Tal Behar’s Post

Treatment for Sickle Cell Disease - FDA Approves Gene Therapies For those suffering from the agonizing effects of sickle cell disease, hope has long remained elusive. The severe inherited blood disorder throttles the body's oxygen supply, unleashing bouts of excruciating pain and life-threatening crises which can claim lives prematurely. But a groundbreaking FDA approval today marks a major milestone that could fundamentally transform treatment options for this debilitating illness. The newly FDA-approved treatment, called Casgevy, represents the first ever approved therapy using CRISPR gene editing. It works by harnessing the power of CRISPR to dial down production of hemoglobin in its adult form, while restoring levels of an alternative fetal hemoglobin that functions better for those with sickle cell disease. Casgevy reprograms the process that normally shuts off fetal hemoglobin after birth, allowing the body to revert to making this alternate form into adulthood instead. This effectively counteracts the defective adult hemoglobin that contorts blood cells into the dysfunctional sickle shape. By enabling patients to produce normal fetal hemoglobin again, Casgevy offers the promise of making sickle cell disease much milder, if not curing it altogether. The approval offers a “revolutionary” new option beyond the currently limited treatment alternatives for a disease affecting 100,000 Americans. After decades of hopes pinned on gene therapy, the ability to precisely edit genes using CRISPR has finally unlocked its vast potential. Casgevy represents the first CRISPR treatment to gain FDA approval for any condition - but likely only the first of many advances to come.  REFERENCE: •FDA: https://lnkd.in/gZBP6pPZ •NY Times: https://lnkd.in/gdFGAW3a •NPR: https://lnkd.in/gqVFnNGB •CHOP: https://lnkd.in/gR4rQ5n5

👨🔬💬 Cell & Gene Therapy News 👩🔬💬 🧬 Dr. Alfonso Sabater from the University of Miami restored the vision of 14-year-old Antonio Vento Carvajal, who was legally blind due to a rare genetic condition causing skin and eye blisters, using a gene therapy delivered through eyedrops. The therapy, named Vyjuvek which was reformulated by Krystal Biotech, Inc., delivered functional genes using an inactivated herpes simplex virus. After two years of testing, Antonio's vision improved significantly, offering hope for treating other eye diseases. The success marks a ground breaking advancement in gene therapy! 🦠 Replay and MD Anderson Cancer Center announced that the FDA has issued a ‘safe to proceed’ for the IND application for NY-ESO-1 TCR/IL-15 NK, a first-in-class engineered TCR-NK cell therapy for relapsed or refractory multiple myeloma. NY-ESO-1 TCR/IL-15 NK is being developed by Syena, an oncology-focused product company launched by Replay and MD Anderson based upon the scientific discoveries of Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation & Cellular Therapy at MD Anderson. Syena has an exclusive licensing agreement for MD Anderson’s TCR-NK platform. 🧬 Stoke Therapeutics announced positive new safety and efficacy data from patients treated with STK-001 in the two ongoing Phase 1/2a studies (MONARCH and ADMIRAL) and the SWALLOWTAIL open-label extension (OLE) study in children and adolescents with Dravet syndrome. These new data suggest clinical benefit for patients 2 to 18 years of age treated with multiple doses of STK-001. The observed reductions in convulsive seizure frequency as well as substantial improvements in cognition and behavior support the potential for disease modification in a highly refractory patient population. 🦠 Imperial College London start up NK:IO has raised an additional £1.2 million in investment, bringing its total seed funding to £5.1 million. This brings total equity financing to £3.2M and will enable the company to reach key milestones in progressing its differentiated NK cell therapy candidates into development. The investment, led by Cancer Research Horizons (the innovation engine at the core of Cancer Research UK), will be used to drive NK:IO’s cell therapy candidates through full pre-clinical testing, with ovarian cancer as the first target. 🧬 Alnylam Pharmaceuticals announced it has entered into a strategic agreement with Roche to develop and commercialize zilebesiran, Alnylam’s investigational RNAi therapeutic for the treatment of hypertension, which is currently in Phase 2 of development. The partnership allows for a bold development plan with the goal of disrupting the hypertension treatment paradigm globally while advancing Alnylam’s P5x25 strategy. 🛎 My aim is to be the messenger when it comes to your cell & gene therapy news. Follow #mCGT to stay up to date! #celltherapy #genetherapy #regenerativemedicine #biotech

