XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Gene Therapy Gene FDA Approval Antibody 98

Pharmaceutical Technology

MAY 9, 2023

FibroGen has signed an exclusive licence agreement with Fortis Therapeutics for the FOR46 antibody-drug conjugate (ADC) that targets a new epitope on CD46, a protein-coding gene. FOR46 is currently being investigated in a Phase I trial as a potential treatment for metastatic castration-resistant prostate cancer (mCRPC).

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Drugs Clinical Development Antibody Protein 130

Pharmaceutical Technology

AUGUST 23, 2022

It comprises messenger ribonucleic acid (mRNA) that encodes for the initial spike protein of SARS-CoV-2 along with mRNA that encodes for Omicron BA.4/BA.5 5 variant spike protein. According to pre-clinical findings, a booster dose of this vaccine elicited a robust neutralising antibody response against the variants Omicron BA.1,

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Vaccination Vaccine Gene Therapy Protein 130

pharmaphorum

APRIL 21, 2022

The pharma company has decided to end its collaboration with Swedish biotech BioArctic on alpha-synuclein-targeting antibody ABBV-0805, which was in early-stage clinical testing, as well as a portfolio of follow-up antibodies. AZ/Takeda are meanwhile running phase 2 trials of their MEDI 1342 antibody candidate.

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Antibody Protein Gene Therapy Pharma Companies 97

XTalks

MAY 9, 2024

Genmab, a trailblazer in the field of antibody therapeutics, celebrates this milestone by reflecting on its impressive journey from a small-scale startup to a global leader in cancer treatment and beyond. Since then, innovative science has been at Genmab’s core, as we harness the power of human antibodies to improve the lives of patients.

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Antibody Clinical Trials Clinical Trials Medicine 52

XTalks

OCTOBER 12, 2021

14-3-3 zeta) protein, which the UToledo researchers had been studying for its role in immune-related pathologies, such as aortic aneurysms and in signaling pathways involving the pro-inflammatory cytokine interleukin-17. However, they found the opposite to be true, where the protein was actually protective against development of the disease.

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Vaccination Vaccine Research Immune Response 105

Roots Analysis

NOVEMBER 30, 2021

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies. How Players are Complying with Developing Market?

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Bioassay Gene Therapy Gene Development 52

XTalks

OCTOBER 22, 2024

Vyloy is a first-in-class monoclonal antibody that targets the CLDN18.2 transmembrane tight junction protein, which is commonly found in some gastric and GEJ cancers. gene, and an estimated 60 percent of all gastric cancers harbor the protein. targeting antibody-drug conjugate. On the other hand, Merck & Co.

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FDA Approval Drugs Licensing Licensing 105

pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.

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Gene Therapy Gene Manufacturing Contract Manufacturing 52

pharmaphorum

FEBRUARY 3, 2022

Al Sandrock’s shock departure from Biogen in November sparked speculation about where he would show up next, and the answer came in today – gene therapy specialist Voyager Therapeutics. Dr. Al Sandrock has joined our Board of Directors & Executive Committee to help Voyager shape the future landscape of AAV #gene therapy.

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Gene Therapy Gene Clinical Trials Clinical Trials 97

pharmaphorum

NOVEMBER 2, 2021

Dr Laura Moriarty, senior marketing manager at Bio-Rad, looks at the impressive immuno-therapeutic potential of bispecific antibodies (bsAbs). Though their bispecific nature complicates large-scale production and purification workflows, with challenges such as antibody chain mispairing, bsAbs have come a long way since first developed.

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Antibody Immune Response Production Engineer 52

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease. was swooped up by Real Chemistry in January.

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Vaccination Vaccine Gene Therapy RNA 98

pharmaphorum

JANUARY 5, 2022

The FDA thinks an antibody-drug conjugate (ADC) developed by AbbVie could set new standards in treatment for certain patients with lung cancer, awarding the drug breakthrough status.

