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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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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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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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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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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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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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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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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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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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

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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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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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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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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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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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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Worldwide Clinical Trials

MAY 30, 2024

These advancements have fostered increased focus from politicians, policymakers, and the investment communities backing academic and industry-sponsored research projects. Researchers established efficacy of edaravone in a 24 week, randomized, placebo-controlled double-blind study in 137 Japanese patients.

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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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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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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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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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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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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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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

DECEMBER 11, 2020

of Children’s Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues found that gene editing using CRISPR-Cas9 is safe and feasible and can provide clinical benefit to patients with sickle cell disease and transfusion-dependent ß-thalassemia. In one study, Haydar Frangoul, M.D.,

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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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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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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR. pyogenes dCas9.

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