Gene therapy is becoming a viable treatment approach for a variety of rare diseases, with numerous advancements occurring in 2023. Here, we delve into several recent successes in gene therapy for rare diseases, substantiated by scholarly data. 1. Advancements in 2023: - The Alliance for Regenerative Medicine anticipates the introduction of at least five more gene therapies for rare diseases in the U.S. market, including potential new treatments for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A. - Various gene therapy approvals for rare disease treatment are gaining momentum in 2023, which has led to the release of a comprehensive set of gene therapy development solutions to further boost rare disease research. 2. Gene Therapy Techniques: - Autologous hematopoietic stem and progenitor cell gene therapy (HSPC-GT) has emerged as an effective treatment modality for several inherited diseases, including primary immunodeficiencies. - The use of adeno-associated virus (AAV) vectors, retrovirus, and lentivirus vectors in gene therapy is showcased by various licensed treatments for a range of disorders from retinal epithelium deficiency to β-thalassaemia and refractory leukaemias. 3. Case Studies: - Research on PMM2-Congenital Disorder of Glycosylation (PMM2-CDG) revealed secondary mitochondrial dysfunction, indicating that manipulating mitochondrial function and autophagy could offer therapeutic benefits when combined with existing treatments. - Gene therapy for Usher 1F blindness is advancing with three candidate gene therapies, each employing a distinct approach to amend the disease-causing mutation. These therapies are under investigation in animal models, with optimistic prospects for clinical trials. 4. Future Prospects: - The surge in gene therapy research and development, primarily targeting loss-of-function mutation diseases, is poised to bring forth a range of new gene therapies, especially for diseases affecting the brain and spinal cord, where gene therapy is surpassing previous therapeutic limitations. 5. Data Sharing and Collaborative Initiatives: - The critical role of data sharing in successful translational drug development for rare diseases is emphasized, highlighting the collective endeavor in advancing gene-targeted therapies. The exploration of gene therapy's potential and the tangible strides made in 2023 highlight a hopeful trajectory for alleviating the burden of rare diseases. To read scholarly articles about this subject, visit us at www.opalbiopharma.com. Opal Bio Pharma (OBP) #OBP #Oman #Muscat #FDA #Opalbiopharma #Khazaen #CDC #Immunotherapy #Immunology #vaccine #medicine #genetherapy #worldhealthorganization #GeneTherapy #RareDiseases #ClinicalTrials #GeneEditing #GeneticMedicine #TherapeuticAdvancements #ResearchAndDevelopment #2023Advancements

ANEW MEDICAL to Advance Patented Klotho Gene Therapy for Neurodegenerative Disorder Treatments Genomics data suggests having an active Klotho gene sequence may help people live longer, healthier lives The Company was granted patents in Europe, China, and Hong Kong and awaits issuance of patents in the U.S. and Canada NEW YORK, July 09, 2024 (GLOBE NEWSWIRE) -- ANEW MEDICAL, INC. (NASDAQ: WENA) (“ANEW”), a biopharmaceutical company specializing in the advancement of novel disease-modifying therapies for neurological and age-related disorders, announces plans to advance its recently patented Klotho gene therapy program for neurodegenerative disorders. ANEW has compelling initial data suggesting that maintaining elevated levels of Klotho in the body significantly contributes to longer, healthier lifespans. Conversely, the data reveals that individuals with depleted or lower-than-normal levels of Klotho are more susceptible to neurodegenerative disorders such as ALS, Alzheimer’s, and Parkinson’s Disease. ANEW will also investigate other age-related disorders associated with depleted Klotho levels, such as memory loss, osteoporosis, sarcopenia (muscle wasting), diabetes mellitus, and certain cardiovascular conditions. Dr. Joseph Sinkule, CEO and Founder of ANEW MEDICAL, commented: “Administering a medication based on the Klotho isoform of interest, secreted Klotho (s-KL), may contribute to longer, healthier lives. Research by our strategic partner, the Okinawa Research Center for Longevity Science (ORCLS) in Japan, suggests that the Klotho gene is a key factor in age-related longevity. Klotho emerged as one of the two most significant genes from their studies, and we look forward to continuing our research with ORCLS as we advance ANEW’s clinical development programs for Neurodegenerative Disorders.” ANEW has development plans to advance its program in three distinct areas: FDA Clinical Trials: The development of therapeutics to treat neurodegenerative diseases and age-related disorders involves using an adenovirus (AAV) vector to deliver the secreted Klotho gene DNA sequence to neurons in the central nervous system (CNS). This approach aims to correct deficiencies in the levels of s-KL. Klotho Diagnostics: The development and commercialization of a diagnostic that will enable healthcare providers to accurately quantify levels of Klotho gene and protein isoforms in blood samples. This tool will also provide ANEW with the necessary diagnostic to screen patients for its clinical trials. Age-Related Disorders: ANEW plans to develop exploratory approaches to promote longevity in healthy populations using gene or cell-based Klotho therapies, as well as other genes of interest that are directly related to longevity.

