BioSpace

SEPTEMBER 28, 2021

?Gene therapy research delivers breakthroughs this week with a CRISPR-edited droplet that could lead to a cure for heart disease and the possibility of turning glial brain cells into neurons.

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Gene Editing Gene Gene Therapy Research 91

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. How does the Gene Editing Tool PASTE Work?

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Medical Xpress

DECEMBER 21, 2022

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

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Gene Therapy Gene Editing Gene Research 98

STAT News

MARCH 22, 2023

DALLAS — It took just three years, after the tool’s invention, for researchers to devise ways of using CRISPR-Cas9 gene editing to treat mice with Duchenne muscular dystrophy. One company was even bought out for over $200 million. One company was even bought out for over $200 million.

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Medical Xpress

FEBRUARY 7, 2023

A recent study by University of Illinois Chicago researchers in the College of Business Administration analyzed how the experiences of racism and discrimination in health care significantly affect the adoption of innovative medical technology like gene therapies and the gene editing tool CRISPR.

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Gene Editing Gene Therapy Gene Marketing 93

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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Gene Editing Gene Genomics Genome 64

pharmaphorum

SEPTEMBER 29, 2020

As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. Pre-competitive collaborations, often in basic and preclinical research, can reduce the barrier of competition and drive benefits for all stakeholders, most notably, the patient.

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Research Gene Editing Development Gene 132

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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pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. The move marks a continuation in its ongoing commitment to exploring gene-editing technology.

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Gene Editing Gene Gene Therapy Genetics 52

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”

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Gene Gene Editing Gene Therapy Clinical Trials 130

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

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XTalks

JANUARY 26, 2024

Technological advancements are revolutionizing cancer research and therapies. Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment.

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Research Gene Editing Gene Genetics 118

pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Scienmag

AUGUST 18, 2020

In Nature Communications, Fred Hutch scientists demonstrate in a mouse model how gene therapy could cure an infection that afflicts billions of people Infectious disease researchers at Fred Hutchinson Cancer Research Center have used a gene editing approach to remove latent herpes simplex virus 1, or HSV-1, also known as oral herpes.

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Gene Therapy Gene Gene Editing Scientist 45

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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BioTech 365

AUGUST 13, 2020

ASC), a leading gene editing company focused on the development of enabling tools for cell and gene therapy has been awarded close to $2.0 million US dollars in a Small Business Innovation Research (SBIR) grant … Continue reading →

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The Pharma Data

DECEMBER 8, 2020

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced.

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Scienmag

JULY 2, 2021

The ongoing effort to explore […].

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pharmaphorum

DECEMBER 14, 2022

Brown brings 20 years of experience at organisations including Quintiles, INC Research, and Syneos Health. Boyds hires cell and gene therapy expert. Ginty is an expert in cell and gene therapies and has previously done regulatory affairs work at AviadoBio and UCB-subsidiary Handl Therapeutics. Other agency hires.

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Life Science Gene Therapy Gene Editing Gene 98

XTalks

FEBRUARY 27, 2024

Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy. Both therapies were awarded their approvals on the same day in December 2023.

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Drug Discovery Today

JANUARY 15, 2020

Next-generation gene editing system has applications in development of cell and gene therapies. Base editing builds on CRISPR offering and will expand Horizon’s research tools and services. Horizon seeks partners to assess and shape platform development

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Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

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DNA Gene Gene Editing Scientist 56

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

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Gene Editing Gene In-Vivo Genetics 52

XTalks

DECEMBER 20, 2023

AI and Machine Learning: Transforming Research Artificial intelligence (AI) and machine learning (ML) are revolutionizing research approaches in the life sciences. CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason.

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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Delveinsight

FEBRUARY 25, 2021

The last few decades have been marked by increased research and development in neuroscience. Positioning of Gene and Cell Therapies in Neurosciences. Decades of technological progress and careful research, Gene and cell therapy (GCT), which were previously viewed as the ‘future’ of medicine, are now all set to become a reality.

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Gene Gene Therapy Gene Editing Development 52

Roots Analysis

FEBRUARY 27, 2024

How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

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Genomics Genome Gene In-Vivo 40

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

XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.

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Roots Analysis

JANUARY 16, 2023

During our research, we found out that most of the at-home self-testing kits are developed in the form of strips; these strips are primarily used for monitoring diabetes and are approved by various regulatory authorities, such as the USFDA, ISO and IVD. Several organizations have extended support to aid research efforts in this domain.

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Bioassay Gene Editing Marketing Gene Therapy 52

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.

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Business Development Development Gene Editing In-Vivo 40

WCG Clinical

OCTOBER 21, 2024

Clinical trials are the cornerstone of advancing medical research, but behind the science, the experiences of participants and their families offer invaluable insight into how research impacts real lives. Many individuals first encounter clinical research due to a personal or family medical diagnosis.

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Delveinsight

SEPTEMBER 14, 2021

The proceeds from the Series B financing will be utilized to progress ADARx’s proprietary RNA platform technologies for base editing, inhibition, degradation, and delivery. Vor Biopharma has declared that the FDA has awarded their acute myeloid leukemia (AML) engineered hematopoietic stem cell therapy (eHSC), VOR33, a Fast Track Designation.

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RNA Clinical Trials Clinical Trials Drugs 67

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. That would just slow the whole field down.

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XTalks

FEBRUARY 10, 2023

“In our experience, the successful operational execution of cell therapy trials involves complex regulatory, clinical and operational considerations, which are not required in trials using small molecules,” stated Dr. Jeff Vassallo, Senior Director of Clinical Trial Management, at the global clinical contract research organization (CRO) Medpace.

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The Pharma Data

NOVEMBER 3, 2020

In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome. LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies.

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The Pharma Data

DECEMBER 28, 2020

Scopus is a biopharmaceutical company whose lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Dr. Hofstein completed his postdoctoral training and research in the Department of Neurobiology at Harvard Medical School. Hofstein received his B.Sc.

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