XTalks

JANUARY 21, 2025

The liquid portion of the blood (serum or plasma) containing antibodies is separated and tested using the Liason Biotrin parvovirus B19 IgG plus test on a Liason XL analyzer. If parvovirus B19 antibodies are present, they bind to the chemicals and particular particles in the test, producing a light signal.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

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. . The Hemophilia B market has several therapies, however, no therapy has been able to kill the disease from its root.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 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.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

Delveinsight

FEBRUARY 11, 2021

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio gene therapy eradicates toxic substrate in Fabry patient. Enhancing the response to immuno-oncology in pancreatic cancer. Median overall survival in those patients was 5.8

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Delveinsight

JULY 28, 2020

AstraZeneca has inked an oncology deal with Daiichi Sankyo to jointly develop and commercialize an antibody-drug conjugate DS-1062 (trastuzumab deruxtecan), worldwide except in Japan. No relief to Solid Biosciences as FDA demands more data to lift the hold on the company’s DMD gene therapy trial.

Gene Therapy 52

Gene Therapy Trials Gene Vaccination 52

Pharmaceutical Technology

APRIL 24, 2023

In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. This design protects antibodies from the innate immune system.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

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. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

Gene Editing 64

Gene Editing Gene Genome Genomics 64

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

pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease. The confounder for both GBT and Novartis’ hopes for their drug could be genetic therapies for SCD, which offer a one-shot treatment for the disease.

Drugs 59

Drugs Gene Therapy Gene Editing Gene 59

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). Hemlibra is a bispecific factor IXa- and factor X-directed antibody.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Scienmag

MAY 17, 2021

Patients’ genetic profile strongly influences size of effect in early-phase trial People with extremely high levels of triglycerides (a type of fat in the blood) and a specific genetic profile saw a substantial reduction in triglycerides after taking the human monoclonal antibody evinacumab compared with those taking a placebo, in a study presented (..)

Genetics 42

Genetics Antibody Trials Gene Therapy 42

pharmaphorum

JUNE 7, 2022

Alectos’ drug helps to maintain the acid pH level in lysosomes required for their function in the hope of restoring their activity and – hypothetically at least – slow the progression of Parkinson’s in patients with and without known genetic risks. Along with the upfront payment, Biogen has pledged up to $77.5

Licensing 80

Licensing Licensing Gene Therapy Genetics 80

Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. The team successfully protected HILOs from the immune system without genetic manipulation. J&J has agreed to pay USD 52.50

Gene Therapy 52

Gene Therapy Research Gene Antibody 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.

Protein 52

Protein Antibody Genetics Gene Therapy 52

XTalks

JUNE 29, 2022

XTALKS WEBINAR: Live Cell Metabolic Analysis — Paving the Way for Metabolic Research and Cell & Gene Therapy. Register for this free webinar to learn how live cell metabolic analysis paves the way not only for metabolic research, but also the manufacturing of significant cell and gene therapy (CGT) products.

FDA Approval 52

FDA Approval Gene Therapy Antibody Gene 52

The Pharma Data

AUGUST 7, 2020

As the pharma industry stands firm in its commitment to advance the sector to fight Covid-19, news has emerged from the European Commission who intend to streamline the development of therapies using genetically modified organisms to treat Covid-19. NIH tests antibodies in Covid-19 clinical trial alongside Eli Lilly and AbCellera .

Clinical Trials 52

Clinical Trials Clinical Trials Gene Trials 52

XTalks

JUNE 4, 2024

Targeting B Cell-Driven Autoimmunity The company’s focus on diseases such as lupus nephritis, multiple sclerosis and myasthenia gravis is driven by the recognition that B cells, a subset of lymphocytes responsible for antibody production, play a pivotal role in these conditions.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Antibody 52

XTalks

NOVEMBER 1, 2024

Price of Ocrevus: $78,858 annually Why it sold so well: Ocrevus is a therapeutic monoclonal antibody that offers a unique scientific approach to treating MS. In 2020, Novartis Gene Therapies launched an innovative early access program for Zolgensma, designed to facilitate prompt patient access.