UMass Chan licenses gene therapy technologies for retinal diseases to Iveric Bio UMass Chan Medical School licensed the exclusive global rights to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of Stargardt disease and Leber congenital amaurosis type 10 (LCA10) to Iveric Bio, An Astellas Company.  While recent forays into gene therapy treatments for other forms of inherited retinal disease have been encouraging, the relatively large size of both the LCA10 and Stargardt genes have been a roadblock to using AAV vectors for treatment. In 2017, Hemant Khanna, PhD, then an associate professor of ophthalmology & visual sciences at UMass Chan, and now vice president of preclinical ocular research at Iveric Bio, developed a “minigene” approach to deliver a shorter version of the gene using clinically standard AAV viral vectors.  This minigene approach allows scientists to deliver an abridged version of the mutated gene to patients using AAV viral vectors as delivery vehicles while still generating clinical results regardless of where the specific mutation occurs in the gene.   “We are excited to be partnering on this minigene approach to treating inherited retinal disorders using standard AAV vectors,” said Parth Chakrabarti, MBA, executive vice chancellor for innovation and business development at UMass Chan. “As part of the Astellas family of companies, Iveric Bio is uniquely positioned to take this research to the next stage of development.” https://lnkd.in/g8uEiQdR

Cell and Gene Therapy: Transforming the Tapestry of Modern Medicine 🧬 For millennia, humanity has been on an unyielding quest for medical solutions, striving to combat the myriad ailments that afflict us. But few fields have proven as transformative and full of promise as cell and gene therapy. Once relegated to the annals of speculative science fiction, these therapies have evolved into tangible lifelines, ushering in a new paradigm of personalized medicine. With every breakthrough, we’ve turned pages of despair into chapters of hope, fundamentally altering our understanding of what’s possible in treating genetic and complex diseases. The Timeline: 2001-2003: Human Genome Project wraps up, casting a spotlight on our genetic intricacies. 2004-2009: The burgeoning field sees Provenge, the first approved cancer vaccine for prostate cancer, heralding the potential of personalized medicine. 2010-2013: 2012: Glybera, a gene therapy for LPLD, breaks ground in Europe. 2013: CRISPR-Cas9 emerges, revolutionizing genome editing. 2014-2016: Imlygic is approved for melanoma, marking a milestone for oncolytic virus therapies. CAR-T cell therapies gain traction. 2016: Strimvelis, for ‘bubble boy’ disease, gets the nod in the UK. 2017: Kymriah and Yescarta, pioneering CAR-T therapies, are endorsed by the FDA for specific blood cancers. Axicabtagene ciloleucel (Yescarta) opens avenues for non-Hodgkin lymphoma patients. 2018: Luxturna offers a ray of hope for inherited retinal diseases. Zolgensma provides a lifeline for spinal muscular atrophy patients. 2019: Tecartus gets approved, expanding CAR-T therapy options for relapsed or refractory mantle cell lymphoma. Zynteglo gets European green light for beta-thalassemia. 2020-2021: Breyanzi, another CAR-T therapy, is approved for certain types of large B-cell lymphoma. Libmeldy becomes the first gene therapy approved for metachromatic leukodystrophy in the EU. 2022: The dynamic market is projected to breach the $10 billion barrier. 2023: Approval of gene therapy for Duchenne Muscular Dystrophy (DMD), altering the trajectory for many affected families. OriVax, a promising gene therapy, offers new avenues for rare inherited neurological disorders. Reflections on the Journey Ahead: Standing at the crossroads of innovation and application, the chronicle of cell and gene therapy is a testament to human ingenuity and the indomitable spirit to heal. As we retrospect, it’s not merely the scientific milestones that leave us in awe, but the stories of countless lives transformed, of families rekindled with hope, and of communities emboldened by the promise of a healthier future. As this journey unfolds, our collective endeavor will be to ensure that these therapies become accessible to all, transcending barriers and frontiers. As we peer into the horizon, it is clear that the confluence of passion, science, and technology will lead us to a dawn where no genetic ailment remains unconquered.

