XTalks

AUGUST 31, 2022

In this episode, Ayesha discussed the FDA approval of Axsome Therapeutics’ rapid-acting oral treatment Auvelity for the treatment of major depressive disorder (MDD). The approval makes Auvelity the first and only rapid-acting oral medication for depression, and the first and only oral NMDA receptor antagonist approved for MDD.

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XTalks

SEPTEMBER 29, 2022

In this episode, Ayesha discussed the FDA approval of bluebird bio’s Skysona for slowing the progression of neurologic dysfunction associated with the rare neurological disorder cerebral adrenoleukodystrophy (CALD) in boys four to 17 years of age with early, active CALD. 7 that the CDC is closely watching. 5 Omicron variant.

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Gene Therapy Life Science FDA Approval Gene 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

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. As such, rare disease patients and their families often face little hope for effective treatments.

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XTalks

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. Polydefkis, professor of Neurology at Johns Hopkins University School of Medicine and an investigator in the Phase III NEURO-TTRansform study on which Wainua’s FDA approval was grounded on, in the news release.

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XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Brukinsa is a small-molecule inhibitor of Bruton tyrosine kinase (BTK), which is a tyrosine kinase encoded by the BTK gene. BTK is expressed in many B cell malignancies and inhibiting its activity has produced anti-tumor effects.

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

NOVEMBER 30, 2022

In this episode, Ayesha shared news about the FDA approval of the first gene therapy for the rare blood disorder Hemophilia B. Read the full articles here: Hemgenix Approved as First Gene Therapy for Hemophilia B. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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Gene Therapy Life Science Gene FDA Approval 52

XTalks

FEBRUARY 4, 2025

Current Share Price: The announcement follows a $115 million Series D financing in December 2024, co-led by Frazier Life Sciences and Deep Track Capital, with participation from Janus Henderson Investors and Logos Capital. Certain genes have been linked to an increased risk of CKD, with APOL1 being one of the most well-studied.

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Clinical Trials Clinical Trials Trials Genetics 66

The Pharma Data

MAY 17, 2021

In fact, in recent times, tens of billions of dollars have flooded into the Midwest to accelerate life sciences innovation, and Illinois is ranked in the top 10 states in funding from the US National Institutes of Health. They provide a deep dive into the windy city’s standing in the life science space.

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XTalks

DECEMBER 21, 2022

The past year has been a strong one for life sciences industries, from pivotal gene therapy approvals to continued innovations in the biotech and medical device spaces. Here are some of the top life science trends and topics from 2022. Gene Therapy Approvals. Manufacturer: bluebird bio Inc.

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Pharmaceutical Technology

JANUARY 22, 2023

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. However, while only around 34% of the approvals in 2018 were for orphan drugs, 54% new approvals in 2022 were for drugs to treat rare diseases. of familial ALS cases. Tofersen has a PDUFA date of April 25.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). It has been modified so that it cannot replicate in normal cells.

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Gene Therapy Gene FDA Approval Dermatology 97

XTalks

OCTOBER 22, 2024

gene, and an estimated 60 percent of all gastric cancers harbor the protein. The FDA had rejected Vyloy in January due to “unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility” for the drug. Gastric cancer often has a poor prognosis, especially in advanced stages.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Gene Therapy Gene FDA Approval Antibody 98

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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XTalks

NOVEMBER 15, 2024

The trial was evaluating the drug in combination with the chemotherapy azacitidine for the treatment of newly diagnosed patients with higher-risk myelodysplastic syndrome (HR-MDS) who harbor overexpression of the RARA (retinoic acid receptor alpha) gene.

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Trials HR Immune Response FDA Approval 105

XTalks

OCTOBER 27, 2021

In this episode, the editorial team discusses the FDA approval of Cyltezo as the first interchangeable biosimilar for AbbVie’s Humira. The approval lends to the growing Humira biosimilars market, which will take off in 2023 with Boehringer Ingelheim’s Cyltezo and biosimilars from Amgen, Merck and Alvotech.

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XTalks

MAY 31, 2023

In this episode, Ayesha talked about the FDA approval of the first topical gene therapy for the treatment of a rare skin condition. Ayesha and the team also discussed the FDA approval of Bausch + Lomb’s first prescription drug for the treatment of dry eye disease. It’s the first treatment for the rare disease.

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XTalks

NOVEMBER 15, 2024

XTALKS WEBINAR: Solutions for Vaccine Innovation and Gene Therapy: Unlocking the Power of RNA Live and On-Demand: Tuesday, December 17, 2024 , at 9am EST (3pm CET / EU-Central) Register for this free webinar to explore how next-generation RNA technologies can provide effective solutions for vaccine innovation and gene therapy.

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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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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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Pharmaceutical Technology

APRIL 18, 2023

This news follows the treatment’s FDA approval on 10 March, making it the first-approved Rett Syndrome treatment in the US. The FDA approved the drug based on positive Phase III data from the Lavender trial. We have worked hard to make Daybue available as quickly as possible following FDA approval.”

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XTalks

MARCH 21, 2025

With a 30-month shelf life and rapid absorption, Neffy offers an on-the-go alternative to auto-injectors. Following Neffys FDA approval , ARS Pharma reported $2.3 Iovance Biotherapeutics Iovances Amtagvi (lifileucel) is FDA-approved as the first tumor-infiltrating lymphocyte (TIL)-based therapy for solid tumors.

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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.

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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.

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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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XTalks

MAY 31, 2022

Tapinarof treatment in a mouse model of psoriasis resulted in the downregulation of inflammatory cytokine expression in skin tissue, which involved IL17A , IL17F , IL19 , IL22 , IL23A and IL1B gene expression. Clinical Trials of Vtama.

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XTalks

FEBRUARY 21, 2025

More recently, MSN reported on a Cell Genomics study where CRISPR reactivated dormant genes in patient-derived cells, restoring normal gene expression and improving metabolic function an innovation that could address PWS at its genetic roots.

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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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XTalks

JANUARY 9, 2023

The US Food and Drug Administration (FDA) granted Roche’s Lunsumio (mosunetuzumab) an accelerated approval near the end of 2022 for the treatment of adult patients with relapsed or refractory follicular lymphoma. It was approved in the EU for the same indication in June last year.

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XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). With a list price of $3.5 Hemophilia B primarily affects men.

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XTalks

MAY 15, 2023

Chiesi Global Rare Diseases in collaboration with Protalix BioTherapeutics announced the US Food and Drug Administration (FDA) approval of their new medication Elfabrio (pegunigalsidase alfa-iwxj) for the treatment of Fabry disease. Genetic confirmatory testing is then conducted to identify the specific mutations in the GLA gene.

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FDA Approval Gene Genetics Clinical Trials 98

XTalks

NOVEMBER 1, 2024

Why it sold so well: Botox is FDA-approved for the treatment of chronic migraines and that is currently the only chronic condition for which it has received approval. Related: Top 30 Drugs to Watch in 2024: Insights from 2023 Sales Data 1. billion in the US and $4.1 billion worldwide last year.

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FDA Approval Drugs Botox Sales 85

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). There are many different compounds currently in testing for ALS, such as gene therapies.

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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

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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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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