XTalks

DECEMBER 27, 2022

Ferring Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

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Gene Therapy FDA Approval Gene Clinical Trials 52

XTalks

FEBRUARY 6, 2023

GlaxoSmithKline LLC (GSK) recently announced that the US Food and Drug Administration (FDA) approved Jesduvroq (daprodustat), a new once-a-day oral treatment for anemia due to chronic kidney disease (CKD). The approval is currently only for adult patients who have been undergoing dialysis for at least four months.

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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.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Brukinsa is now approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). million price tag of Elevidys, a one-time gene therapy. Options for managing the symptoms of DMD have been limited.

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Gene Therapy Gene Protein FDA Approval 98

XTalks

NOVEMBER 7, 2022

Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. Liquid Biopsy Use in Oncology Clinical Trials. Regulatory Approvals for CTC-Based Assays in Oncology. These tumor-derived entities are used to derive genomic and proteomic data.

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Clinical Trials Clinical Trials Trials FDA Approval 98

XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. It is caused by a mutation in the FXN gene , which is responsible for the production of the protein frataxin. Skyclarys was approved based on the results of the MOXIe Part 2 clinical trial.

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XTalks

JUNE 2, 2021

KRAS mutations have previously been considered ‘undruggable’ targets, but they have been a big focus in cancer research as they are one of the most common gene mutations across all cancers. The sotorasib approval is therefore a major breakthrough in the world of cancer and targeted therapies.

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XTalks

MAY 19, 2022

Mitsubishi Tanabe Pharma America last week announced US Food and Drug Administration (FDA) approval of RADICAVA ORS® (edaravone), a medication for patients with amyotrophic lateral sclerosis (ALS). RADICAVA ORS and RADICAVA are indicated to treat ALS, with clinical trials demonstrating that RADICAVA slows loss of physical function.

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XTalks

JUNE 17, 2022

Eli Lilly’s Olumiant (baricitinib) has won US Food and Drug Administration (FDA) approval as the first systemic treatment for severe alopecia areata (AA), an autoimmune disorder that leads to patchy baldness. The Olumiant approval is therefore a big win as it helps fulfill a significant unmet need in the area.

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FDA Approval Gene Expression Cosmetics Trials 97

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

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XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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FDA Approval Manufacturing Trials Clinical Trials 52

XTalks

MAY 31, 2022

XTALKS WEBINAR: All Means All: The Road to Inclusivity in Clinical Trials. Register for this free webinar to learn what the FDA’s new draft guidance means for diverse and inclusive trials. Clinical Trials of Vtama. Live and On-Demand: Tuesday, July 12, 2022, at 11am EDT (4pm BST/UK).

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XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

FEBRUARY 27, 2024

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.

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XTalks

APRIL 23, 2021

The therapy is indicated for endometrial cancer that has progressed during, or following, prior treatment with a platinum-based chemotherapy, and in women with dMMR tumors as determined by an FDA-approved test. The immunotherapy has also become the first targeted therapy for the dMMR positive subset of endometrial cancers.

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FDA Approval Trials Antibody DNA 105

XTalks

DECEMBER 10, 2021

Zavegepant is an antibody drug that prevents binding of the calcitonin gene-related peptide (CGRP) to the CGRP receptor. Drugs in this class include Eli Lilly’s Emgality (galcanezumab) and AbbVie’s Qulipta (atogepant), which are approved for the treatment of migraine. The pain relief effects were sustained for up to 48 hours.

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XTalks

JUNE 12, 2023

Recent FDA Approvals of Cell Therapy Manufacturing Facilities in the US Successful commercialization of novel cell therapy requires exemplary performance at all stages, including upstream and downstream processing, process development, testing and manufacturing services. percent during the 2022 to 2030 forecast period.

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XTalks

MAY 24, 2023

Eyecare giant Bausch + Lomb received its first prescription drug approval from the US Food and Drug Administration (FDA) for its dry eye disease med Miebo (perfluorohexyloctane ophthalmic solution). Last year, the FDA approved the first generic version of Restasis.

