Gene Therapy, Genome and In-Vivo - Clinical Research Informer

Gene Therapy

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Genome Editing Market Update: Intellia Therapeutics Presents Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy

Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% 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.

Genomics

Genomics Genome Gene In-Vivo

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

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

In-Vivo Gene Editing Trials Gene

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Webinars

From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

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Trending Sources

Scribe and Prevail partner for CRISPR-based genetic medicines

Pharmaceutical Technology

MAY 17, 2023

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics

Genetics Medicine In-Vivo Gene Therapy

Webinars

From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

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. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy

Gene Therapy Gene Gene Editing In-Vivo

Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

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.”

Gene Therapy

Gene Therapy Gene Gene Editing In-Vivo

Gene editing: beyond the hype

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers. billion in funding.

Gene Editing

Gene Editing Gene Gene Therapy In-Vivo

Scribe and Sanofi partner to develop cell therapies for cancer

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Development

Development In-Vivo Genomics Genome

Gene Editing Innovations with Programmable Gene Insertion (PGI) Technology ft. John Finn, PhD, CSO, Tome Biosciences – Xtalks Life Science Podcast Ep. 160

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.

Gene Editing

Gene Editing Life Science Gene Gene Therapy

Precision Bio climbs on near-$1.5bn sickle cell pact with Novartis

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

Gene Editing

Gene Editing In-Vivo Gene Sequencing Gene

EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. 14, 2020 10:00 UTC.

Business Development

Business Development Development Gene Editing In-Vivo

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

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

Genetics Medicine Gene Therapy Marketing

Life Science Trends to Look Out for in 2024

XTalks

DECEMBER 20, 2023

In 2024, pharma companies and emerging biotechs will improve candidate safety profiles and reduce off-target effects by exploiting predictive intelligence unlocked by AI,” she predicts. This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease.

Life Science

Life Science Gene Editing Development Clinical Trials

Necessity Is the Mother of Innovation: Key Trends and Predictions from Biotech Showcase Digital After an Unprecedented Year That Drove Momentous Changes in Healthcare

Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

Vaccination

Vaccination Vaccine In-Vivo Genetics

Genome Editing Market Update: Intellia Therapeutics Presents Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

Webinars

Trending Sources

Scribe and Prevail partner for CRISPR-based genetic medicines

Webinars

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

Moderna partners with Life Edit for mRNA gene editing therapies

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

Gene editing: beyond the hype

Scribe and Sanofi partner to develop cell therapies for cancer

Gene Editing Innovations with Programmable Gene Insertion (PGI) Technology ft. John Finn, PhD, CSO, Tome Biosciences – Xtalks Life Science Podcast Ep. 160

Precision Bio climbs on near-$1.5bn sickle cell pact with Novartis

EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Life Science Trends to Look Out for in 2024

Necessity Is the Mother of Innovation: Key Trends and Predictions from Biotech Showcase Digital After an Unprecedented Year That Drove Momentous Changes in Healthcare

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