairman at Apertura, expressed confidence in the pairing: "We believe this combination has the opportunity to provide transformational benefit to patients in a safe and scalable approach." That said, AVB-406 remains at the preclinical stage, and the distance from preclinical promise to clinical validation in Alzheimer's disease is well-documented and rarely straightforward. Gene therapy for CNS indications brings its own manufacturing, safety, and immunogenicity considerations, and no AAV-based therapy for Alzheimer's has yet demonstrated clinical efficacy. The field is watching closely, but cautious optimism is warranted rather than anything stronger. The deal's near-term significance will become clearer in the coming weeks. AviadoBio is scheduled to present three oral presentations on AVB-406 at the American Society of Gene & Cell Therapy Annual Meeting in Boston, taking place 11th - 15th May 2026. The presentations will cover preclinical, translational, and manufacturing data, offering the scientific community its most comprehensive public look yet at a programme that, if the underlying biology holds up, could eventually represent a genuinely novel approach to one of medicine's most intractable diseases. Whether TfR1 CapX and vMiX together can deliver on their mechanistic rationale remains to be seen. But as delivery technology goes, the combination is scientifically credible, and for a disease that has resisted treatment for so long, credible is a reasonable place to start. Author BioFocus Newsroom Previous Next

< Back 12th November, 2024 New York, NY NY Metro Builds Bio+ 7th Annual Symposium for Life Science Innovation and Development Join us at the Seventh Annual Symposium for Life Science Innovation and Development to hear how private and public investments are driving exponential growth for the life science industry, what new initiatives are furthering innovation in the space and what this means for the Greater NY Metropolitan Area.The symposium will also highlight a new London/UK life sciences chapter and we’ll present the research report from the London life sciences study tour which took place in April 2024. Previous Register now Next

The drug, Wainzua, specifically targets type 2 inflammation-driven asthma, a form associated with severe, persistent symptoms and significant impairment in quality of life. < Back AstraZeneca's Wainzua Recommended for EU Approval as New Therapy for Severe Asthma The drug, Wainzua, specifically targets type 2 inflammation-driven asthma, a form associated with severe, persistent symptoms and significant impairment in quality of life. In a significant development for asthma treatment, AstraZeneca announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of its new therapy, Wainzua (inavolisib). The drug, designed to address severe asthma in adults, holds promise for individuals whose condition remains poorly controlled with currently available therapies. The European Commission will consider the CHMP recommendation and is expected to make a final decision within the coming months. Wainzua specifically targets type 2 inflammation-driven asthma, a form associated with severe, persistent symptoms and significant impairment in quality of life. Patients with this type of asthma often rely on high doses of corticosteroids and may still experience frequent exacerbations, raising the need for more effective treatment options. By acting on this specific inflammatory pathway, Wainzua offers a targeted therapeutic approach to reduce exacerbations, lower corticosteroid use, and improve respiratory function. Key Findings and Clinical Impact In Phase III trials, Wainzua demonstrated remarkable results. Patients receiving Wainzua experienced a substantial reduction in exacerbations compared to those on standard care, as well as improved lung function and symptom control. The benefits observed in these studies underscore the potential of Wainzua to help patients regain control of their condition and enhance their overall quality of life. Dr. Mene Pangalos, Executive Vice President of BioPharmaceuticals R&D at AstraZeneca, stated: “This CHMP recommendation underscores our commitment to advancing science and providing innovative solutions for chronic respiratory diseases. Wainzua has the potential to transform the lives of patients living with severe asthma, an area where unmet needs remain high.” Anticipated Approval and Launch Pending approval by the European Commission, Wainzua could be available to patients across the EU by early 2025. AstraZeneca is poised to work closely with healthcare systems across Europe to ensure Wainzua’s availability, with a strong emphasis on affordability and accessibility. AstraZeneca’s success with Wainzua highlights the company's ongoing dedication to developing specialized treatments for chronic respiratory diseases. If approved, Wainzua will join AstraZeneca’s expanding portfolio of respiratory and immunology therapies, marking a significant advancement in the treatment of severe asthma and promising a new wave of relief for patients throughout Europe. Author BioFocus Newsroom Previous Next

