Pharmaceutical Technology

MAY 22, 2023

The US Food and Drug Administration (FDA) has granted approval for Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). As a condition of the SKYSONA Accelerated Approval, the manufacturer will provide confirmatory long-term clinical data to the FDA.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries.

Gene Therapy 93

Gene Therapy Gene Trials Protein 93

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.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

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). Additionally, the FDA said a “clinical benefit of Elevidys, including improved motor function, has not been established.”

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The study is one of the largest DMD Phase III trials to date. million last year, of which $131.3

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

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.

Gene Therapy 52

Gene Therapy FDA Approval Life Science Gene 52

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Safety and Efficacy of Filsuvez The FDA’s approval of Filsuvez is supported by compelling clinical evidence. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector.

FDA Approval 111

FDA Approval Gene Therapy Gene Trials 111

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).

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

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. percent Roctavian has for its European Union (EU) approval. percent Roctavian has for its European Union (EU) approval. Roctavian, a $2.9

Gene Therapy 98

Gene Therapy Gene FDA Approval Antibody 98

pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

pharmaphorum

JUNE 10, 2022

The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks. bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

Gene Therapy 98

Gene Therapy Gene FDA Approval Trials 98

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. Here’s what this means for pharmaceutical leaders and patients.

Gene Therapy 64

Gene Therapy Gene In-Vitro Development 64

Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances. The post The evolution and future of gene therapy appeared first on Drug Discovery World (DDW).

Gene Therapy 52

Gene Therapy Gene FDA Approval Clinical Trials 52

Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

FDA Approval 52

FDA Approval Trials Radiology In-Vivo 52

Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances. He co-founded AskBio in 2001 and in October 2020, Bayer acquired AskBio for $4 billion.

Gene Therapy 52

Gene Therapy Gene FDA Approval Clinical Trials 52

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.

Gene Therapy 119

Gene Therapy Gene FDA Approval Production 119

The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV.

Gene Therapy 52

Gene Therapy Gene FDA Approval Trials 52

pharmaphorum

MARCH 6, 2022

As bluebird bio struggles to get its gene therapies over the finishing line, the company says its financial position raises “substantial doubt” about its ability to continue as a going concern. The post bluebird says future uncertain as delays to gene therapies bite appeared first on.

Gene Therapy 103

Gene Therapy Gene FDA Approval Sales 103

pharmaphorum

NOVEMBER 23, 2022

uniQure and CSL have become the first pharma companies to get regulatory approval for a gene therapy for haemophilia B anywhere in the world, after the FDA cleared their Hemgenix therapy for adults with the bleeding disorder in the US. link] pic.twitter.com/QWy0WgNGQu.

Gene Therapy 59

Gene Therapy Gene FDA Approval Trials 59

Drug Discovery World

FEBRUARY 19, 2024

“The approval of Amtagvi represents the culmination of scientific and clinical research efforts leading to a novel T cell immunotherapy for patients with limited treatment options.” Amtagvi is the first FDA-approved tumour-derived T cell immunotherapy. Among patients who were responsive to the treatment, 56.5%, 47.8%

FDA Approval 52

FDA Approval Contract Manufacturing Manufacturing Trials 52

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). months in the KEYNOTE-057 trial.

FDA Approval 52

FDA Approval Vaccination Vaccine Sales 52

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. It was first approved in the US in 2019 to treat children below the age of two with SMA.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

Pharmaceutical Technology

JUNE 9, 2023

Rocket Pharmaceuticals has received fast track and orphan drug designations for RP-A601 from the US Food and Drug Administration (FDA) to treat plakophilin-2-related arrhythmogenic cardiomyopathy (PKP2-ACM). In April 2023, the FDA approved the investigational new drug (IND) application for RP-A601 to begin a Phase I clinical trial.

Gene Therapy 147

Gene Therapy Gene FDA Approval Trials 147

XTalks

NOVEMBER 10, 2023

Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.” The study evaluated both prophylactic and on-demand Adzynma ERT compared to plasma-based therapies in cTTP patients. Adzynma’s approval is the second for Takeda this week.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

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.

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 52

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.

Manufacturing 95

Manufacturing Gene Therapy FDA Approval Gene 95

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.

FDA Approval 52

FDA Approval Manufacturing Trials Clinical Trials 52

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

Gene Therapy 59

Gene Therapy Gene Antibody FDA Approval 59

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.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

Gene Therapy 264

Gene Therapy Gene Trials Clinical Trials 264

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.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Trials 52

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.

FDA Approval 98

FDA Approval Gene Therapy Gene Marketing 98

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.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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.

FDA Approval 52

FDA Approval Gene Therapy Antibody Gene 52