Drug Discovery World

AUGUST 28, 2023

It is the method for removing unwanted debris, e.g., old proteins, and breaking them down so their constituent parts can be used again. The ones we have focussed on, because the data is so promising and the opportunity so big, are neurodegenerative disorders, in particular Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS).

Genetics 52

Genetics Protein Genetic Disease Clinical Trials 52

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. PK Planning for Gene Therapy Development Programs. Exposure-Response Considerations.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

OCTOBER 4, 2020

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Gene Therapy 98

Gene Therapy Gene Genetic Disease Protein 98

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

Genetic Disease 104

Genetic Disease Protein RNA Doctors 104

Drug Discovery World

FEBRUARY 28, 2024

Each of the three vaccines in Tri-Ad5 targets different proteins associated with precancer and cancer cells. The vaccine combination is studying whether activation of dendritic cells and training the immune system to recognise those proteins will destroy the precancer cells before the cancer occurs.

Vaccination 52

Vaccination Vaccine Trials Protein 52

XTalks

MAY 26, 2023

DEB patients are also at an increased risk of aggressive skin cancer.DEB usually presents at birth and is caused by one or more mutations in the COL7A1 gene, which encodes type VII collagen (COL7), an important protein that helps connect, strengthen and stabilize the outer and middle layers of the skin.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

Delveinsight

JANUARY 12, 2021

Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. Five of the mice had adenomas and one had a hepatocellular carcinoma (HCC).

Gene Therapy 94

Gene Therapy Gene Genetic Disease DNA 94

XTalks

AUGUST 6, 2020

Epidiolex is a plant-derived CBD solution developed by GW Pharmaceuticals. TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease usually presents within the first year of life, displaying subtle signs that can take years to develop.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

pharmaphorum

OCTOBER 5, 2022

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. The aim will be to identify promising candidates in areas of “unmet patient need.”

RNA 105

RNA Genetics Dermatology Antibody 105

Delveinsight

AUGUST 26, 2021

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. Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. 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.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). Without treatment, cTTP is ultimately fatal.

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Sigilon Therapeutics

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

MARCH 16, 2021

“Von Hippel-Lindau disease is a rare genetic condition for which there is no systemic treatment option available and is associated with a high risk of cancer development in multiple organs. Proteins known as hypoxia-inducible factors, including HIF-2?, About Von Hippel-Lindau Disease and Renal Cell Carcinoma.

Drugs 52

Drugs Trials Genetics Protein 52

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

Drug Discovery World

MAY 11, 2023

However, she also presented thoughts in a technical update, Targeted Protein Degradation – Progress, Direction and Prospects. At previous events, there has been an air of excitement but uncertainty of how quickly to adopt ‘business as usual’. This uncertainty seems to be a thing of the past, if BioTrinity is anything to go by.

Life Science 52

Life Science Genetics Vaccination Vaccine 52

The Pharma Data

OCTOBER 27, 2020

The company plans to tackle the existing challenges of precision medicine with a three-prong approach – developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs. Sirnaomics. Be Biopharma .

Gene 52

Gene Gene Therapy Medicine Genetics 52

XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Rett syndrome is characterized by a distinctive pattern of symptoms that gradually emerge after a period of seemingly normal development. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

The Pharma Data

NOVEMBER 8, 2020

The submission includes data from the ENLIGHTEN clinical development program in schizophrenia, and pharmacokinetic (PK) bridging data comparing ALKS 3831 and Zyprexa (olanzapine), to support an indication for schizophrenia. Reese, executive vice president of Research and Development at Amgen.

Trials 52

Trials Protein Gene Genetic Disease 52

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Proteins perform myriad functions within the human body and scientists have estimated that there are at least 20,000 canonical proteins that we know about.

RNA 52

RNA Protein Vaccination Vaccine 52

The Pharma Data

DECEMBER 20, 2020

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. Klisyri is a home-grown product discovered and characterized by Athenex scientists and developed from pre-IND to NDA by the Athenex team. and Europe, including Russia.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Licensing 52

XTalks

SEPTEMBER 17, 2020

While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. Dietary changes, including restriction of salt and animal protein, are also recommended.

Trials 98

Trials Gene Hormones Clinical Trials 98

The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Shortening of telomeres is associated with the symptoms of aging, heart disease, DNA damage and uncontrolled cell replication, which can lead to cancer.

DNA 52

DNA Scientist Genetic Disease Research 52

pharmaphorum

SEPTEMBER 9, 2020

She developed fine for the first six months of her life,” he says, “but then she stopped smiling, she stopped giggling, and stopped taking an interest in her surroundings. My daughter was born at 30 weeks and spent her first month in intensive care. She had a glazed look in her eyes, almost like she was blind.

Genome 103

Genome Genomics Genetics Genome Project 103

Drug Discovery World

FEBRUARY 28, 2023

Three hundred and fifty million people in the world, with 37 million in the United States and Canada, have been diagnosed with rare disease, 80% of which are inherited and genetic in origin 2. Therefore, the risk for off-target insertional mutagenesis is greatly reduced.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

Genetics 111

Genetics Vaccination Vaccine DNA 111

pharmaphorum

SEPTEMBER 9, 2020

She developed fine for the first six months of her life,” he says, “but then she stopped smiling, she stopped giggling, and stopped taking an interest in her surroundings. My daughter was born at 30 weeks and spent her first month in intensive care. She had a glazed look in her eyes, almost like she was blind.

Genome 80

Genome Genomics Genetics Genome Project 80

pharmaphorum

JANUARY 10, 2022

With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies. Outside of developing its own internal pipeline, the company has made significant investments into snapping up mRNA-focused biotechs.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

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.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Drug Discovery World

MARCH 6, 2024

The prevalence of these diseases has grown substantially in recent decades due to an increasing global population, longer life expectancies and increasing morbidity with predisposing risk factors. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

The Pharma Data

DECEMBER 3, 2020

The majority of patients were able to complete commonly used cognition assessments including either the BSID-III (Bayley Scales of Infant and Toddler Development) or the WPPSI-IV (Wechsler Preschool and Primary Scale of Intelligence Fourth Edition), indicating that these measures are valid and appropriate for use in patients with Dravet syndrome.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

Protein 52

Protein RNA Production Genetics 52

XTalks

DECEMBER 22, 2021

The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. They can be developed and adapted quickly. It is also prone to quick destruction by the body’s immune system before it can be translated into protein in cells.

Life Science 98

Life Science RNA Vaccination Vaccine 98

Drug Discovery World

OCTOBER 12, 2023

She says: “From turning genes off to look at the effect on cellular function, making single base changes within a coding or non-coding region to see if it impacts protein function, to testing drug candidates on genetic variants, we now have unprecedented genetic control to understand the link between genetics, disease and treatment.”

Drugs 59

Drugs Life Science Genome Genomics 59