Pharma Mirror

JANUARY 30, 2022

Ben Beckley, Global Lead at EmerGENE explores the market challenges holding back Cell and Gene Therapy (C&GT) from taking its place as an established treatment area. Some 3,366 treatments are currently in preclinical and clinical development[1]. As a treatment area, C&GT has shown robust growth in recent years.

Gene Therapy 130

Gene Therapy Gene Clinical Development Marketing 130

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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Gene Therapy Gene Packaging Packaging 155

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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Development Gene Therapy Protein Gene 246

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Editing Gene Gene Therapy In-Vivo 130

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.

Clinical Trials 88

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

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

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

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Gene Editing Genetics Drugs Gene 52

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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RNA Vaccine Vaccination In-Vivo 147

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

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

MARCH 2, 2023

QN-023a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for Relapsed Acute Myeloid Leukemia. QN-023a overview QN-023a is under development for the treatment of relapsed or refractory acute myeloid leukemia (AML). The company develops gene-editing organ transplantation technology.

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Gene Editing Genetic Engineering Engineer Clinical Trials 100

Pharmaceutical Technology

MARCH 2, 2023

QN-019a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). The company develops gene-editing organ transplantation technology.

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Gene Editing Clinical Trials Clinical Trials Genetics 100

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.

Genomics 105

Genomics Genome Clinical Trials Clinical Trials 105

Worldwide Clinical Trials

JULY 8, 2024

With radiopharmaceuticals emerging as a diagnostic and therapeutic (theranostics) procedure, many are in the clinical development pipeline and are expected to play a crucial role in the future of healthcare. Genetic Factors : Certain genetic mutations, such as those in DNA repair genes, can affect how cells respond to radiation.

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Hormones Regulation Clinical Trials Clinical Trials 130

Pharmaceutical Technology

APRIL 5, 2023

More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. This includes the first potential approval of a CRISPR-based gene therapy called exa-cel , which is developed by CRISPR Therapeutics and Vertex Pharmaceuticals. AZ: Cell and gene therapies often come with a high price.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

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

pharmaphorum

JULY 5, 2022

It can be caused by exposure to loud noises – including prolonged use of earbuds at high volumes – as well as cancer treatment, illnesses, genetic mutations, or aging. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide. An estimated 1.57

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In-Vivo Bioinformatics Genetics Licensing 110

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

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

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. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Clinical Trials Clinical Trials Vaccine Vaccination 75

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. We are excited to investigate the potential of PBFT02 as a treatment for FTD-GRN as we initiate our clinical development program in the coming months.”.

Gene Therapy 52

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.

Gene Therapy 52

Gene Therapy Trials Gene Vaccine 52

pharmaphorum

AUGUST 12, 2020

Fabry disease is a rare genetic disease caused by a deficiency of an enzyme called alpha-galactosidase A that causes a build-up of toxic metabolites that cause a range of symptoms including pain, gastrointestinal symptoms and kidney damage. billion last year to $3.8

Drugs 98

Drugs Genetic Disease Gene Therapy Trials 98

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

Roots Analysis

FEBRUARY 27, 2024

Over the last few decades, various viral vector manufacturing have been developed, optimized and standardized for introduction of therapeutic DNA / gene of interest into a patient’s body / cells. On the other hand, among non-viral gene delivery tools, plasmid DNA has emerged as the preferred option.

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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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Clinical Research Research Clinical Trials Clinical Trials 130

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. Regenerative medicine and advanced therapies thriving despite Covid-19 disruption.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Trials 52

The Pharma Data

DECEMBER 13, 2020

As part of the collaboration, Hummingbird will be leveraging Tempus’ AI-enabled platform and proprietary data, as well as joining its TIME Trial® Network, for rapid identification, site activation and efficient enrollment of cancer patients who have NRG1 fusions and meet eligibility criteria for HMBD-001 clinical trials. About HMBD-001.

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Clinical Development Medicine Development Antibody 40

The Pharma Data

JULY 6, 2021

PGRN is a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, making it one of the most attractive genetically validated targets for the development of new immuno-neurology treatments. ” Arnon Rosenthal, Ph.D.,

Genetics 52

Genetics Gene Antibody 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. The tender offer is approximately $23.5

Gene Therapy 52

Gene Therapy Gene Production Development 52

The Pharma Data

OCTOBER 27, 2020

The Massachusetts-based biotech is investing in their proprietary “Gene Traffic Control” system, which tells the cells when to express which genes where and in what order within the chromatin regulatory system. The bladder cancer therapy is expected to be in the clinic by 2022. Sirnaomics.

Gene 52

Gene Gene Therapy Medicine Development 52

Scienmag

FEBRUARY 10, 2021

today announced a strategic research and development collaboration to expand the evaluation of Mirati’s two investigational small molecule, potent and selective KRAS inhibitors – adagrasib (MRTX849), a G12C inhibitor in clinical development, and MRTX1133, a G12D inhibitor in […].

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Development Research Clinical Development Gene 66

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

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 billion in cash. If approved, it could become a $500 million-a-year product, according to the company.

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pharmaphorum

MARCH 29, 2022

C9orf72-associated ALS is a complex genetic form of ALS and there are multiple mechanisms by which the scientific community believes the C9orf72 gene causes disease. Based on the results from this study, Biogen and Ionis have confirmed that the BIIB078 clinical development programme will be discontinued.

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Trials RNA Containment Genetics 49

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