FDA Approval 85

FDA Approval Drugs Botox Sales 85

The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Goater is CEO at Surface Oncology, an immuno-oncology company developing next-generation antibody therapies.

Development 52

Development Gene Therapy Engineer Life Science 52

XTalks

MAY 17, 2024

Dr. Philipson’s transition to the biotech sector began with a position at UK-based biotech Trizell, where his efforts culminated in a gene therapy FDA submission for bladder cancer. Reflecting on the evolution of rare disease treatments, Dr. Philipson emphasizes the critical role of understanding disease biology and genetics.

Development 52

Development Drugs Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LEXINGTON, Mass., About LogicBio Therapeutics.

Clinical Trials 40

Clinical Trials Clinical Trials Gene Editing Genome 40

XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. What About Small Proteins and Antibodies Reaching Extracellular CNS Targets? Reporter genes to understand dose and distribution.

Trials 83

Trials Drug Delivery Drugs Gene 83

Roots Analysis

APRIL 15, 2024

Over the years, these large molecules have demonstrated promising therapeutic outcomes in the treatment of myriad of chronic and challenging disorders, including autoimmune disorders, infectious diseases, genetic disorders, and oncological disorders.

Manufacturing 40

Manufacturing Production Contract Manufacturing Contamination 40

Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

Genome 244

Genome Genomics Medicine Gene Therapy 244

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

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.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

Gene 262

Gene Gene Therapy Genetics DNA 262

XTalks

APRIL 12, 2021

Like other neurodegenerative disorders, such as Alzheimer’s disease and dementias, Parkinson’s disease research has been immensely challenging for biotechs, with a high rate of late-stage attrition in trials leading to a lack of disease-modifying therapies being approved. Axovant Gene Therapies. Prevail Therapeutics.

Gene Therapy 96

Gene Therapy Gene Trials Development 96

Delveinsight

DECEMBER 3, 2020

Roche receives FDA authorization for the quantitative COVID-19 antibody test. Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Janssen buys a gene therapy asset.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. The company has developed various assets based on lentivirus vectors.

Gene Therapy 246

Gene Therapy DNA Gene Genetics 246

FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

XTalks

APRIL 1, 2025

Qfitlia is also the first antithrombin-lowering therapy approved for routine prophylaxis in hemophilia A or B. Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Among these are three gene therapies.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Delveinsight

FEBRUARY 18, 2021

Bluebird suspends gene therapy trials after two sickle cell patients develop cancer. Bluebird bio has stopped two clinical trials of its sickle cell disease gene therapy after participants developed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Cortexyme tripped up by FDA hold over liver toxicity.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Delveinsight

DECEMBER 8, 2020

As its lead program, KaliVir is developing VET2-L2 leveraging its unique technology platform based on a genetically modified vaccinia virus. Canadian antibody-drug discovery platform AbCellera Biologics is setting the groundwork for a massive Initial public offering worth USD 391 million. million from the Canadian government. .

Licensing 52

Licensing Licensing Gene Therapy Antibody 52

Cloudbyz

MARCH 6, 2024

This allows for the creation of highly specific and targeted therapies that can modulate biological pathways with remarkable precision. Personalized Medicine: Biologics offer the potential for personalized treatment approaches by taking into account individual variations in genetics, immune responses, and disease characteristics.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

pharmaphorum

AUGUST 8, 2022

As we reported this morning, the deal gives Pfizer already-approved SCD therapy Oxbryta (voxelator) – which industry watchers reckon could see a dramatic uptick in sales with Pfizer’s marketing muscle – plus a phase 3 antibody candidate, a phase 1 follow-up to Oxbryta that could offer improved dosing.

Marketing 111

Marketing Sales Gene Editing Antibody 111

pharmaphorum

AUGUST 22, 2022

” For example, Torrance recently met with an expert from the Broad Institute who has been looking into gene therapies and says gene therapy can completely change how drugs are built. The Fund has owned Genmab , a Danish biotechnology company that creates antibody therapies for cancer, for 12 years.

Gene Therapy 102

Gene Therapy Antibody Gene Medicine 102