Pharmaceutical Technology

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.

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Bio Pharma Dive

MAY 6, 2021

Completion of the buyout eases concerns the arrangement might be held up by the FTC, which has signaled it will step up scrutiny of life sciences deals.

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Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Pharmaceutical Technology

DECEMBER 14, 2022

Merck and Synplogen have signed a non-binding Memorandum of Understanding (MoU) to expedite the development and manufacturing of viral vector-based gene therapy applications. The firms intend to merge their expertise to provide simplified viral vector gene therapy development, production and testing in Japan.

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XTalks

JUNE 14, 2023

Courtney Silverthorn who is an Associate Vice President at the Foundation for the National Institutes of Health (FNIH) and the Director of the Accelerating Medicines Partnership (AMP) program including the Bespoke Gene Therapy Consortium. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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XTalks

OCTOBER 15, 2024

Seven cases of blood cancer have been identified in new trial data for bluebird bio’s gene therapy Skysona (elivaldogene autotemcel). The patients had received the autologous hematopoietic stem cell-based gene therapy as a treatment for early-stage cerebral adrenoleukodystrophy (CALD), a rare and fatal neurodegenerative disorder.

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XTalks

OCTOBER 22, 2024

Canadian contract development and manufacturing organization (CDMO) OmniaBio has debuted its new state-of-the-art facility focused on cell and gene therapy (CGT) manufacturing. Focused on advancing regenerative medicine, CCRM supports the development of cell and gene therapies, along with the associated enabling technologies.

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XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

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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

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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pharmaphorum

OCTOBER 22, 2020

Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences investors. Unlike a one-shot gene therapy, omaveloxolone would require continuous dosing to maintain its effects.

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pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

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XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

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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

DECEMBER 8, 2020

has announced that it has acquired the rights to an investigational gene therapy, HMR59, from Hemera Biosciences, LLC designed to help treat a severe form of age-related macular degeneration (AMD). The gene therapy was developed to treat both dry and wet macular degeneration. Janssen Pharmaceuticals Inc.

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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. Prior to Zolgensma, treatments could only help manage symptoms and attempt to improve quality of life.

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BioPharma Reporter

FEBRUARY 9, 2023

The Cell and Gene Therapy Catapult (CGT Catapult) has confirmed its involvement in the development of a new Â900m ($1,097m) life science campus in Stevenage, UK: which is set to become one of the largest in Europe.

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XTalks

JUNE 8, 2023

Over the past few years, there has been a significant expansion in the cell and gene therapy landscape, with an increasing number of therapies entering clinical trials and receiving regulatory approvals. “I think if we all go into these relationships with this mindset, we’ll achieve great things together.”

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XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

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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

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

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XTalks

AUGUST 30, 2022

The hemophilia A gene therapy Roctavian delivers a functional gene to enable the body to make an essential blood clotting protein on its own. Last week, the first gene therapy for severe hemophilia A , Roctavian (valoctocogene roxaparvovec), developed by BioMarin Pharmaceutical Inc.,

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XTalks

JANUARY 16, 2025

BIALs Phase II ACTIVATE trial of BIA 28-6156, which targets GBA1 gene mutations, is ongoing, with results expected in 2026 to determine its potential to modify disease progression. With its Phase III trial on the horizon, bemdaneprocel exemplifies the growing potential of cell and gene therapies for Parkinsons disease.

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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). As a topical treatment, it is also the first readily redosable gene therapy.

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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). It is indicated for patients who do not have a pre-existing medical reason preventing treatment with the therapy.

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Gene Therapy Gene Protein FDA Approval 98

XTalks

JANUARY 6, 2025

The CFTR gene itself is complex, with over 2,000 known mutations. The diversity of CF treatments is expanding with investigational therapies such as Spirovants inhaled gene therapy SP-101 in a Phase I/II trial and Kither Biotechs inhaled peptide therapy KIT2014 in a Phase I trial.

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Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more. So, we set out to solve some of the problems,” she explains.

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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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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

MARCH 8, 2022

On International Women’s Day, Xtalks is celebrating women’s leadership in the life sciences by highlighting some of the female leaders at the forefront of scientific discovery, as well as the continuing challenges of attaining more equitable representation. Challenges in Women’s Leadership.

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Pharmaceutical Technology

APRIL 21, 2023

IBM will also provide its knowledge to help Moderna explore the potential use cases of quantum technologies in life sciences. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Moderna will join the IBM Quantum Network and the IBM Quantum Accelerator programme.

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XTalks

MARCH 25, 2021

Xtalks is proud to announce the launch of the Xtalks Life Science podcast. Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Fresh Conversations About Life Science Topics. She focuses on news relating to the food industry and writes blogs on recruitment and HR in the life sciences.

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Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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XTalks

APRIL 14, 2021

In this episode of the Xtalks Life Science Podcast, Sarah revisits two Parkinson’s biotech companies — Prevail Therapeutics and Voyager Therapeutics — to see what progress they’ve made in their gene therapy clinical development programs since April 2019. Parkinson’s and Melanoma Share an Amyloid Link, Says New Research.

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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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