XTalks

MARCH 28, 2024

Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein. Duvyzat will be competing with Sarepta Therapeutics’ bag of exon-skipping DMD therapies, which include Exondys 51 (eteplirsen), Vyondys (golodirsen) and Amondys (casimersen).

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FDA Approval Genetics Gene Therapy Gene 105

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Clinical Trials Clinical Trials Vaccination Vaccine 75

pharmaphorum

JANUARY 13, 2022

Clinical trials will be needed to see if doing so can interrupt the neurodegeneration seen in Parkinson’s. Alpha-synuclein is a protein that gets misfolded and clumps together in the brains of patients with Parkinson’s and other diseases collectively known as synucleinopathies.

Antibody 98

Antibody Drugs Protein Licensing 98

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

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Gene Therapy Drugs Gene Manufacturing 52

The Pharma Data

DECEMBER 31, 2020

where he led global clinical development programs including clinical strategy, clinical development, and regulatory affairs. Prior to Taiho, he held the positions of senior vice president of Clinical Oncology and CMO at Geron Corporation. and CMO for Taiho Oncology, Inc.

Gene Therapy 52

Gene Therapy Engineer Gene Research Analyst 52

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 52

In-Vivo Gene Editing Trials Gene 52

pharmaphorum

OCTOBER 13, 2022

million in future payments tied to preclinical, clinical development, and commercial achievements. Vividion will contribute its ability to find previously unknown functional binding pockets on molecules like proteins or transcription factors that can be targeted with drug compounds. Vividion is making an upfront payment of $17.5

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Gene Therapy Genome Genomics Drugs 64

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 removal of an AdComm was seen as a positive. J&J has agreed to pay USD 52.50

Gene Therapy 52

Gene Therapy Research Gene Antibody 52

pharmaphorum

APRIL 15, 2021

There are several treatment options now available for SMA, with Novartis’ ultra-pricey Zolgensma offering a one-off gene therapy, while Biogen’s antisense drug Spinraza instructs the body to produce a working version of the SMN protein that is the root cause of the disease.

Gene Therapy 52

Gene Therapy Protein Clinical Development Regulation 52

XTalks

OCTOBER 22, 2020

In this sense, pharmaceutical companies have developed different strategies to produce a wide variety of advanced therapy medicinal products (ATMP) and biological products, such as monoclonal antibodies, recombinant proteins, gene therapy viral vectors, CAR-T cells and oncolytic viruses, in order to deliver new medical needs in our society.

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Manufacturing Development Production Gene Therapy 98

Drug Discovery World

NOVEMBER 24, 2022

The US Food and Drug Administration (FDA) has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with haemophilia B. . The FDA approval is supported by results from the ongoing HOPE-B trial, the largest gene therapy trial in haemophilia B to date.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. This includes support for export and expansion into new territories as well as for innovation and development.

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Drugs Life Science Gene Therapy Genome 75

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

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Development Gene Therapy Protein Gene 246

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.

Gene Therapy 52

Gene Therapy Gene Manufacturing Contract Manufacturing 52

The Pharma Data

OCTOBER 27, 2020

“Once we’re in the clinic, we will continue to add multiple programs into the clinic with no rate abatement, or speed abatement, in the next five- to 10-year horizon.” . Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBio .

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Gene Gene Therapy Medicine Genetics 52

Drug Discovery World

FEBRUARY 22, 2023

The multi-year clinical development of Hemgenix was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment. The post EU-approved gene therapy reduced haemophilia bleeds by 64% appeared first on Drug Discovery World (DDW).

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

XTalks

MAY 9, 2024

Second, new modalities, such as antibody-drug conjugates, bispecific antibodies and cell and gene therapies, have shown great promise, offering potential in previously difficult-to-treat diseases, including advanced cancer and rare diseases.

Antibody 52

Antibody Clinical Trials Clinical Trials Medicine 52

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.

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Gene Therapy Gene Trials Development 96

Delveinsight

JULY 30, 2020

Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. Adrenoleukodystrophy Pipeline.

Hormones 52

Hormones Marketing Gene Trials 52

The Pharma Data

JANUARY 27, 2021

Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. PGRN is a complex and highly conserved protein.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

JUNE 26, 2021

The safety profile of PDS in the clinical trial setting is well understood and will continue to be closely monitored. If approved, PDS would be the first and only nAMD therapy indicated to allow six months between treatments. Portal is an extension study evaluating the long-term safety and efficacy of PDS in nAMD.

