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

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

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

APRIL 24, 2024

ImmunityBio’s Anktiva (N-803, or nogapendekin alfa inbakicept-pmln) along with the Bacillus Calmette-Guérin (BCG) vaccine has won US Food and Drug Administration (FDA) approval for the treatment of non-muscle invasive bladder cancer (NMIBC).

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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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Drug Discovery World

MARCH 16, 2023

The cell and gene therapy (CGT) market is rapidly expanding, and projections suggest it will reach over $90B by 2023. With a maturing clinical pipeline, many FDA-approved treatments are on the horizon. The post Can characterisation industrialise cell and gene therapy? appeared first on Drug Discovery World (DDW).

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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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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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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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XTalks

SEPTEMBER 12, 2022

After approvals in Japan and Europe, Sanofi’s Xenpozyme has secured one from the US Food and Drug Administration (FDA) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Patients in the trial received Xenpozyme or placebo for 52 weeks.

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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

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

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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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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XTalks

MARCH 10, 2023

But with Skyclarys (omaveloxolone), the first and only drug to treat Friedreich’s ataxia in patients aged 16 years and older, Reata Pharmaceuticals has developed a new treatment option to improve neurological function. This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher.

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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

JUNE 12, 2023

Additionally, BMS is developing another manufacturing site in Leiden, Netherlands. Over the past decade, this site has been pivotal in the development, production and testing of both clinical and commercial medications. In recent years, several cell therapy manufacturing facilities in the US have garnered FDA approval.

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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

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). XTALKS WEBINAR: Development of Covalent Drugs with New Emerging Technologies. percent of patients taking RADICAVA ORS.

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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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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

NOVEMBER 30, 2023

Global entities such as the WHO, the Global Fund and the Joint United Nations Programme on HIV/AIDS (UNAIDS) have developed comprehensive strategies aligned with Sustainable Development Goal (SDG) target 3.3, This marked the first FDA-approved injectable, complete regimen for HIV-infected adults, administered once a month.

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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

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 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. Lymphoma is one of the most aggressive types of cancer.

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XTalks

JUNE 16, 2023

Foundation Medicine recently announced that the US Food and Drug Administration (FDA) approved FoundationOne Liquid CDx to serve as a companion diagnostic for identifying metastatic colorectal cancer patients with BRAF V600E alterations, suitable for treatment with Pfizer’s Braftovi (encorafenib) with cetuximab.

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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

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

MAY 31, 2022

Psoriasis is considered a life-long ailment with no permanent cure. The disease usually develops around 15 to 25 years of age and can recur at any point of time in the affected individual’s life. There are five distinct types of psoriasis, namely plaque, guttate, pustular, inverse and erythrodermic psoriasis.

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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

JANUARY 26, 2024

Key breakthroughs at the forefront include novel approaches to cancer treatment and diagnosis, the integration of artificial intelligence (AI) in oncology and the development of advanced preventive measures and care. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

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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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Drug Discovery World

APRIL 27, 2023

The report notes that the focus on science and pharma throughout the Covid-19 pandemic has resulted in the emergence of new locations as life science hubs and name checks Boston, Massachusetts and San Francisco, California as examples of centres with heightened investment appeal in the life sciences 1.

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pharmaphorum

SEPTEMBER 30, 2022

Although the sticker shock that was at first associated with Yervoy (ipilimumab) in 2011 – in the early days of immuno-oncology monoclonal antibodies – has fallen by the wayside, especially in light of CAR-Ts and other “one and done” cell and gene therapies, the field of non-orphan oncology remains a target for cost savings by payers.

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XTalks

AUGUST 15, 2023

struck a worldwide collaboration (excluding Japan) and license agreement with niraparib developer Tesaro (acquired by GSK in 2019) for exclusive rights to the drug in prostate cancer. Lynparza received FDA approval in May 2020 for mCRPC in patients with homologous recombinational repair ( HRR ) gene mutations.

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XTalks

MARCH 28, 2024

Colorectal Cancer: Advances in Treatment Options In recent years, there has been considerable progress in understanding the biology of CRC, which has helped develop more effective and personalized treatment options. An overview of some of the most promising developments in CRC treatment is provided below.

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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. What is SOD1 -ALS? How Does Qalsody Work?

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