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By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and genetherapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.
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.
Bluebird bio’s genetherapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three genetherapies in its pipeline.
Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and genetherapy product approvals.
The US Food and Drug Administration (FDA) has approved the first genetherapies for the treatment of sickle cell disease, approving two on the same day. Both genetherapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.
Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered genetherapies. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.
After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based genetherapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.
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 vivotherapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.
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.
Lifesciences tools company Esco Lifesciences Group has scored $200m in a series A and crossover round co-led by Novo Holdings and Vivo Capital. Funds will be used to expand the company’s presence across Asia and create an innovation hub in Boston for cell and genetherapy technologies.
To achieve this, the agenda will allow delegates to: Explore the translation of BBB-penetrating antibodies to non-human primates , and new possibilities for in vivo brain imaging of antibody neuropharamacokinetics. A Meeting Point for the Field’s Experts. 3-day Conference | June 28-30, 2021. About Hanson Wade.
According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. The rapidly expanding landscape of cell therapy trials has put an operational strain on many clinical sites due to limited resources and infrastructure. “In
The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and genetherapy and others. A solution could be at hand with Overland Pharmaceuticals.
Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.
This model has the benefit of giving companies direct oversight of their in vivo studies, allowing them to move faster while operationalizing every dollar of their seed financing. Photo courtesy of Mispro. There’s an emerging business model in biotech town, and it’s called the Contract Vivarium (CV).
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. Additionally, Parag V.
Since the activity of TILs can be suppressed by tumor cells, TIL therapies are designed to bolster the number of TILs. Related: FDA Issues Letters to CAR T-Cell Therapy Makers to Include Boxed Warning Amtagvi was approved through the FDA’s Accelerated Approval pathway. TILs are immune cells that attack and penetrate tumors.
Poseida’s expertise in donor-derived, allogeneic CAR-T therapies offers a transformative edge. The acquisition also grants Roche access to Poseida’s P-CD19CD20-ALLO1, a dual-target CAR-T therapy currently in Phase I trials for B-cell malignancies. Meanwhile, Novartis acquired Kate Therapeutics in a $1.1
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