< Back The 2024 Nobel Prize in Physiology or Medicine Winners The 2024 Nobel Prize in Physiology or Medicine was awarded to U.S. scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (miRNA) and its role in gene regulation. The 2024 Nobel Prize in Physiology or Medicine was awarded to U.S. scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (miRNA) and its role in gene regulation. MicroRNAs are small RNA molecules that regulate gene expression at the post-transcriptional level, providing critical insights into how cells control protein production. This discovery has significantly expanded our understanding of gene regulation and has had profound implications for medical research, particularly in the study of diseases such as cancer and developmental disorders. Background on MicroRNA MicroRNAs (miRNAs) are small, non-coding RNA molecules that play an essential role in regulating gene expression across various organisms, including plants, animals, and some viruses. First discovered in the developmental model organism Caenorhabditis elegans, it is now known that the human genome encodes over 2,600 miRNAs , with some estimates suggesting that these collectively target approximately 60% of all genes. miRNAs bind to complementary sequences in messenger RNA (mRNA), typically in the 3' untranslated region (UTR), leading to either the degradation of the mRNA or repression of its translation into protein. This post-transcriptional regulation is crucial for controlling protein production, influencing key cellular processes such as growth, differentiation, apoptosis, and metabolism. The discovery of miRNAs in the early 1990s , led by Ambros and Ruvkun, revolutionized our understanding of gene regulation. MicroRNAs are involved in nearly every biological process, and their dysregulation has been linked to various diseases, including cancer, cardiovascular diseases, and neurodegenerative disorders. Some miRNAs function as tumor suppressors , blocking cancer-causing pathways, while others may promote tumor growth by silencing protective genes. This dual role makes miRNAs a central focus in cancer research, where they are being studied as potential diagnostic markers and therapeutic targets. Beyond their role in disease, miRNAs hold significant promise in personalized medicine. Their expression patterns vary in specific diseases, offering opportunities for more precise diagnostics and targeted treatments. Current therapeutic strategies include using miRNA mimics to restore normal function in diseased cells or antagonists to inhibit overactive miRNAs. These approaches could potentially revolutionize treatments for complex diseases like cancer, signalling a new era in medical innovation. About the Laureates Victor Ambros, a professor of molecular medicine at UMass Chan Medical School, is renowned for his discovery of the first known microRNA, lin-4 , in 1993. While studying gene regulation in the roundworm Caenorhabditis elegans , Ambros found that this small RNA molecule regulates gene expression by binding to mRNA, revealing a new layer of genetic control. This breakthrough laid the foundation for understanding how miRNAs influence many biological processes, such as development, cell differentiation, and disease mechanisms like cancer. Ambros continues to investigate the roles of miRNAs in gene regulation, contributing to our knowledge of developmental and disease-related pathways. Gary Ruvkun, a professor of genetics at Harvard Medical School and an investigator at Massachusetts General Hospital, expanded on Ambros' work with his discovery of the second microRNA, let-7 , in 2000. This microRNA is conserved across multiple species, from worms to humans, emphasizing the universal role of miRNAs in gene regulation. Ruvkun’s pioneering research illuminated how miRNAs control essential gene functions that are crucial for the development and functioning of multicellular organisms. His work has not only advanced the field of microRNA research but has also contributed to our understanding of metabolism and aging through insulin-like signalling pathways. Together, Ambros and Ruvkun have reshaped the field of molecular biology. Their Nobel-winning research has opened new avenues for studying genetic regulation, with far-reaching implications for treating diseases. Significance of the Award The 2024 Nobel Prize in Physiology or Medicine honors the revolutionary contributions of Ambros and Ruvkun, whose discovery of microRNAs has fundamentally transformed our understanding of gene regulation. These small, non-coding RNA molecules serve as critical regulators of gene expression, with wide-ranging implications for human health. By silencing or degrading specific mRNAs, miRNAs influence vital biological processes including development, differentiation, and cell survival. This discovery has allowed scientists to understand how genes are selectively activated or deactivated in various cell types, which is essential for normal development and disease prevention. The medical potential of miRNAs extends beyond foundational science. Their roles as tumor suppressors or oncogenes make them significant targets in cancer therapies. For instance, researchers are developing miRNA-based treatments aimed at replenishing tumor-suppressing miRNAs or inhibiting those that promote cancer. Furthermore, miRNAs are being explored as biomarkers due to their stability in human fluids, which enables non invasive disease monitoring. Although challenges remain, such as delivery and toxicity, clinical trials of miRNA-based therapies , including MRG-201 for fibrosis and MRG-229 for pulmonary fibrosis, are paving the way for future breakthroughs in personalized medicine. This Nobel Prize emphasizes the critical importance of miRNAs, not only in advancing genetic research but also in shaping the future of disease treatment. Conclusion The discovery of microRNAs by Victor Ambros and Gary Ruvkun has unveiled a new dimension of gene regulation, with lasting impacts on both basic science and medicine. Their pioneering work revealed how microRNAs control gene expression, revolutionizing our understanding of cell function and development in multicellular organisms. As research into miRNAs advances, especially regarding their therapeutic potential for diseases such as cancer and epilepsy, this groundbreaking discovery will continue to inspire innovations in personalized medicine and biotechnology. Author Ramya Nadig , freelance contributor Previous Next

