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The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia A custom-made, one-dose genetherapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.
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.
Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.
Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. 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.
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.
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”
Gong Chen at Jinan University, Guangzhou, China recently reported the first non-human primate study demonstrating successful in vivo neural regeneration from brain internal glial cells for stroke repair. Credit: Jinan University Stroke is a leading cause of death and severe long-term disability with limited treatment available.
These programmes will include therapies and vaccines in infectious disease and oncology areas. oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. .
Takeda oncology drug discovery unit head Kathy Seidl stated: “We are encouraged by KSQ’s CRISPRomics platform and its ability to perform in vivogenetic screens for the discovery and validation of tumour targets, which have the potential to modulate the innate and adaptive immune system. “We
Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivogene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). The company will receive a combined upfront payment and equity investment of $60m.
Novartis has shouldered its way into the in vivogene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivogene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4
Deloitte goes on to say “while biologics generally target smaller populations, “next-gen” therapies often target individual patients—sometimes as single treatments—resulting in truly personalized medicine. Still, she is missing a lot of facts at a time when hospital and health insurance costs are soaring. percent from 2018.
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.
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.
LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and genetherapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.
Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivogenetherapies, and 2 gene-modified cell therapies.
Bayer has bolstered its cell and genetherapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.
Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio genetherapy eradicates toxic substrate in Fabry patient.
The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.
This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease. Below are some of the key trends that will be shaping the future of the life sciences in 2024 and beyond. The technologies enable the analysis of vast amounts of biological data at speeds and accuracies previously unimaginable.
The American Society of Gene and Cell Therapy (ASGCT) defines cell therapy as a therapeutic modality that involves the administration of either normal or modified cells to patients for the treatment of various diseases. The major steps involved in cell therapy manufacturing have been briefly outlined below.
SVP, Chief Medical Officer, Novartis GeneTherapies. “We SVP, Chief Medical Officer, Novartis GeneTherapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of genetherapy to treat SMA. 2 years and <5 years old with SMA Type 2.
Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2
Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.
The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Bluebird spins off to two companies, cleaving off its genetherapy, and cancer units. Further, Birinapant also complements the anti-tumor activity of the immune system.
Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.
A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. They are due to enrol 45 apiece, and are scheduled to readout next year.
“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies.
Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. An expected and well-known side effect of these B cell-targeted therapies is “B cell aplasia”— i.e. partial or complete depletion of B cells from circulation and immune organs.
PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 genetherapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).
In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.
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.
Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. Usually, only a single copy of the gene is required that too from the mother, however, in cases of AS, either the child does not get any copy, or the child receives two copies – each from mother and father.
The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. Axovant – Kristin Vuori was named to the board of directors at Axovant GeneTherapies Ltd. BeyondSpring – BeyondSpring Inc. Additionally, Parag V. At Spark, he served as head of U.S.
Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.
Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.
Moreover, the exuberant development of biologics has revolutionized the treatment of a range of therapeutic conditions, which has further contributed to the exponential growth in the current demand for biologic therapies developed by biologics manufacturing companies.
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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