XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. Previous animal model studies of Friedreich’s ataxia showed a dysfunction in the regulation of Nrf2 signaling, a pathway associated with reduced inflammatory response and improved mitochondrial function.

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pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The post FDA approves Alnylam’s ultra-rare disease drug Oxlumo appeared first on.

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BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

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Gene Therapy Gene FDA Approval Genetics 105

The Pharma Data

NOVEMBER 21, 2021

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth. Source link: [link].

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Pharmaceutical Technology

AUGUST 16, 2022

antibody, ADX-914 re-regulates adaptive immune function by hindering IL-7 and TSLP-facilitated signalling. In July, the US Food and Drug Administration (FDA) approved Horizon’s supplemental Biologics License Application for Krystexxa (pegloticase) plus methotrexate, to aid uncontrolled gout patients attain a complete response to treatment.

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Development Clinical Trials Clinical Trials FDA Approval 130

The Pharma Data

AUGUST 13, 2020

NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy. The drug was trialled in two studies of a total of 32 male participants with genetically confirmed DMD.

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FDA Law Blog

JULY 8, 2021

Javitt — For many years, one of the most controversial topics in device regulation has been the dual-track oversight of in vitro diagnostics (IVDs) and laboratory developed tests (LDTs). At various points and in various ways, FDA has sought – mostly unsuccessfully – to regulate LDTs (see here ).

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

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FDA Approval Genetic Disease Genetics Clinical Trials 52

XTalks

SEPTEMBER 15, 2022

According to the order, it will improve and expand biomanufacturing production capacity and processes, train a biotech workforce, expand market opportunities for these products and streamline regulation. Related: Top Food Industry Regulations to Watch in 2022. Here’s everything you need to know.

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XTalks

FEBRUARY 5, 2024

Revlimid (Lenalidomide) Revlimid 2022 sales: $9.978 billion Company/Developer: Bristol Myers Squibb Date of first FDA approval: December 27, 2005 Indications Revlimid is FDA-approved for: Myelodysplastic disease (MDS), multiple myeloma, mantle cell lymphoma (MCL), follicular lymphoma and marginal zone lymphoma.

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Drug Discovery World

SEPTEMBER 23, 2022

Study reveals first genetic links in ME and Chronic Fatigue Syndrome. A new study has provided the first detailed genetic insights into the pathophysiological mechanisms underpinning Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). FDA approves gene therapy for rare neurological disorder.

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pharmaphorum

AUGUST 13, 2020

The FDA controversially approved Vyondys 52 late last year after a previous rejection and an appeal from Sarepta. Viltepso was evaluated in two clinical studies with a total of 32 patients, all of whom were male and had genetically confirmed DMD. The post NS Pharma takes on Sarepta as FDA approves DMD drug appeared first on.

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XTalks

DECEMBER 28, 2023

XTALKS WEBINAR: Impacts of the New IVD Regulation (IVDR) for Manufacturers and Users Live and On-Demand: Tuesday, January 9, 2024, at 10am EST (4pm CET/EU-Central) Register for this free webinar to learn how the new In Vitro Diagnostic Regulation (IVDR) impacts manufacturers and users of diagnostic devices.

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XTalks

FEBRUARY 5, 2024

Revlimid (Lenalidomide) Revlimid 2022 sales: $9.978 billion Company/Developer: Bristol Myers Squibb Date of first FDA approval: December 27, 2005 Indications Revlimid is FDA-approved for: Myelodysplastic disease (MDS), multiple myeloma, mantle cell lymphoma (MCL), follicular lymphoma and marginal zone lymphoma.

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The Pharma Data

NOVEMBER 23, 2021

The agency also is responsible for the safety and security of our nation’s food force, cosmetics, salutary supplements, products that give off electronic radiation, and for regulating tobacco products. Livtencity works by preventing the activity of human cytomegalovirus enzyme pUL97, thus blocking virus replication. Source link: [link].

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Drug Discovery World

APRIL 4, 2023

Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February. This made Jesduvroq is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) to be approved in the US.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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Roots Analysis

APRIL 28, 2024

Factors Contributing to Kidney Diseases There are several factors which contribute to an increased risk of developing kidney diseases, including genetic causes and underlying metabolic causes ( diabetes, high blood pressure, aging and obesity ), which when combined with other factors increase the risk of developing kidney diseases.