< Back 16th – 19th June, 2025 Boston, MA 2025 BIO International Convention The BIO International Convention is the world’s largest biotechnology event, bringing together industry leaders from around the world to learn, network, and build relationships. The convention is produced by the Biotechnology Innovation Organization (BIO), the world’s largest advocacy association for biotechnology. Previous Register now Next

Allogene Therapeutics has revised its ALPHA3 CAR T trial to use only standard lymphodepletion after a patient death linked to its investigational antibody. < Back Allogene Pivoting Lymphodepletion Strategy in ALPHA3 CAR‑T Trial Following Patient Death Allogene Therapeutics has revised its ALPHA3 CAR T trial to use only standard lymphodepletion after a patient death linked to its investigational antibody. Allogene Therapeutics (Nasdaq: ALLO), a pioneer in off-the-shelf CAR T cell therapy, has announced a significant update to its ALPHA3 Phase 2 clinical trial of cemacabtagene ansegedleucel (cema-cel) in first-line consolidation for large B-cell lymphoma (LBCL). In response to an adverse event, the company will now use the standard fludarabine and cyclophosphamide (FC) lymphodepletion regimen exclusively. The move comes after the trial’s FCA arm—which combined FC with ALLO‑647, an anti-CD52 monoclonal antibody—was halted following a Grade 5 adverse event involving liver failure due to a disseminated adenovirus infection. This fatality, occurring on Day 54 post-infusion, was attributed to ALLO‑647 rather than cema-cel, prompting an immediate safety review. Allogene will discontinue use of ALLO‑647 in all future protocols. Per CEO David Chang, the decision was made in coordination with the ALPHA3 Data and Safety Monitoring Board (DSMB), Steering Committee, and the U.S. Food and Drug Administration. Chang emphasized that no cases of severe viral infection or hepatic failure have occurred with FC-only lymphodepletion in any trial participants. Under the revised protocol, ALPHA3 will now proceed as a two‑arm randomized study: cema-cel following standard FC lymphodepletion versus observation alone (current standard of care). The study’s statistical design remains unaltered, with a futility analysis—focused on minimal residual disease (MRD) conversion—expected in the first half of 2026. Over 50 clinical sites across the U.S. and Canada remain active in the study. Chang commented: “The loss of a patient is always deeply saddening, […] administering cema-cel following standard FC lymphodepletion in an outpatient setting will simplify treatment, accelerate enrollment, and streamline regulatory pathways.” Analysts say this adjustment reinforces Allogene’s strategic pivot toward its Dagger® Platform, which aims to reduce reliance on antibodies like ALLO‑647 and minimize lymphodepletion altogether. All trials and pipeline programs will now focus on Dagger‑enabled candidates such as ALLO‑316 and ALLO‑329, which are being developed for renal cancer and autoimmune diseases, respectively. This protocol change closely follows a Fierce Biotech report revealing the patient death and Allogene’s swift decision to discontinue its investigational antibody use. The article confirmed that this was the only active trial still utilizing ALLO‑647. About Allogene Therapeutics Allogene Therapeutics is a clinical-stage biotechnology company at the forefront of developing allogeneic (“off-the-shelf”) chimeric antigen receptor T cell (AlloCAR T™) therapies for cancer and autoimmune diseases. Headquartered in South San Francisco, Allogene is led by a team of industry pioneers with deep expertise in cell therapy and immuno-oncology. The company’s proprietary technology platform is designed to deliver CAR T cell therapies that are readily available, scalable, and do not require patient-specific cell collection, aiming to make advanced cell therapies more accessible to patients around the world. Allogene’s pipeline includes multiple product candidates, such as cemacabtagene ansegedleucel (cema-cel) for large B-cell lymphoma, as well as innovative programs in solid tumors and autoimmune disorders. Author BioFocus Newsroom Previous Next

