Pharmaceutical Technology

SEPTEMBER 22, 2022

AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein. Cystinosis patients have a high deficiency of this protein. A rare and fatal ailment, cystinosis is characterised by cystine buildup in cellular organelles called lysosomes.

Gene Therapy 147

Gene Therapy Gene Protein Clinical Trials 147

The Pharma Data

JUNE 14, 2023

Overexpression or genetic activation of Cyclin Dependent Kinase 2 (CDK2) binding partner cyclin E is a key oncogenic process in several cancers. Cedilla’s small molecules conditionally modulate the protein complex in its functional state resulting in highly selective inhibition.

Licensing 40

Licensing Licensing Life Science Development 40

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Delveinsight

JANUARY 12, 2021

Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

pharmaphorum

JULY 5, 2022

It can be caused by exposure to loud noises – including prolonged use of earbuds at high volumes – as well as cancer treatment, illnesses, genetic mutations, or aging. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide. An estimated 1.57 An estimated 1.57

In-Vivo 106

In-Vivo Bioinformatics Genetics Licensing 106

The Pharma Data

NOVEMBER 4, 2020

Ltd (“Juyou”), a biotechnology company that develops and sells medical and cosmetic skincare products, for the commercialization and development of Pliaglis ® in mainland China (the “License Agreement”). million and US$1.8

Licensing 40

Licensing Licensing Dermatology Production 40

pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.

RNA 103

RNA Genetics Dermatology Antibody 103

XTalks

MAY 1, 2023

This makes Qalsody the first approved treatment to target a genetic cause of ALS. Tofersen was discovered by Ionis Pharmaceuticals, and Biogen licensed tofersen from Ionis under a collaborative development and license agreement. The recommended dosage of Qalsody is 100 mg (15 mL) per administration. What is SOD1 -ALS?

FDA Approval 89

FDA Approval Gene Genetics Protein 89

pharmaphorum

DECEMBER 2, 2021

It will focus its efforts initially on neurological diseases like Alzheimer’s and Parkinson’s, using technologies that can measure how disease alters tissues, cells and proteins, with the aim of increasing the success rate of drug discovery and development. Milestone payments tied to progress of the two drugs could add another $1.5

Biobanking 81

Biobanking Genome Genomics Genetics 81

XTalks

APRIL 3, 2024

The RBCs in circulation are marked by proteins in the complement system for removal by the spleen and liver. Through its Alexion deal, AstraZeneca is looking to expand into other rare diseases, including various forms of amyloidosis for which it has secured licensing deals and buyouts.

FDA Approval 102

FDA Approval Sales Trials Development 102

Drug Discovery World

JANUARY 15, 2024

The NVIDIA DGX SuperPOD will be installed at Amgen’s deCODE genetics’ headquarters in Reykjavik, Iceland and will be used to build a human diversity atlas for drug target and disease-specific biomarker discovery. of Perspective’s shares for up to $33 million.

Drugs 59

Drugs Licensing Licensing Protein 59

pharmaphorum

DECEMBER 22, 2020

Since then Barron has been busy signing deals for therapies that are validated genetically, hoping that this will make them more likely to succeed in the clinic. The latest deals include a license agreement with San Diego-based Ligand Therapeutics , which has a subsidiary producing technology for neurological disorders.

Licensing 52

Licensing Licensing Genetics Sales 52

Drug Discovery World

AUGUST 8, 2023

The total $50 million will include $25 million to acquire 8,333,333 shares of common stock of Poseida and a one-time $25 million payment for first refusal to license allogeneic CAR-T cell therapy P-MUC1C-ALLO1.

Gene Therapy 52

Gene Therapy Licensing Licensing Genetics 52

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. Translarna was the first licensed treatment for DMD which addresses the loss of dystrophin. Eventually, they will need assistance with breathing.

Gene Therapy 94

Gene Therapy Gene FDA Approval DNA 94

pharmaphorum

JANUARY 22, 2021

According to the US biopharma company, genetic analyses of publicly available sequences for the new variants of SARS-CoV-2 suggest the mutations in the viral spike protein that have made them more transmissible shouldn’t affect the way Veklury (remdesivir) works. The UK strain – known as B.1.1.7

In-Vitro 141

In-Vitro RNA Licensing Licensing 141

XTalks

DECEMBER 20, 2021

It has also applied for regulatory licensing in the UK, European Union (EU), Australia, Singapore, India, United Arab Emirates (UAE), New Zealand and Japan. Covovax is a recombinant protein-based vaccine and can be regarded as a more “traditional” vaccine as it isn’t genetic-based like mRNA or viral vector vaccines.

Vaccination 96

Vaccination Vaccine Manufacturing Protein 96

Drug Discovery World

APRIL 5, 2023

It targets a pathway that cancer cells are highly dependent upon, involving the heat shock factor 1 protein (HSF1). Mechanism of action HSF1 is a transcription factor, meaning that it controls the production of other important proteins in the cell, through its regulation of the process of reading the genetic code stored in DNA.

Drugs 52

Drugs Scientist Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 21, 2021

The world-leading team of academic researchers will use their newly designed technical approach to assess which proteins are selectively produced by the kalirin gene in the human brain, and how this differs from other human tissues. Dr. The significant gap in treatment options leaves many with poor long-term quality of life and disability.

Medicine 111

Medicine Protein Drugs Research 111

pharmaphorum

JUNE 16, 2022

It also probably marks the end of the road for crenezumab, which Roche’s Genentech subsidiary licensed from AC Immune in 2006 in a deal initially valued at $300 million. Before the end of the year, Roche should report results from two phase 3 studies in early-stage patients.