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FDA Approval Gene Therapy Gene Marketing 98

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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Gene Therapy Gene Protein Trials 110

XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

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Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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XTalks

MARCH 21, 2023

It is estimated that Rett syndrome affects 6,000 to 9,000 patients in the US, with a diagnosed population of approximately 4,500 patients, according to a press release from Acadia announcing Daybue’s approval. However, in animal studies, trofinetide has been shown to increase dendritic branching and synaptic plasticity signals.

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FDA Approval Gene Therapy Gene Genetics 94

XTalks

JUNE 29, 2022

The new FDA approval gives Breyanzi (lisocabtagene maraleucel) the broadest patient eligibility of any CAR T-cell therapy in relapsed or refractory LBCL. XTALKS WEBINAR: Live Cell Metabolic Analysis — Paving the Way for Metabolic Research and Cell & Gene Therapy. Outcomes of Clinical Trials for Breyanzi.

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FDA Approval Gene Therapy Antibody Gene 52

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences 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.

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XTalks

DECEMBER 4, 2023

The FDA said it is investigating the identified risk of developing the T-cell malignancies as secondary cancers with serious outcomes, including hospitalization and death, and also looking at the need for regulatory action. However, the FDA’s investigation has now revealed actual reports of T-cell malignancies linked to the therapies.

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XTalks

OCTOBER 7, 2022

The US Food and Drug Administration (FDA) has granted approval to Taiho Oncology, Inc. About 20 percent of patients diagnosed with CCA have the intrahepatic form of the disease and among them, ten to 16 percent of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation. Taiho Oncology, Inc.

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FDA Approval Gene Trials Drugs 98

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

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XTalks

MARCH 28, 2024

Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks Life Science Podcast Ep. Early clinical trials have shown promising results and this therapy could become a valuable addition towards CRC treatment in the future.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

SEPTEMBER 22, 2022

On September 1, 2022, Boehringer Ingelheim Pharmaceuticals announced in a press release that the US Food and Drug Administration (FDA) approved Spevigo (spesolimab-sbzo) intravenous injections for GPP flares in adults. Spevigo is the first approved treatment option for GPP in adults. Clinical Trial Behind Spevigo.

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FDA Approval Clinical Trials Clinical Trials Dermatology 52

XTalks

SEPTEMBER 12, 2023

Life science podcasts have emerged as an invaluable tool for building connections with audiences in the digital era. Furthermore, we’ll explore the unique advertising opportunities that this platform offers, positioning brands at the forefront of the life science industry. The result?

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Life Science Branding Clinical Trials Clinical Trials 105

XTalks

FEBRUARY 9, 2024

XTALKS WEBINAR: Case Study: EEG Digital Biomarkers in Neuropsychiatric Clinical Studies Live and On-Demand: Thursday, February 15, 2024, at 12pm EST (9am PST) Register for this free webinar to learn how to leverage neuroassessment platforms to advance novel therapeutics focused on neuropsychiatric conditions.

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FDA Approval Clinical Trials Clinical Trials Genetics 64

XTalks

AUGUST 17, 2020

Duchenne muscular dystrophy (DMD) patients with a mutation in exon 53 of the dystrophin gene now have a second approved treatment option, and Xtalks spoke with Sharon Hesterlee, PhD, Muscular Dystrophy Association executive vice president and chief research officer, to learn more.

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pharmaphorum

JUNE 27, 2022

Jennifer Mathieu, director of government relations at the Academy of Managed Care Pharmacy (AMCP), tells us why the organisation backed the pre-approval information exchange (PIE) Act of 2022 and how the legislation will empower companies to share information with healthcare payers and plans during the FDA approval process. .

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XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. How Does Qalsody Work?

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FDA Approval Gene Genetics Protein 89

XTalks

FEBRUARY 3, 2023

Until now, fulvestrant was the only other selective estrogen receptor degrader (SERD) therapy approved for ER+ breast cancer, delivered via intramuscular injections. This approval follows the promising results of the Phase III EMERALD global clinical trial published in the Journal of Clinical Oncology.

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Clinical Trials Clinical Trials FDA Approval Trials 97