Gene, Genetic Disease and In-Vivo - Clinical Research Informer

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. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

Researchers reverse the in vitro and in vivo effects of the mutation that causes Stormorken syndrome

Medical Xpress

MAY 16, 2023

They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material. That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo.

In-Vitro

In-Vitro In-Vivo Genetic Disease Genetics

Moderna seals pact with Life Edit Therapeutics to tackle genetic diseases with mRNA-based gene editing

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

Gene Editing

Gene Editing Genetic Disease Gene In-Vivo

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

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

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing

Gene Editing Gene In-Vivo Clinical Trials

Bayer trumpets $1bn CRISPR deal with Mammoth Bio

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo

In-Vivo Gene Editing Gene Therapy Gene

Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

AUGUST 17, 2022

These programmes will include therapies and vaccines in infectious disease and oncology areas. oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. .

RNA

RNA Vaccination Vaccine In-Vivo

Vertex builds in gene editing yet again, with $1.2bn Arbor deal

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

Gene Editing

Gene Editing Gene In-Vivo Genetics

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Delveinsight

FEBRUARY 11, 2021

Avrobio gene therapy eradicates toxic substrate in Fabry patient. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate. Enhancing the response to immuno-oncology in pancreatic cancer.

Gene Therapy

Gene Therapy Gene Immune Response In-Vivo

Sana Bio’s $150 Million IPO Expected to Provide Market Valuation of $10 Billion

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

In-Vivo

In-Vivo Marketing Engineer Genetics

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

“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 Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.

Business Development

Business Development Development Gene Editing In-Vivo

Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

Delveinsight

JANUARY 12, 2021

The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Bluebird spins off to two companies, cleaving off its gene therapy, and cancer units. Further, Birinapant also complements the anti-tumor activity of the immune system.

Licensing

Licensing Licensing Antibody Gene Therapy

Lysogene Reports Positive Biomarker Data With LYS-SAF302

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

Gene Therapy

Gene Therapy Clinical Trials Clinical Trials In-Vivo

ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders

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.

Gene Editing

Gene Editing Gene In-Vivo Genetics

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

The Pharma Data

AUGUST 3, 2021

While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We 2 years and <5 years old with SMA Type 2.

Clinical Trials

Clinical Trials Clinical Trials Gene Therapy Trials

BioSpace Movers & Shakers, Oct. 16

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. BeyondSpring – BeyondSpring Inc.

In-Vivo

In-Vivo Business Development Manufacturing Development

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery.

Drugs

Drugs Gene Clinical Trials Clinical Trials

Clinical Research Informer

Moderna partners with Life Edit for mRNA gene editing therapies

Researchers reverse the in vitro and in vivo effects of the mutation that causes Stormorken syndrome

Trending Sources

Moderna seals pact with Life Edit Therapeutics to tackle genetic diseases with mRNA-based gene editing

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

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

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

Bayer trumpets $1bn CRISPR deal with Mammoth Bio

Merck and Orna partner for RNA technology-based vaccines and therapies

Vertex builds in gene editing yet again, with $1.2bn Arbor deal

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Sana Bio’s $150 Million IPO Expected to Provide Market Valuation of $10 Billion

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

Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

Lysogene Reports Positive Biomarker Data With LYS-SAF302

ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

BioSpace Movers & Shakers, Oct. 16

Advances in neuroscience drug discovery

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