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 92

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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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

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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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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Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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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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Pharmaceutical Technology

NOVEMBER 16, 2022

A custom-made viral vector and cell therapy centre of excellence, ElevateBio BaseCamp has an end-to-end process development and existing Good Manufacturing Practice (cGMP) production expertise for research, clinical and commercial cell and gene therapies and regenerative treatments.

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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 82

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

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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Gene Editing Gene Gene Therapy Development 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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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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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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Gene Therapy Gene Gene Editing Clinical Trials 52

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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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec.

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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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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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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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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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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

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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Clinical Trials Clinical Trials Trials Clinical Research 52

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). Lilly will provide funding for the Phase I clinical trials. “In

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Gene Editing Gene Gene Therapy In-Vivo 130

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

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Gene Editing Gene Gene Therapy Genetics 130

Scienmag

JULY 2, 2021

The ongoing effort to explore […].

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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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XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

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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

XTalks

NOVEMBER 30, 2023

Over the last 35 years, substantial strides have been made in tackling HIV/AIDS, propelled by advancements in medical research, enhanced accessibility to treatment and prevention and a more comprehensive understanding of the virus. World AIDS Day offers an occasion for reflection on the strides achieved thus far.

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FDA Approval Gene Therapy Gene Clinical Trials 110

XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the 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

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029. FDA backing.

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Pharmaceutical Technology

FEBRUARY 13, 2023

While several therapeutics are available for treating symptoms associated with epilepsy, researchers and patients have strongly called out the need for more holistic treatments that would address the condition as a whole. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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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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