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XTalks

NOVEMBER 20, 2023

The newly approved Truqap and Faslodex combo is restricted to patients with a genetic alteration in one or more of the following PI3K/AKT pathway genes: PIK3CA , AKT1 or PTEN. PI3K/AKT is a key intracellular signaling pathway that regulates cell survival and proliferation. It was also given FDA priority review.

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Drug Discovery World

MAY 21, 2024

Modern experimental procedures, such as immunisation, B-cell screening, and synthetic library generation, have been pivotal in developing approximately 80 FDA-approved antibody therapeutics. Existing antibody discovery methods have mostly failed for certain target groups, such as G protein-coupled receptors (GPCRs) and ion channels.

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WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

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In-Vivo Clinical Trials Clinical Trials FDA Approval 52

XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s Accelerated FDA Approval Modeled on Aduhelm. This has brought criticism that US regulators are moving too slow on a drug that has clinical promise and is being backed by other international regulators.

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XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

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Gene Therapy Gene Protein FDA Approval 98

Advarra

JANUARY 11, 2024

Personalized medicine using genetic resequencing techniques, such as cell and gene therapies, enables researchers to create more customized therapies for combatting cancer. Many monoclonal antibodies are used to treat cancer by targeting specific cell structures regulating cell activity, causing the death of tumor cells.

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Pharmaceutical Technology

MARCH 6, 2023

However, struggles to prove to regulators the validity of or need for drugs in this realm still serve as a major barrier to success. Currently, most drugs being investigated for their healthy aging or anti-aging properties are being repurposed from readily accessible FDA-approved drugs.

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pharmaphorum

NOVEMBER 27, 2020

A new EMA approval has expanded the use of Vertex Pharma’s exon-skipping cystic fibrosis therapy Symkevi to children as young as six if they have specific gene mutations. Symkevi has been an option for CF patients in this group aged 12 or over for two years, but the new approval means treatment will now be able to begin much earlier.

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FDA Law Blog

MARCH 3, 2024

The Act is intended to address national security concerns by prohibiting certain conduct by regulated industry. Effect on FDA approvals— In cases where a biological product or drug needs to change aspects of its manufacturing processes to avoid using a covered equipment or service, will it need to file supplements with FDA for the CMC update?

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Drug Discovery World

NOVEMBER 14, 2023

A very rare, inherited blood clotting disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

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FDA Approval Gene Clinical Trials Clinical Trials 52

pharmaphorum

FEBRUARY 11, 2022

The subject from the initial study of RGX-111 that led to FDA approval of the phase 1/2 trial also showed better-tan-expected improvements on those skills over a follow-up period of around 20 months. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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The Pharma Data

JULY 6, 2021

PGRN is a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, making it one of the most attractive genetically validated targets for the development of new immuno-neurology treatments. ” Arnon Rosenthal, Ph.D.,

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Genetics Gene Antibody Development 52

The Pharma Data

JANUARY 21, 2021

INSTIs, like cabotegravir, inhibit HIV replication by preventing the viral DNA from integrating into the genetic material of human immune cells (T-cells). This step is essential in the HIV replication cycle and is also responsible for establishing chronic infection.

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FDA Approval RNA Drugs Containment 52

The Pharma Data

SEPTEMBER 7, 2020

The personalised cancer treatment has received conditional approval in the US. US regulators have approved Genentech’s Gavreto (pralsetinib) for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC). Source link.

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FDA Approval Genome Genomics Genetics 52

Drug Discovery World

OCTOBER 18, 2023

What’s more, they are expensive, not readily available, and in many countries, there are a host of stringent regulations, as well as ethical and legal considerations around their use. Mouse models, on the other hand, are cost-effective and readily available, making them a favourite for many researchers. Accessed 26 Jul 23.

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Pharmaceutical Technology

APRIL 5, 2023

It will be six years since the US FDA approval of Kymriah in August this year. Bruce Levine [BL]: We now have six approved CAR-T therapies. But I think we’ve never had more tools than we have now in synthetic biology, genetic engineering, enhanced potency design switches and CRISPR. One challenge is in potency.

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XTalks

AUGUST 6, 2020

The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant.

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