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Astounding progress in the treatment of Sickle Cell Anemia! With the new treatment, exa-cel, we’re seeing life-changing results. Out of 44 patients, 97% remained free from vaso-occlusive crises for over a year, and all avoided hospitalizations for these crises. Kevin Davies, when is your book about these SCA patients and research coming out?! I can’t wait to read it. You guys, this a pivotal moment in medical history, demonstrating the power of innovative treatments to dramatically improve quality of life. Up next, CRISPR is expected to tackle the CFTR gene responsible for cystic fibrosis, probably Hereditary Blindness targeting Leber congenital amaurosis, a genetic disorder that causes severe vision loss at birth. The future looks so hopeful as we continue pushing the boundaries of what’s possible in medicine. In ten years, will technologies like this let us attack MDS/AML, or even mutation-driven solid tumors?! #CRISPR #EditingHumanity #SickleCellTreatment #MedicalInnovation #ExaCel Mary Ann Liebert, Inc. CRISPR Medicine News The CRISPR Journal Damian Doherty Inside Precision Medicine Jaime Grund Jody Wallace, MS, MBA, CGC

Unveiling the future of lupus treatment! This insightful article explores the incredible strides being made in CAR-T cell therapy in autoimmune disease. #BiotechInnovation  https://lnkd.in/eV_ZpscK

Investigation of long term effects for any type of new treatments, especially involving #ATMPs is critical, #cellandgenetherapies are complex, relatively new and come with many challenges on the way, #FDA actions are totally understandable, it’s necessary to keep learning and to make any type of therapies even safer and more standardised for future generations

Solid first step of "off-the-shelf" stem cell derived product for Parkinson's disease therapy. Keep patient, keep curious and keep deep-thinking

In the age of cell and gene therapies, how much of an impact will J&J's CAR T-Cell therapy have in multiple myeloma? Read below 👇

What’s new in the #cellandgenetherapy sector? Check out our first weekly news roundup of the year. ✅ FDA approves Casgevy to treat transfusion-dependent beta thalassemia ⏩ CAR-T to treat refractory progressive multiple sclerosis granted FDA Fast Track Designation 🏥 First patient in Phase 1 trial of CAR-T to treat B-ALL reached complete remission Read the full stories below, and subscribe to our Sector Vector newsletter for more weekly cell and gene therapy clinical, business, and manufacturing news: https://lnkd.in/eD7ZNaU Vertex Pharmaceuticals and CRISPR Therapeutics announced that Casgevy, the first-ever #CRISPR-based therapy approved by the FDA, received an expanded approval this week to treat TDT. The FDA approved it in December for severe sickle cell disease. https://lnkd.in/gK554kzK Kyverna Therapeutics granted FDA Fast Track Designation for CAR-T therapy to treat patients with refractory progressive multiple sclerosis. https://lnkd.in/ewf3b2xs Pepromene Bio, Inc. announces complete remission of the Cohort 1 first patient treated for relapsed and refractory B-cell acute lymphoblastic leukemia (B-ALL) in the Phase 1 clinical trial of CAR-T cell therapy. https://lnkd.in/et8DnyRb

CAR-T cell therapy is an example of an innovative treatment that can help improve lives. In recent news, a CAR-T cell therapy is being tested for the potential treatment of autoimmune diseases, and the results are positive so far. Read the Fierce Biotech article for more, and let us know in the comments ⬇ what other innovations you think we will see on the rise in our industry. Read the article: https://lnkd.in/erWcyBB5 #CellTherapy #CARTCellTherapy #ClinicalResearch #ClinicalTrials

Wugen has dosed the first patient in a Phase I clinical trial of natural killer (NK) cell therapy product WU-NK-101 to treat relapsed or refractory (r/r) acute myelogenous #leukaemia (AML). The global, open-label, dose-escalation and cohort expansion study will assess the pharmacokinetics, pharmacodynamics, tolerability, safety, immunogenicity, and preliminary anti-leukemic activity of WU-NK-101. #ClinicalTrials 

The excitement around CAR-T #celltherapy is undeniable. In her latest guest column in Cell & Gene, our President and CEO, Kristin Yarema, Ph.D., looks at different ways that clinical trial data can be presented, and why it is important for both autologous and allogeneic CAR-T companies to present results through the lens of the entire treatable patient population – and why a so-called intent-to-treat analysis is important to understand the real benefit and value of these various cell therapy approaches for patients, physicians, and the #healthcare system. https://lnkd.in/dWwD9JUh Erin Harris #CapacitytoCure

T cells are essential in the adaptive immune response, and there is particular interest in genetically engineering these cells for therapy against cancer and other diseases. STEMCELL Technologies provides tools for a complete #Tcell workflow—from cell sourcing and isolation, to gene editing, expansion, and analysis—helping you move your research forward with confidence. #Immunology #CellTherapy https://lnkd.in/gBUbWSYP