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

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 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. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

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

LNP formulations produce strong immune responses, data shows

Drug Discovery World

FEBRUARY 15, 2024

The etherna paradigm for cLNP design is ushering in the rapid generation of novel families of delivery systems for not only vaccination but also for in vivo tolerisation for a host of autoimmune diseases.”

Immune Response

Immune Response In-Vivo Vaccination Vaccine

€2m raised in seed funding to develop novel therapies for Cystic Fibrosis

Drug Discovery World

JUNE 19, 2023

Anoat will use the funds to conduct additional in vivo and in vitro pharmacology assessments and select a lead candidate. We are thrilled to have co-founded this first company with Inserm Transfert, bringing together state-of-the-art science developed by Inserm researchers and leading biotech development experts.

Development

Development Life Science In-Vivo In-Vitro

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

pharmaphorum

AUGUST 24, 2021

The latest deal allows Vertex to tap into Arbor’s technology platform to develop cell therapies for diseases like type 1 diabetes and blood disorders sickle cell disease (SCD) and beta thalassaemia. If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

Gene Editing

Gene Editing Gene In-Vivo 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

Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. Prior to that, he held various oncology research and development positions at Merrimack Pharmaceuticals and Archemix. Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.

Business Development

Business Development Development Gene Editing In-Vivo

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

Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. . Enhancing the response to immuno-oncology in pancreatic cancer. They reported their observations in the journal JCI Insight.

Gene Therapy

Gene Therapy Gene Immune Response 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 bio recently announced its plans to split its genetic disease and oncology businesses. and Europe.

Licensing

Licensing Licensing Antibody Gene Therapy

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

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 Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019.

Clinical Trials

Clinical Trials Clinical Trials Gene Therapy Trials

Transitioning Between Academia and Industry: Advice from Leading Scientists That Made the Switch

XTalks

OCTOBER 1, 2020

WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation. Lin has made several transitions throughout her career, from being a clinician at an academic center, to academic research, to heading business development in industry. Motivations for Transition. Key Moments.

Scientist

Scientist In-Vitro Research Life Science

BioSpace Movers & Shakers, Oct. 16

The Pharma Data

OCTOBER 15, 2020

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. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

In-Vivo

In-Vivo Business Development Manufacturing Development

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8 to £104.7m

Drugs

Drugs Gene Clinical Trials Clinical Trials

Clinical Research Informer

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

Moderna partners with Life Edit for mRNA gene editing therapies

Trending Sources

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

LNP formulations produce strong immune responses, data shows

€2m raised in seed funding to develop novel therapies for Cystic Fibrosis

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

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

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

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

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

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

Transitioning Between Academia and Industry: Advice from Leading Scientists That Made the Switch

BioSpace Movers & Shakers, Oct. 16

Advances in neuroscience drug discovery

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