Drug Discovery World

FEBRUARY 1, 2024

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. CTX is a rare autosomal recessive genetic disorder that affects the body’s ability to metabolise fats known as cholesterols. Vivet Therapeutics has received €4.9

Gene Therapy 52

Gene Therapy Gene Genetics Manufacturing 52

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

Gene Therapy 92

Gene Therapy Gene Manufacturing Clinical Trials 92

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. How Can Study Protocols Be More Effective?

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Medical Xpress

APRIL 26, 2023

A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa.

Clinical Development 133

Clinical Development Gene Therapy Development Gene 133

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

Gene Therapy 155

Gene Therapy Gene Packaging Packaging 155

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.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

Gene Therapy 52

Gene Therapy Gene Development Production 52

pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. The gene therapy firm will be focused on its most important therapies in beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US And Europe.

Gene Therapy 59

Gene Therapy Gene Genetic Disease Manufacturing 59

Pharmaceutical Technology

AUGUST 24, 2022

Additionally, the AAV capsid platform of Lacerta will be leveraged for discovering and developing new AAV capsids that are streamlined for acting on preferred CNS tissues and cell types. Prevail will also handle the complete production, clinical development and marketing activities.

Development 130

Development Gene Therapy Genetics Gene 130

Roots Analysis

FEBRUARY 27, 2024

On the other hand, among non-viral gene delivery tools, plasmid DNA has emerged as the preferred option. Overall, eleven gene therapies have been approved so far.

Manufacturing 52

Manufacturing Gene Therapy Gene DNA 52

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

Gene Therapy 40

Gene Therapy Gene In-Vivo Genetics 40

Pharmaceutical Technology

AUGUST 17, 2022

Biopharmaceutical contract development and manufacturing organisation (CDMO) AGC Biologics has entered a strategic collaboration with RoosterBio to expedite the manufacturing of cell and exosome therapies. These capabilities comprise cell and exosome genetic engineering for expressing therapeutic targets.

Manufacturing 130

Manufacturing Gene Therapy Genetic Engineering Gene 130

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

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. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics. By Cytiva Thematic.

Life Science 162

Life Science Development Gene Therapy Genetics 162

Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. AZ: Cell and gene therapies often come with a high price.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

Pharmaceutical Technology

AUGUST 17, 2022

Merck will also make royalty payments on any approved products developed out of the partnership. The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Delveinsight

JULY 28, 2020

Moderna has initiated the first large-scale Phase III clinical trial of its COVID-19 vaccine , mRNA-1273 after an expanded contract with the Biomedical Advanced Research and Development Authority (BARDA). At present, more than 150 vaccine candidates are in various stage of clinical development against SARS-CoV-2.

Gene Therapy 52

Gene Therapy Trials Gene Vaccination 52

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.

Gene Therapy 52

Gene Therapy Gene Genetics Clinical Development 52

pharmaphorum

OCTOBER 25, 2022

If completed, the acquisition – which has been termed a “low ball bid” by some – of AGTC will become Syncona’s third company focused on retinal gene therapy (after Nightstar and Gyroscope , acquired by Biogen and Novartis, respectively). which develops innovative therapeutics for neurotology. million in cash ($0.34 per share).

Gene Therapy 52

Gene Therapy Gene Production Development 52

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. “They called him ‘The Boy.’ Who wasn’t supposed to sit up.

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

pharmaphorum

SEPTEMBER 21, 2021

In other cases, rare CNS conditions may be passed on from one generation to the next without knowledge or understanding that the reason for suffering or death among previous generations was genetic in basis and therefore identifiable, and perhaps even treatable.

Gene Therapy 74

Gene Therapy Gene Clinical Trials Clinical Trials 74

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform.

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

pharmaphorum

AUGUST 12, 2020

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. billion by 2027, driven by new oral drugs such as Galafold as well as gene therapies that are still in clinical development.

Drugs 96

Drugs Genetic Disease Gene Therapy Trials 96

The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

Gene Therapy 40

Gene Therapy Gene Containment Development 40

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.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 52

pharmaphorum

OCTOBER 27, 2020

The disappointing result comes as three potential gene therapies for DMD that could tackle the underlying genetic cause of the disorder are making their way through clinical development.

Gene Therapy 52

Gene Therapy Trials Genetics Drugs 52

The Pharma Data

OCTOBER 27, 2020

Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders.

Gene 52

Gene Gene Therapy Medicine Genetics 52

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.

Gene Therapy 246

Gene Therapy Gene Genotype Genetics 246

Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. The team successfully protected HILOs from the immune system without genetic manipulation. J&J has agreed to pay USD 52.50

Gene Therapy 52

Gene Therapy Research Gene Antibody 52

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. This includes support for export and expansion into new territories as well as for innovation and development.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

The Pharma Data

JANUARY 27, 2021

As a clinician, healthcare administrator, biotech entrepreneur and venture capital investor, Steve’s leadership and breadth of experience in the clinical development of cell and gene therapies will be invaluable to PlateletBio as we progress our cell therapy platform into the clinic later this year,” said Sam Rasty, Ph.D.,

Genetic Engineering 52

Genetic Engineering Gene Therapy Gene Engineer 52

The Pharma Data

DECEMBER 13, 2020

Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. Before that, he was Director of Development at Cobalt Biomedicine leading CAR-T and other cell/gene therapy programs, and R&D Director at OvaScience developing stem cell-based products.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Drug Discovery World

SEPTEMBER 14, 2023

joined Pfizer’s Executive Leadership Team as Chief Oncology Research and Development Officer and Executive Vice President reporting to Chairman and Chief Executive Officer, Albert Bourla. Chris Boshoff, M.D.,

Drugs 59

Drugs Vaccination Vaccine Medicine 59

The Pharma Data

OCTOBER 27, 2020

NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. There are currently no disease-modifying therapies for Krabbe disease, and we believe incidence may be 2.6 PHILADELPHIA, Oct. About PBKR03.

Gene Therapy 40

Gene Therapy Drugs Clinical Trials Clinical Trials 40