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

pharmaphorum

DECEMBER 7, 2021

Eplontersen – previously known as IONIS-TTR-L RX – is designed to switch off the production of transthyretin (TTR), a protein which builds up in the disease to toxic levels causing heart problems (cardiomyopathy) and nerve damage (polyneuropathy). billion in milestones depending on sales levels if the antisense-based drug reaches the market.

Licensing 59

Licensing Licensing Drugs Marketing 59

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 110

In-Vivo Bioinformatics Genetics Licensing 110

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

XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions. Priced at $3.5

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

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 105

RNA Genetics Dermatology Antibody 105

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 93

Biobanking Genome Genomics Genetics 93

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

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.

Vaccine 111

Vaccine Vaccination Doctors Doctor 111

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.

Vaccine 96

Vaccine Vaccination Protein Manufacturing 96

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 98

Gene Therapy Gene FDA Approval DNA 98

XTalks

DECEMBER 13, 2023

3) Fulgent Genetics Compound annual growth rate: 260 percent Fulgent Genetics, headquartered in California, is a technology-driven company with a strong presence in clinical diagnostics and therapeutic development. The company achieved core revenues of $67 million, reflecting a remarkable 48 perecent year-over-year growth.

Genetics 111

Genetics Vaccine Vaccination DNA 111

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.

Vaccine 111

Vaccine Vaccination Immune Response Trials 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

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

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

Velocity Clinical Research

AUGUST 8, 2024

The presence of amyloid proteins has been known since the disease was named, and the initial thinking was that their build-up caused it. We understand the tau protein, that some people have inflammation in the brain or a genetic predisposition. But now we know it’s not just that,” explains Dr. Cupelo. “We

Clinical Trials 105

Clinical Trials Clinical Trials Genetics Clinical Research 105

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

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

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

JANUARY 3, 2023

Haemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot because of a deficiency in one or several blood clotting factors. SPK-9001 is a novel, investigation vector containing a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity human coagulation FIX gene.

Gene Therapy 98

Gene Therapy Gene Medicine Trials 98

The Pharma Data

OCTOBER 14, 2020

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

Protein 52

Protein RNA Production Genetics 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

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

pharmaphorum

OCTOBER 18, 2022

Mutations in the gene – which codes for a protein in the inner hair cells of the ear – are seen in around 1% to 5% of deafness cases depending on the population studied, with an estimated 200,000 people affected worldwide. Lilly said there’s no guarantee the CVRs will be paid, and that the payments will shrink by $0.42

Gene Therapy 52

Gene Therapy Gene Genetics FDA Approval 52

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

JANUARY 10, 2022

There are still licensing deals to be done in the COVID-19 category it seems, after Novartis paid around $163 million to license rights to an antiviral drug developed by Molecular Partners. Ensovibep has the potential to become the first multi-specific antiviral molecule for the treatment of COVID-19, according to Novartis.

Trials 52

Trials Drugs Antibody Genetic Engineering 52

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.

Vaccine 52

Vaccine Vaccination Licensing Licensing 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

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

XTalks

SEPTEMBER 17, 2020

Dietary changes, including restriction of salt and animal protein, are also recommended. Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males. What Is a Surrogate Endpoint? Lipodystrophy.

Trials 98

Trials Gene Hormones Clinical Trials 98

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.

Vaccine 52

Vaccine Vaccination Immune Response Medicine 52

pharmaphorum

DECEMBER 13, 2021

In November, it signed a licensing deal with Chinese tech giant Baidu to partner on its artificial intelligence platform, which has been developed specifically to optimise mRNA sequences for mRNA-based therapeutics. The Massachusetts-based biotech company uses AI to analyse errors in RNA splicing, which can result in genetic disorders.

RNA 105

RNA Vaccine Vaccination Development 105

pharmaphorum

SEPTEMBER 18, 2020

The same year, AZ also entered into its third collaboration agreement with Astex Therapeutics, this time to develop small-molecule protein kinase B (PKB) to further contribute in the fight against cancer. The AZD1222 vaccine was invented by the University of Oxford and its spin-out company Vaccitech, then later licensed by AZ.

Pharma Companies 116

Pharma Companies Sales Vaccination Vaccine 116

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 Vaccine Vaccination Drugs 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

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