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Antibody Drugs Protein Gene 111

The Pharma Data

NOVEMBER 2, 2020

2, 2020 — An experimental antibody drug that targets one of the body’s key metabolism regulators may help obese people lose weight — at least briefly. It would take much more research to prove the antibody is safe and effective, Ryan said. MONDAY, Nov. She was not involved in the study.

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Antibody Drugs Hormones Insulin 52

Pharmaceutical Technology

MARCH 1, 2023

The drug candidate targets DNA-dependent protein kinase (DNA-PK). Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems. The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

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Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

The drug candidate targets DNA-dependent protein kinase (DNA-PK). Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems. The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

Delveinsight

DECEMBER 22, 2020

FDA Puts uniQure’s Hemophilia B Gene Therapy on Leash. In a recent, uniQure faced backlash after its hemophilia B gene therapy got hit with clinical hold by the USFDA. . It substantially increased the production of the blood-clotting protein factor IX, which is the factor missing for hemophilia B patients, in all the enrolled patients.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

pharmaphorum

MARCH 9, 2022

Sanofi and Sobi’s haemophilia partnership has been under competitive pressure from new therapies like Roche’s antibody Hemlibra, so the two companies are hoping a new long-acting drug candidate can revitalise the franchise. The half life of the new drug – one of the main draws for Sanofi’s $11.6

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Protein Gene Therapy Sales Regulation 98

Delveinsight

AUGUST 6, 2020

ZyCoV-D is designed to introduce DNA that encodes for a viral protein into human cells. By ordering human cells to make the protein, ZyCoV-D could cause an immune response against the coronavirus. Taysha raises USD 95 Million to take 4 CNS gene therapies into the clinic.

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Allergies Vaccination Vaccine Gene Therapy 53

pharmaphorum

NOVEMBER 30, 2020

DLB and PD are both so-called synucleinopathies, diseases that are characterised by mutations in the SNCA gene coding for alpha-synuclein, leading to the production of misfolded, toxic forms of the protein that clump together and are thought to damage nerve cells. Denali’s project is earlier in development.

Antibody 52

Antibody Protein Trials Drugs 52

BioTech 365

SEPTEMBER 2, 2020

(Nasdaq: SRNE, “Sorrento”) and SmartPharm Therapeutics, Inc. (“SmartPharm”) announced today that Sorrento has completed the acquisition of SmartPharm, a gene-encoded protein therapeutics company developing non-viral DNA and … Continue reading →

DNA 40

DNA In-Vivo Protein Gene 40

Roots Analysis

DECEMBER 9, 2021

The phosphorylation of the intracellular tyrosine kinase activates intracellular signaling, thereby, leading to the activation of genes responsible for cellular growth. The functional activation of HER2 promotes oncogenesis, which further leads to the investigation of HER2-directed agents in cancers with HER2 alterations.

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Marketing Development Gene Protein 52

XTalks

SEPTEMBER 17, 2020

Inherited mutations in the SLC3A1 and SLC7A9 genes cause cystine to be abnormally transported within the kidney leading to cystinuria. Dietary changes, including restriction of salt and animal protein, are also recommended. Mutations in the gene encoding ?-Gal Prevalence: ABCG5 gene mutation: 1 in 2.6 galactosidase A (?-Gal

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Trials Gene Hormones Clinical Trials 98

pharmaphorum

MARCH 31, 2021

The diagnostic imaging agents all target fibroblast activation protein (FAP), a molecule that is only expressed at low levels on healthy cells but is found much more abundantly in solid tumours. In 2018 it paid $2.1 “We believe this technology has the potential to transform many patients’ lives,” she added.

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Licensing Licensing Gene Therapy Sales 98

pharmaphorum

AUGUST 17, 2020

With Massachusetts-based Dragonfly BMS is taking a different tack and focusing on DF6002, a long-lasting fusion protein that looks like the “tail” (Fc) end of an IL-12 antibody. Ziopharm is using a gene therapy to instruct brain tumours to produce IL-12, then hitting them with a PD-1 checkpoint inhibitor.