Trials 111

Trials Clinical Trials Clinical Trials Licensing 111

pharmaphorum

NOVEMBER 6, 2020

Novartis noted that the results from this trial do not affect Ilaris’ licensed indications or other trials and the safety profile was comparable to placebo plus standard of care. Novartis is also working with Molecular Partners to develop therapies based on genetically engineered proteins known as DARPins.

Trials 40

Trials Genetic Engineering Licensing Licensing 40

Drug Discovery World

JUNE 1, 2023

The platform technology of Synaffix is capable of consistently converting any antibody into a best-in-class ADC (in terms of efficacy and safety profile) without any engineering of the genetic sequence of the antibody.” 2.

Antibody 98

Antibody Marketing Drugs Licensing 98

pharmaphorum

OCTOBER 29, 2020

The Swiss pharma group already sells one gene therapy for inherited retinal diseases (IRDs) – Luxturna (voretigene neparvovec) – for which it licensed ex-US rights from Spark Therapeutics (now part of Roche) in 2018. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7

Gene Therapy 40

Gene Therapy Gene Gene Silencing Protein 40

pharmaphorum

DECEMBER 21, 2020

As it’s just a messenger molecule, it does not affect the body’s own genetic code when it is injected as a vaccine – but what it does do is instruct cells to code for copies of a certain protein. In this case that is the “spike” protein seen on the surface of the coronavirus that it uses to invade host cells.

Vaccination 93

Vaccination Vaccine Doctors Doctor 93

pharmaphorum

JANUARY 10, 2022

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. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

Drug Discovery World

NOVEMBER 9, 2023

This prognostic benefit is mainly restricted to patients who have ‘hot’ tumours which have multiple genetic mutations and are rich in killer T-cells. CL: The major histocompatibility complex (MHC) traffics fragments of intracellular proteins known as peptides to the cancer cell surface.

Antibody 65

Antibody Protein Development Engineer 65

XTalks

FEBRUARY 19, 2024

Moderna’s vaccine not only targets gB but also incorporates a second target, a five-unit protein complex vital for the virus’ ability to infect epithelial cells in the mouth and nose. After more than 50 years of research, we are closer than ever to having a licensed CMV vaccine,” Permar said.

Vaccination 59

Vaccination Vaccine Trials Clinical Trials 59

pharmaphorum

AUGUST 16, 2020

ChAdOx1 – which is in large-scale phase 2/3 trials – was licensed by AZ in April and renamed AZD1222, and should be ready for first deliveries before the end of the year. It will be provided on a no-profit basis while the pandemic is ongoing.

Vaccination 111

Vaccination Vaccine Immune Response Trials 111

The Pharma Data

MARCH 14, 2021

This is much lower than would be expected to occur naturally in a general population of this size and is similar across other licensed COVID-19 vaccines. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.

Vaccination 52

Vaccination Vaccine Licensing Licensing 52

pharmaphorum

AUGUST 7, 2020

Inhibition of LRRK2 activity may slow the progression of Parkinson’s disease in patients with and without known genetic risks based on restoration of lysosomal function.

Bacteria 69

Bacteria Marketing Licensing Licensing 69

The Pharma Data

MAY 27, 2022

It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein. About AstraZeneca.

Vaccination 52

Vaccination Vaccine Immune Response Medicine 52

pharmaphorum

MAY 14, 2021

Cambridge, Massachusetts-based Envisagenics focuses on using AI to analyse errors in RNA splicing, a process in which a precursor form of messenger RNA (mRNA) – which is used as a template for protein synthesis in cells – is transformed into a mature form.

RNA 60

RNA Licensing Licensing Genetics 60

The Pharma Data

APRIL 12, 2023

Nasdaq: DNLI) today announced that Biogen has exercised the option to license Denali’s Antibody Transport Vehicle (ATV): Amyloid beta program (ATV:Aβ). We are pleased with Biogen’s decision to license ATV: Aβ and we are hopeful this will foster the development of next-generation anti- Aβ therapeutics.”

Antibody 40

Antibody Development Engineer Licensing 40

pharmaphorum

JANUARY 31, 2022

It’s a setback to Pfizer’s late-stage pipeline, given that at one point the company was projecting $3 billion in potential sales for vupanorsen, which targets ANGPTL3 protein and is playing catch-up in the category with Regeneron’s already-marketed Evkeeza (evinacumab).

Production 52

Production Vaccination Vaccine Trials 52

The Pharma Data

NOVEMBER 17, 2021

It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein. COVID-19 Vaccine AstraZeneca is known as Vaxzevria in Europe. About AstraZeneca.

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

Delveinsight

JANUARY 7, 2021

The immunotherapy pipeline is led by an in-licensed EP4 antagonist that Ikena is testing combined with Keytruda in phase 1b/2 colorectal and non-small cell lung cancer clinical trials. Biotheranostics’ two PCR-based genetic tests are well established in the U.S.: Ikena will back a pipeline that falls into two categories.

RNA 52

RNA Clinical Trials Clinical Trials Bioavailability 52

XTalks

JULY 25, 2022

Byondis announced that the US Food and Drug Administration (FDA) accepted their Biologics License Application (BLA) for [vic-]trastuzumab duocarmazine (SYD985), an investigational therapy being studied to treat HER2-positive unresectable locally advanced or metastatic breast cancer.

Antibody 59

Antibody Drugs Clinical Trials Clinical Trials 59

The Pharma Data

DECEMBER 9, 2020

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. which will remain a key shareholder. SciNeuro Pharmaceuticals. It’s like taking out the trash.

Marketing 52

Marketing In-Vivo Protein In-Vitro 52