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.

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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.

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Clinical Development Gene Therapy Development Gene 133

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

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XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Cross-border enrollment captures a wider range of genetic backgrounds, environmental influences, and cultural perspectives.

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Clinical Trials Clinical Trials Trials Gene Therapy 88

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.

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Development Gene Therapy Genetics Gene 130

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.

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Gene Therapy Gene Genetic Disease Manufacturing 66

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.

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Life Science Development Gene Therapy Genetics 162

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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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.

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Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics will be responsible for advancing the research and development of the Lp(a) programme by completing Phase I clinical development. Lilly will handle the subsequent development, manufacture and commercialisation activities of the programme.

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Gene Editing Gene Gene Therapy In-Vivo 130

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.

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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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Gene Therapy Gene Clinical Trials Clinical Trials 52

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.

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Genetics Medicine Gene Therapy Marketing 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.

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Gene Therapy Gene In-Vivo Genetics 40

The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. “We About Passage Bio.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

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.

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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.

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Clinical Trials Clinical Trials Vaccination Vaccine 75

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.

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Drugs Genetic Disease Gene Therapy Trials 98

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.

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Manufacturing Gene Therapy Gene DNA 52

Worldwide Clinical Trials

JULY 29, 2024

Obesity is a major factor in the prevalence of T2D, and it is vital to develop T2D treatments with this in mind. Evolving sentiments recognize obesity as a medical condition and lifestyle choice as well as a chronic disease with genetic, psychological, cultural, and environmental causes (Figure 1).

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pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.

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Genome Genomics Clinical Trials Clinical Trials 105

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).

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Gene Therapy Gene Production Development 52

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

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.

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Gene Therapy Trials Genetics Drugs 52

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

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Gene Therapy Research Gene Antibody 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 Development 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.

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In-Vivo Gene Editing Trials Gene 52

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.,

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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.

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Business Development Development Gene Editing In-Vivo 40

The Pharma Data

DECEMBER 17, 2020

Cadavid will be responsible for the strategy, direction and execution of the company’s clinical development programs. Prior to Fulcrum, Cadavid held several leadership positions at Biogen, including Senior Medical Director of the multiple sclerosis clinical development group. based Rinri Therapeutics.

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Development Gene Therapy Engineer Life Science 52

The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). LEXINGTON, Mass.,

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Clinical Trials Clinical Trials Gene Editing Genome 40

The Pharma Data

AUGUST 7, 2020

As the pharma industry stands firm in its commitment to advance the sector to fight Covid-19, news has emerged from the European Commission who intend to streamline the development of therapies using genetically modified organisms to treat Covid-19.

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Clinical Trials Clinical Trials Gene Trials 52

Advarra

JUNE 20, 2023

Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 Typical clinical development timelines for anticancer drugs average an estimated 6.7

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Trials Clinical Trials Clinical Trials Research 52

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?

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Gene Therapy Gene Trials Clinical Trials 95

Delveinsight

JULY 30, 2020

However, the clinical presentation of the disease vary, and neither genetic mutation nor studying the biochemical activity of the brain is sufficient to speculate the phenotypic variation in individuals. The pharma players are evaluating their pipeline candidates in different stages of clinical development.

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Hormones Marketing Gene Trials 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.

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Gene Therapy Gene Manufacturing Clinical Trials 97

XTalks

APRIL 12, 2021

Like other neurodegenerative disorders, such as Alzheimer’s disease and dementias, Parkinson’s disease research has been immensely challenging for biotechs, with a high rate of late-stage attrition in trials leading to a lack of disease-modifying therapies being approved. Axovant Gene Therapies. Prevail Therapeutics.

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