The treatment of sickle cell disease is set to transform with the upcoming US approval decisions for two gene therapies this December, but access to the potentially curative treatments remains an ongoing question. Although there are approved treatments for sickle cell disease, the condition remains a devastating disease, says Dr. Mitch Weiss, chair of the haematology department at the St. Jude Children’s Research Hospital, Memphis, Tennessee. Advances over the last 30 years mean that most US-based patients reach the age of 18, when earlier a substantial number of children passed away from the condition, says Weiss.  However, many sickle cell disease patients still have recurring pain episodes and progressive organ damage, requiring them to spend a large part of their lives in the hospital, he says. These problems may be circumvented by emerging gene therapy approaches, adds Weiss. Notably, while bone marrow transplantation is available as the only potentially curative option, it works only for about 20% of patients. New treatments are also needed that can improve a patient’s quality of life, and help the community determine who needs expensive gene therapies the most. New approaches include gene therapies and treatments like Agios Pharmaceuticals’ pyruvate kinase activator Pyrukynd (mitapivat). Sickle cell disease is a genetic disease caused by mutated copies of the β-globin gene where high concentrations of sickle haemoglobin cause red blood cells to sickle (deform). Sickled cells often die prematurely, resulting in haemolytic anaemia, vasculopathy, and vaso-occlusion. Patients often receive blood transfusions alongside recently approved treatments that target the sickling of red blood cells and vaso-occlusive crises. That includes Pfizer’s Oxbryta (voxelotor) and Novartis’smonoclonal antibody Adakveo (crizanlizumab).  Now, the sickle cell disease community is closely watching the potential FDA approval of two gene therapies with different mechanisms. Vertex Pharmaceuticalsand CRISPR Therapeuticshave a PDUFA date of December 8 for exagamglogene autotemcel (exa-cel), while bluebird bio’s gene therapy lovotibeglogene autotemcel (lovo-cel) has a PDUFA date of December 20.  Lovo-cel is a one-time gene therapy that adds working copies of a modified form of the β-globin gene into hematopoietic stem cells. This produces anti-sickling haemoglobin, which reduces the proportion of sickle hemoglobin that causes red blood cells to sickle at high concentrations.  Exa-cel is a CRISPR/Cas9 gene-edited therapy where hematopoietic stem cells are edited to produce higher levels of foetal haemoglobin. Depending on the FDA’s verdict, exa-cel could be the first approved CRISPR-based gene therapy. Read more: https://lnkd.in/enS4eKj6

The promise of gene therapies It was exciting to see the news recently that the first patient – a 12-year-old boy – has begun a new gene therapy for sickle cell disease. It’s one of two recent gene therapies for this disease approved by the U.S. Food and Drug Administration. Both treatments target the underlying cause of the disease – a faulty gene that the patient has inherited from their parents. Sickle cell disease is a debilitating and life-threatening blood disorder that affects about 100,000 people in the United States, alone, and these treatments have the potential to transform patients’ lives. Since 1990, when the first gene therapy was approved for a severe immunodeficiency disease, others have emerged for Duchenne muscular dystrophy, hemophilia and a retinal disorder that causes blindness, among other hereditary diseases. I’m excited to see the pace of discovery is accelerating. While most of the progress has been in the realm of rare inherited diseases, gene therapy techniques – like CRSPR gene editing and lentiviral vector delivery – are being applied to development of potential treatments for various forms of cancer and other more common illnesses – even ways to address aging. It’s an incredibly promising frontier. There are still major hurdles to overcome as more gene therapies emerge: treatment can be long and grueling for the patient, the therapies are costly and availability can be limited. That’s why Thermo Fisher is committed to doing everything we can to help our customers bring new treatments to market faster and with lower cost, so we can improve lives worldwide. Our enabling technologies, services and expertise advance research, cutting-edge drug discovery and development, and our capabilities to support clinical trials and manufacture new therapies at scale help our customers accelerate development and commercialization. We continue to invest in these capabilities so we can help our customers move their breakthroughs forward and reach patients faster. Thank you to all of our colleagues for the passion they bring to this work, and congratulations to our customers and all the innovators who are bringing new hope to patients everywhere.