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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Gene Therapy Life Science Gene Clinical Trials 98

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

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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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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Pharmaceutical Technology

JANUARY 26, 2023

Cell and gene therapies are predicted to be the future of medical treatment by providing the body with the means to repair itself and recover from a range of serious conditions and severe diseases. Often, cell and gene therapies are spoken of as miracle treatments, yet this progress has hardly occurred overnight.

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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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Gene Therapy FDA Approval Gene Clinical Trials 95

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

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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Trials Clinical Trials Clinical Trials Gene Therapy 104

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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Gene Therapy Gene FDA Approval Dermatology 97

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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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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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

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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Gene Therapy Life Science Gene FDA Approval 52

Cloudbyz

SEPTEMBER 10, 2023

The challenge of managing and securing sensitive data, including PHI and PII, is significant for life sciences organizations. Additionally, the consequences of data breaches can be severe for life sciences organizations. The challenge is further compounded by the need for fast and accurate analysis of data.

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XTalks

JANUARY 4, 2023

In this episode, Ayesha talked about some of the trends to look out for in the life sciences in 2023. Read the full article here: 4 Life Sciences Trends for 2023. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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XTalks

AUGUST 31, 2022

Ayesha shared news about another significant FDA approval for a gene therapy to treat transfusion-dependent beta thalassemia. Bluebird bio was awarded the approval for its gene therapy Zynteglo (beti-cel), which is a one-time treatment for the rare blood disorder. Bluebird’s $2.8M

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BioPharma Reporter

MARCH 22, 2022

Fujifilm Irvine Scientific, Inc, has announced the acquisition of Shenandoah Biotechnology, a company supplying recombinant proteins to the drug discovery, life science research, and cell and gene therapy markets.

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XTalks

SEPTEMBER 29, 2022

The one-time administered gene therapy has also taken the title of being the world’s most expensive drug at $3 million. Hear more about the treatment and how the high prices of gene therapies can be alarming, but reimbursement programs are designed to pick up the costs. 7 that the CDC is closely watching.

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BioPharma Reporter

AUGUST 14, 2023

Teknova, a producer of life science reagents, has opened a new GMP-certified production facility which it claims will enable bioprocessing and gene therapy companies to get into the clinic faster.

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Reagent Gene Therapy Life Science Manufacturing 98

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

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Gene Editing Life Science Gene Gene Therapy 52

XTalks

JANUARY 3, 2024

Novartis and Voyager Therapeutics have struck a licensing deal that will see Novartis pay Voyager $100 million upfront to develop gene therapy candidates targeting Huntington’s disease (HD) and spinal muscular atrophy (SMA). The total value of the deal could potentially reach $1.2 Sandrock, Jr., MD, PhD, CEO of Voyager.

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BioPharma Reporter

MARCH 30, 2023

Pharmaron Beijing Co Ltd says its Liverpool, UK based gene therapy CDMO has obtained a grant from the UK governmentâs life sciences innovation manufacturing fund (LSMIF) to expand its facilities.

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Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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pharmaphorum

NOVEMBER 16, 2022

This is the case for the new generation of treatments known as advanced therapies, which encompasses cell and gene therapies, as well as tissue engineering. Of the innovations that have happened recently in pharma drug development, it is cell and gene therapy that have received the most attention.

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XTalks

APRIL 8, 2025

Specialized facilities such as gene therapy dosing centers or advanced MRI sites can serve as treatment hubs, even if they are far from patients homes. In gene therapy studies, for instance, patients may need to travel to specialized dosing locations outside their home country.

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Clinical Trials Clinical Trials Trials Gene Therapy 88

pharmaphorum

OCTOBER 4, 2022

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a deal which is expected to result in a combined sum of $215 million in cash and investments – projected to be enough for funding into 2025. This is due to close concurrently with the merger.

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Gene Therapy Gene Life Science Genetics 115

BioPharma Reporter

JULY 3, 2023

Donaldson, a provider of filtration products and solutions, has acquired Univercells Technologies, a producer of biomanufacturing solutions for cell and gene therapy research, development, and commercial manufacturing, to broaden its life sciences reach.

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Gene Therapy Life Science Gene Manufacturing 93