Revolutionary 3D multi-omics platform reveals new therapeutic targets, promising faster, more successful drug development. < Back Enhanced Genomics and ALBORADA Drug Discovery Institute Accelerate Alzheimer’s Drug Discovery Revolutionary 3D multi-omics platform reveals new therapeutic targets, promising faster, more successful drug development. A pioneering collaboration between biotech innovator Enhanced Genomics and The ALBORADA Drug Discovery Institute at the University of Cambridge has delivered a major breakthrough in Alzheimer’s research. The partnership, supported by Alzheimer’s Research UK, is already generating multiple novel drug targets that were previously undetectable using traditional genomic methods. By harnessing Enhanced Genomics’ proprietary 3D multi-omics platform, scientists have been able to map complex gene-regulatory interactions at an unprecedented scale and resolution. This has revealed critical new insights into the underlying biology of Alzheimer’s disease and accelerated the discovery of high-confidence, genetically validated drug targets. “This collaboration marks a turning point in how we discover drugs for complex diseases like Alzheimer’s,” said Dr. Dan Turner, Chief Scientific Officer at Enhanced Genomics. “Our platform doesn’t just look at the genome—it interprets how different regions of DNA interact in 3D space across specific human cell types. This gives us the power to uncover therapeutic targets hidden in the non-coding genome, something conventional tools simply can’t do.” The ALBORADA Drug Discovery Institute, based at the University of Cambridge, has used these findings to open new research pathways into drug development. “The insights provided by Enhanced’s technology have already led us to previously unknown targets with real therapeutic potential,” said Dr. John Skidmore, CSO at The ALBORADA Drug Discovery Institute. “This could significantly advance our ability to develop effective treatments for Alzheimer’s disease.” Alzheimer’s Research UK, the UK’s leading dementia research charity, sees this partnership as a model for future industry-academic collaborations. “This is the kind of ambitious, high-impact science we need to bring hope to the millions affected by dementia,” said Dr. Julia Dudley, Head of Strategic Programmes. “By combining cutting-edge technology with deep disease expertise, we’re accelerating progress toward the next generation of treatments.” The collaboration not only delivers immediate value in Alzheimer’s research, but also underscores the wider potential of Enhanced’s 3D multi-omics platform. By defining causal biology from disease-associated genetic variants, the technology dramatically improves both the speed and likelihood of success in early drug discovery. Looking ahead, Enhanced Genomics and The ALBORADA Drug Discovery Institute are exploring ways to expand their work into other neurodegenerative diseases. The company is also actively seeking new partnerships with pharmaceutical and biotech organizations interested in incorporating 3D multi-omics into their own drug discovery pipelines. About Enhanced Genomics: Enhanced Genomics is a biotechnology company revolutionizing drug discovery through its proprietary 3D multi-omics platform, capable of identifying high-confidence therapeutic targets from disease-associated variants across the entire genome. The platform enables faster, more efficient, and more successful drug development for common diseases with high unmet need. About The ALBORADA Drug Discovery Institute: Established by Alzheimer’s Research UK, The ALBORADA Drug Discovery Institute focuses on developing treatments that tackle the biological mechanisms driving neurodegenerative diseases, including Alzheimer’s. About Alzheimer’s Research UK: Alzheimer’s Research UK is the UK’s leading dementia research charity, dedicated to accelerating the discovery of new treatments and, ultimately, a cure for dementia. Author BioFocus Newsroom Previous Next

< Back 15th – 17th April, 2025 Rome, Italy The Cell & Gene Meeting on the Med The Cell & Gene Meeting on the Mediterranean is the leading conference bringing together the ATMP community from Europe and beyond. Covering a wide range of commercialization topics from market access and regulatory issues to manufacturing and financing the sector, this program features expert-led panels, extensive one-on-one partnering capabilities, exclusive networking opportunities, and 60+ dedicated presentations by leading publicly traded and privately held companies in the space. Join ARM for Europe’s premier conference for advanced therapies. Previous Register now Next