Voting yes on Prop 14 means supporting stem cell research through a $5.5 billion state bond. Its a loan that California, through its taxpayer-supported general fund, would have to pay back with interest over 30 years. Prop 14 would extend funding that has almost run out for the states stem cell research institute, which was created with the approval of California voters in 2004 after President George W.
PITTSBURGH, Oct. 16, 2020 (GLOBE NEWSWIRE) -- Those who are looking for an alternative treatment option when compared to traditional medicine are now in luck
DetailsCategory: DNA RNA and CellsPublished on Friday, 16 October 2020 14:20Hits: 473 First therapy recommended for full marketing authorization in the EU for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants One-time treatment with Libmeldy has been shown to preserve cognitive and motor function in most patients Libmeldy is backed by data across 35 patients with follow-up of up to 8 years post-treatment, demonstrating the potential durability of HSC gene therapy BOSTON, MA, USA and LONDON, UK I October 16, 2020 I Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA, gene), an investigational gene therapy for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. The CHMPs positive opinion will now be reviewed by theEuropean Commission(EC), which has the authority to grant marketing authorization for Libmeldy in theEuropean Union(EU). A final decision by the EC for Libmeldy is anticipated before the end of 2020
Building on the transformative impetus from the first Food and Drug Administration (FDA)-approved Janus kinase (JAK) 1/2 inhibitor, ruxolitinib (Jakafi), in the clinical landscape of myeloproliferative neoplasms (MPNs), we are entering a new era of multiple JAK inhibitors and other diverse classes of drugs in rapid clinical development. Advancements in elucidating the pathophysiology of MPNs have spurred significant progress in developing novel promising agents or combination regimens with ruxolitinib to treat patients who are unresponsive to standard treatments or have specific clinical needs. In myelofibrosis (MF), the most aggressive MPN, with an average survival of 5 to 7 years, abnormal clonal hematopoietic stem cell proliferation in the bone marrow (BM) leads to liberation of pro-inflammatory cytokines and extensive fibrosis, causing progressive pancytopenia, especially anemia and thrombocytopenia, along with splenomegaly and other symptoms, compromising quality of life.1 For nearly a decade, ruxolitinib has been the centerpiece therapy for patients with MF, markedly improving splenomegaly and constitutional symptoms and providing survival benefit.2 The second FDA-approved JAK2 inhibitor, fedratinib (Inrebic), may actually be a good second-line option for patients who are ruxolitinib-resistant with intermediate-2 and high-risk MF (primarily thrombocytopenic and characterized by platelet counts 50100 109/L).3 At present, 2 ongoing phase 3 clinical trials, the single-arm FREEDOM trial (NCT03755518) and the double-arm FREEDOM 2 trial (NCT03952039), are assessing the efficacy and safety of fedratinib in patients with MF who are resistant/refractory/intolerant to ruxolitinib
SAN DIEGO, Oct. 12, 2020 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc.
Israeli innovator and designer Dror Benshetrit once said, Thinking outside the box means crossing disciplines and pulling in expertise and perspective from outside of the standard boundaries. Thats a concept that Dr.
New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Regenerative Medicine Market by Product, Application, Geography - Global Forecast to 2025" - https://www.reportlinker.com/p04700208/?utm_source=GNW However, the high cost of cell and gene therapies and ethical concerns related to the use of embryonic stem cells in research and development are expected to restrain the growth of this market during the forecast period.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market, by product, during the forecast periodBased on products, the regenerative medicine market is segmented into tissue-engineered products, cell therapies, gene therapies, and progenitor and stem cell therapies.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market in 2019. The increasing adoption of tissue-engineered products for the treatment of chronic wounds and musculoskeletal disorders and the rising funding for the R&D of regenerative medicine products and therapies are the major factors driving the growth of this segment
Last October, the FDA OKd a semi-controversial new antibiotic from Shionogi. In one of two large studies for the drug, known chemically as cefiderocol and commercially as Fetroja, more patients died in the treatment arm than the control arm
The global blood and bone marrow cancer treatment market was valued at $38.8B (approx 32.8B) in 2018 and is reportedly expected to reach $74.9B (approx 63.4B) by 2027, expanding at a CAGR of 7.7% from 2019 to 2027. Blood cancer begins in the bone marrow which is the integral source of stem cells, which are later differentiated into different types of blood cells in the human body. Researchers have stated that approximately 1.85 million new cases of blood cancer will be diagnosed by 2040 throughout the globe
The only curative approach to treating myelofibrosis (MF) at this time is hematopoietic stem cell transplant, but the treatment landscape for this patient population continues to expand, particularly since the identification of the JAK-STAT pathway as a potential target in 2005.
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