Pharmaceutical Technology

JANUARY 17, 2023

Spain’s Hospital Universitario 12 de Octubre researchers, along with the Josep Carreras Leukaemia Research Institute, have developed a new cell therapy based on STAb cells to treat a type of leukaemia. The Spanish Association Against Cancer (AECC) provided funding for the new STAb therapy development.

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Camargo

JULY 30, 2021

Barry Mangum talks the importance of pediatric research, its evolution over the years, and the obstacles and opportunities that the industry faces as it moves forward. How long have you been in pediatric clinical research, and how did you enter the field? We used drugs we developed in adults off-label in children.

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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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Pharmaceutical Technology

JUNE 16, 2023

Along with iCGD and MGB, DOH will facilitate the clinical research and treatment associated with prioritised global genetic diseases in the fields of rare diseases, oncology, cardiovascular and metabolic diseases and neurology.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Medical Xpress

NOVEMBER 21, 2022

In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).

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Roots Analysis

AUGUST 29, 2023

Over the years, cell and gene therapies (CGT) and other advanced therapy medicinal products (ATMPs) have managed to capture the interest of both drug developers and healthcare investors. Their popularity can be attributed to various factors, such as minimal side effects, clinical efficacy and their personalized nature.

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XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

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Outsourcing Pharma

OCTOBER 31, 2022

Thermo Fisherâs clinical research subsidiary, PPD, invests $59 million in sample management and testing capacity for vaccines and cell and gene therapy products with its expansion of laboratory operations in Kentucky.

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Velocity Clinical Research

OCTOBER 4, 2023

Velocity Clinical Research, the leading multi-specialty clinical sites business, announced it is opening greenfield clinical research sites in Bristol, Leicester, and Romford, demonstrating the company’s commitment to the U.K. The sites launch at a time when clinical trial activity in the U.K.

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BioPharma Reporter

FEBRUARY 18, 2021

Massachusetts-based clinical research organization (CRO), Charles River Laboratories, is to acquire Cognate BioServices, a cell and gene therapy contract development and manufacturing organization (CDMO) for around US$875m in cash.

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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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BioTech 365

DECEMBER 2, 2020

4D Molecular Therapeutics Strengthens Leadership Team with Key Appointments in Clinical Research and Development 4D Molecular Therapeutics Strengthens Leadership Team with Key Appointments in Clinical Research and Development EMERYVILLE, Calif.–(BUSINESS

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XTalks

JUNE 4, 2024

In the ever-evolving landscape of pharmaceutical development, the complexity of early phase clinical trials is increasing. This surge in complexity is driven by escalating drug development costs, heightened competition and advanced scientific innovations. And that has a lot of downstream consequences.” This trend, which Dr.

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Cloudbyz

JUNE 26, 2023

Generative artificial intelligence (AI) techniques are increasingly being applied in clinical research to enhance various aspects of medical data analysis, diagnosis, treatment planning, and drug discovery. These notes have been used to advance research in NLP and clinical decision support systems.

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Worldwide Clinical Trials

JANUARY 24, 2024

However, clinical research around Fragile X is considerably robust, making the possibility of clinical trial participation a potential motivation for parents to choose to undergo genetic testing for their children. As of July 2023, the American Society of Cell + Gene Therapy stated there were 3,905 therapies in development.

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The Pharma Data

OCTOBER 18, 2020

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. Ricci told BioSpace the company has been “investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area.”

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Worldwide Clinical Trials

MAY 20, 2022

The past few years we’ve had exciting opportunities to work on more cell and gene therapy programs for our sponsors. The industry is just at the tip of the iceberg for exploring the potential of these therapies in revolutionizing how we treat disease, and as the approvals start to trickle in, the pipeline only grows.

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XTalks

OCTOBER 18, 2022

A clinical research organization (CRO) is a company that delivers outsourced services to plan, manage and execute clinical trials for biotechnology, pharmaceutical, and medical device companies. Read on to learn why life science and clinical research professionals are choosing a career with Medpace.

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Cloudbyz

AUGUST 9, 2023

The clinical research landscape is rapidly evolving. As it becomes more complex with growing volumes of data, evolving regulations, and the pressure for faster drug development, traditional methods of clinical research management are no longer sufficient. This is where the platform approach comes into play.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023. Why is Casgevy a Breakthrough Gene Therapy for Patients with Sickle Cell Disease? What Does the Future of Gene Editing Hold?

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BioSpace

AUGUST 8, 2021

Voyager’s primary focus will be on developing gene therapies for Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), and pre-clinical research in spinal muscular atrophy and diseases linked to GBA1 mutations.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. The post Risk Assessment for use of Engineered Genetic Materials in Clinical Research appeared first on Advarra.

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Pharmaceutical Technology

MAY 3, 2023

Janssen Biotech and the Cellular Biomedicine Group (CBMG) have entered a global collaboration and licence agreement for the development of next-generation chimeric antigen receptor (CAR) T-cell therapies to treat B-cell malignancies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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XTalks

FEBRUARY 24, 2023

When life sciences industry partners with patients and their advocacy communities, the patient voice becomes integrated into the drug development process. It’s an increasingly common and evolving practice that has brought about meaningful change in the clinical trial process.

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XTalks

JANUARY 17, 2024

Estimates based on publicly available information suggest more than 40 percent of all new therapies in development are cancer treatments. Dr. Morrison has over 30 years of oncology preclinical and clinical experience between academia and the industry sector in pharma and clinical research organizations (CROs).

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Worldwide Clinical Trials

OCTOBER 24, 2023

While this is a small number of people in each rare disease patient community, the significant and continued growth of rare disease drug development is unsurprising given that there are over 10,000 rare diseases , with more being discovered every year. Roughly 80% of rare diseases are due to a known genetic driver.

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XTalks

MAY 17, 2024

Xtalks spoke with Richard Philipson, MD, Chief Medical Officer (CMO) at Calliditas Therapeutics, a Sweden-based company with full commercial operations in the US focused on developing treatments for rare diseases to learn more about the continuing evolution of rare disease drug development.

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XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​.

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Drug Discovery World

FEBRUARY 12, 2024

Cameron Ross, SVP, Generative AI, Elsevier “AI has the potential to dramatically shorten one of the most costly areas of early drug development – small molecule discovery. Matt Todd, Head of Architecture, Ori Biotech “We expect AI and data analytics to support pivotal advances in cell and gene therapy (CGT) manufacturing.

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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. Gene Therapy Approvals. Bluebird bio is a notable mention as it secured FDA approvals for two of its gene therapies, Zynteglo and Skysona.

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ACRP blog

SEPTEMBER 27, 2023

Food and Drug Administration’s (FDA’s) Rare Disease Endpoint Advancement (RDEA) Pilot Program , the clinical research enterprise gains new support for efforts aimed at efficacy endpoint (or clinical outcome) development and the timely approval of drugs and biological products that treat rare diseases, including rare diseases in children.

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XTalks

MAY 23, 2024

With over 25 years of experience, Dr. Duke’s expertise in growth and bone disorders has been instrumental in advancing clinical research and improving patient outcomes. In addition, ethical considerations are paramount in developing treatments for children.

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Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). News round-up for 6-10 March by DDW Digital Content Editor Diana Spencer.

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Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient.

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Drug Discovery World

JANUARY 26, 2024

In the first case of its kind, gene therapy has restored hearing in a patient in the United States. AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF).

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Drug Discovery World

FEBRUARY 10, 2023

The research will support the clinical development of CTx001, CTx’s lead gene therapy asset for the treatment of GA, by enabling the identification of patients with complement-driven AMD for future interventional studies.

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