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As we step into 2024, the lifesciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables.
Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.
The ability to modify or introduce genetic material in human cells in such a precise and patient-centered manner clearly constitutes a breakthrough in personalized medicine. CGT drugs fall into two major categories: autologous and allogeneic. What are the general differences in the supply chain of CGT vs. traditional clinical trials?
By reducing the disease to its key components, a computer-readable network of disease markers was characterised using genetic, biomarker, and clinical data; the model was also populated with ‘disease vs control’ data. Disease modelling Gulf War Illness. Tinker, tailor, soldier, therapy.
Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.
Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Rare diseases can often be progressive, chronic and fatal. Sadly, one-third of children with rare diseases die before their first birthday.
The recurrence of these events, or crises, can lead to life-threatening disabilities and/or early death. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.
This typically involves taking T cells from the body, engineering chimeric receptor antigens (CAR) and inserting them into the cells, expanding the cells ex vivo and injecting them back into the body. Cell-Based Immunotherapy vs. Immune Biologics. Immunotherapy involves leveraging components of the immune system (i.e.
The creation of Overland isn’t the only exciting thing happening for international lifesciences companies and organizations. A solution could be at hand with Overland Pharmaceuticals. Overland aims to build a leading, fully integrated biopharma company by leveraging global networks and formative R&D partnerships.
In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.
Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.
Assembly Biosciences – Former Gilead Sciences CEO John McHutchison, currently president and CEO of Assembly, was named the recipient of the Advance 2020 Global Impact award. The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. BeyondSpring – BeyondSpring Inc.
CRISPR works as genetic scissors to edit parts of the genome. Related: Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug The advisors appeared convinced that exa-cel can offer significant benefits to people with sickle cell. I think in this case, there is a huge unmet need.”
A life-changing moment came for Dr. Leaving academia for industry is a route that academic scientists and professors did not traditionally pursue in the past due to perceived differences in skillsets, experiences and even associated stigmas. The journey first starts with the decision to make the switch. Key Moments.
Yesterday, the biotechnology company Capstan Therapeutics launched with $165 million in seed and Series A funding for it to build on its foundational insights into precision in vivo engineering technology. Our ambition at Capstan is to invent new clinical paradigms through targeted in vivo reprogramming of cells.
Kristin Yarema, President of Cell Therapy at Poseida Therapeutics – Xtalks LifeScience Podcast Ep. Poseida’s genetic engineering platforms stand out for their precision, safety and potential to open new therapeutic avenues. Poseida’s expertise in donor-derived, allogeneic CAR-T therapies offers a transformative edge.
Zenocutuzumab-zbco is a first-in-class bispecific antibody that targets NRG1 fusions, a rare genetic driver implicated in several tumor types. Zenocutuzumab-zbco is a first-in-class bispecific antibody that targets NRG1 fusions, a rare genetic driver implicated in several tumor types. percent of NSCLC and 0.3 months to 16.6
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