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Gene Therapy Clinical Trials Clinical Trials Gene 59

Find Me Cure

OCTOBER 13, 2019

HER2 is a protein that plays an important role in cancer cell growth and is found on the surface of cancer cells in a very high quantity in about 10-20% of breast cancers. There are antibody drugs targetting those cells that are already FDA approved and there are antibody drugs still in trials.

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Development DNA Clinical Trials Clinical Trials 52

pharmaphorum

AUGUST 7, 2020

Mutations in the LRRK2 gene can cause Parkinson’s disease. Denali’s TV platform is a proprietary technology designed to effectively deliver large therapeutic molecules such as antibodies, enzymes, proteins and oligonucleotides across the blood-brain barrier after intravenous administration.

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Bacteria Marketing Licensing Licensing 69

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Roche’s Chief Medical Officer and Head of Global Product Development.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

The Pharma Data

JULY 6, 2021

GlaxoSmithKline plc (LSE/NYSE: GSK) and Alector (Nasdaq: ALEC), today announced a strategic global collaboration for the development and commercialisation of two clinical-stage, potential first-in-class monoclonal antibodies (AL001 and AL101) designed to elevate progranulin (PGRN) levels.

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Genetics Gene Antibody Development 52

The Pharma Data

JANUARY 7, 2021

To meet that goal, Sanofi is investing in areas like synthetic biology and cell and gene therapy. He also noted innovations are occurring with protein degraders, antibody conjugates, nanobodies and multi-specific antibodies, specifically bi- and tri-specific. billion acquisition of Synthorx.

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Protein Antibody Genetics Gene Therapy 52

Delveinsight

AUGUST 27, 2020

As observed in 45 younger adults, the middle dose of the vaccine triggered the production of neutralizing antibodies against SARS-CoV-2, the virus, which causes COVID-19 , in 10 patients aged 56 to 70 and 10 patients over 71. The BET inhibitor worked by increasing beta-3 tubulin (TUBB3), a protein found in brain metastases.

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Vaccination Vaccine Research Trials 53

XTalks

JUNE 9, 2022

When a child born from parents with FH genes, inherits one FH gene from each parent, they might develop homozygous familial hypercholesterolemia (HoFH). Evkeeza — generically known as evinacumab — is a monoclonal antibody. The ANGPTL3 protein inhibits the enzyme activity of lipoprotein lipase (LPL).

Trials 52

Trials Antibody Genetics Gene 52

The Pharma Data

OCTOBER 14, 2020

15, 2020 /PRNewswire/ — Bio-Techne Corporation (NASDAQ: TECH), a leading provider of proteins, antibodies and cytokines today announced that its Woburn, Massachusetts site received ISO 13485:2016 certification for Medical Device and Quality Management Systems. MINNEAPOLIS , Oct.

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Manufacturing Reagent Gene Therapy Protein 40

pharmaphorum

AUGUST 31, 2022

The study also found that the drug was well-tolerated, with no evidence for the emergence of inhibitors – antibodies that can be stimulated in the body against replacement Factor VIII and lead to a reduction in its ability to prevent bleeds. The drug was one of the primary drivers for Sanofi’s $11.6

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Drugs Protein Marketing Gene Therapy 52

The Pharma Data

MARCH 11, 2021

Clinical trial to assess safety, immune response and reactogenicity, after preclinical data showed high neutralizing antibody levels. Preclinical data showed that two immunizations of the mRNA vaccine induced high neutralizing antibody levels that are comparable to the upper range of those observed in infected humans.

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Clinical Trials Clinical Trials Vaccination Vaccine 52

The Pharma Data

DECEMBER 13, 2020

HMBD-001 is a uniquely differentiated anti-HER3 neutralizing antibody that was developed using Hummingbird’s proprietary Rational Antibody Discovery platform. Cancer Research UK has partnered with Hummingbird Bioscience to advance this novel antibody drug into clinical trials for the treatment of HER3-driven cancer.

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Clinical Development Medicine Development Antibody 40