Drug Delivery 52

Drug Delivery Clinical Trials Clinical Trials Medicine 52

Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

Protein 59

Protein Antibody Engineer Scientist 59

pharmaphorum

JANUARY 16, 2023

This is linked to wider questions about the role damaged proteins and their build up plays in ageing across different tissues and organs. Gene therapies, some of which are already in market, will see a strong uptick in 2023. Ferring squeezed a global first for the approval of its faecal microbiota therapy Rebyota for C.

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Gene Therapy In-Vitro Gene Medicine 98

Pharmaceutical Technology

AUGUST 26, 2008

. “The inherent advantages of biopharma are that it allows you to pursue therapies against targets that are not small molecule therapies, for example by targeting protein-protein interactions and targeting GPCRs (G-protein coupled receptors),” he says.

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The Pharma Data

AUGUST 25, 2020

senior vice president, Global Clinical Development, Hematology, Bristol Myers Squibb. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies.

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Trials FDA Approval Development Clinical Trials 52

The Pharma Data

MARCH 20, 2023

2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry. Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

pharmaphorum

AUGUST 10, 2020

for a single shot of the gene therapy, making it the most expensive drug in the world by many people’s reckoning, although the manufacturer argues this is justified given the costs of treating the condition in later life. Evrysdi is designed to treat SMA by increasing production of the survival of the motor neuron (SMN) protein.

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FDA Approval Drugs Gene Therapy Protein 59

The Pharma Data

AUGUST 15, 2021

Vivet Therapeutics (“Vivet”), a clinical-stage biotechnology company, and Pfizer Inc. The FDA’s Fast Track program is designed to facilitate the development, and expedite the review of, novel potential therapies that are designed to treat serious conditions and fill unmet medical need. NYSE: PFE) today announced the U.S.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

SEPTEMBER 12, 2022

Providers are creating individualised treatment plans for their patients based on their genes, proteins, or other bodily substances, resulting in better, faster, and more effective patient outcomes.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

Drug Discovery World

MAY 30, 2023

At PhoreMost, we have been particularly excited about the emergence of targeted protein degradation (TPD) as a new therapeutic modality. The concept of harnessing the ubiquitin-proteosome system to ‘destroy’ or degrade disease causing proteins has been established for some time.

Research 52

Research Drugs Protein RNA 52

Drug Discovery World

OCTOBER 17, 2023

Today, spatial omics technologies enable imaging-based identification of cells in their native tissue context while simultaneously detecting hundreds of RNAs or tens of protein markers in the same cells. Spatial omics technologies are ideally suited for characterising cells and their interactions within a tissue.

Gene Expression 52

Gene Expression Drugs Protein Gene 52

The Pharma Data

OCTOBER 14, 2020

About VBL’s VB-600 Platform VBL is conducting two parallel drug development programs that are exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases and cancer.

Antibody 52

Antibody Gene Therapy Containment Clinical Trials 52

The Pharma Data

SEPTEMBER 15, 2020

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. 20vPnC showed a safety and tolerability profile that was similar to Prevnar 13 ® Pneumococcal 13-valent Conjugate Vaccine [Diphtheria CRM197 Protein].

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Gene Therapy Vaccination Vaccine Trials 52

XTalks

AUGUST 2, 2023

Stelara’s patents are expiring in September 2023, while a handful of biosimilar drugs are in late-stage development or seeking FDA approval. 2) Darzalex (daratumumab) Darzalex is a targeted monoclonal antibody that slows or stops the progression of multiple myeloma (a blood cancer) by binding to the CD38 protein on multiple myeloma cells.

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Sales Manufacturing FDA Approval Life Science 98

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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Clinical Development Gene Therapy Development Gene 52

Pfizer

OCTOBER 19, 2022

Monovalent refers to any authorized and approved COVID-19 vaccine that contains or encodes the spike protein of only the Original SARS-CoV-2 virus. Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment. COMIRNATY® (COVID-19 Vaccine, mRNA). INDICATION.

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Vaccination Vaccine Clinical Trials Clinical Trials 40

Pfizer

SEPTEMBER 28, 2022

Monovalent refers to any authorized and approved COVID-19 vaccine that contains or encodes the spike protein of only the Original SARS-CoV-2 virus . Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment. COMIRNATY® (COVID-19 Vaccine, mRNA) INDICATION.

Vaccination 101

Vaccination Vaccine Medicine Clinical Trials 101