Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111

Disease Clinical Trials Therapies Science 111

Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year.

Vaccine 102

Vaccine Trials Therapies Treatment 102

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

FDA Approval 52

FDA Approval Therapies FDA Disease 52

Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

SugarCone Biotech

MARCH 24, 2025

Consequently, these drugs have limited ability to keep patients disease-free and limited impact on patient survival. When we look at how TCEs interact with targeted cancer cells we can quickly see how cancers respond to T Cell-based therapies. Other mechanisms that cancer cells use to escape from TCE treatment are less expected.

Immune Response 67

Immune Response Therapies Treatment Disease 67

Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. 2 It is this complexity that necessitates powerful, targeted combination therapies. 2 It is this complexity that necessitates powerful, targeted combination therapies.

Small Molecule 111

Small Molecule Therapies Treatment Disease 111

Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

Disease 59

Disease Clinical Trials Therapies Science 59

Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

Drugs 111

Drugs RNA DNA Disease 111

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

Disease 59

Disease Production FDA Drug Development 59

Drug Target Review

NOVEMBER 7, 2023

To target proteins involved in causing or spreading disease, typically a drug will obstruct a protein’s active site so it cannot function. Proteins can become part of a cancerous tumour and allow for its unrestricted growth, cause diseases like Alzheimer’s , and build up in the heart to impact how it pumps blood around the body.

Therapies 59

Therapies Science Disease Treatment 59

The Pharma Data

NOVEMBER 9, 2020

Talaris Chief Executive Officer Scott Requadt sees FCR001, the company’s investigational cell therapy, as a potential pipeline in a product (an experimental treatment that could have multiple uses across a number of indications). We can treat organ transplant and autoimmune diseases in the same manner.”.

Therapies 52

Therapies Production Disease Treatment 52

The Pharma Data

DECEMBER 22, 2020

Tagrisso Approved as Adjuvant Therapy for NSCLC With EGFR Mutations. Tagrisso was approved in 2018 for first-line treatment of patients with metastatic non-small cell lung cancer with EGFR exon 19 deletions or exon 21 L858R mutations. Professional. TUESDAY, Dec. Food and Drug Administration announced Friday.

Therapies 52

Therapies Treatment FDA Disease 52

Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Uplifting Athletes As a 501(c)(3) nonprofit, UA harnesses the power of sport and partnerships with patient advocacy groups to fund the researchers developing datasets for rare diseases that have never been studied or understood before.

Clinical Trials 52

Clinical Trials Disease Trials Research 52

The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. It also increases your risk for inflamed cornea, cornea eye disease, corneal ulcers and injuries, and eye infections. .

Treatment 52

Treatment Disease FDA FDA Approval 52

Drug Target Review

JULY 7, 2023

Many disease states are caused by a web of complex interactions rather than single-gene mutations. This gives epigenetic editing the unique ability to treat complex multigenic diseases and opens the door to enhancing cell and regenerative therapies, which require the manipulation of multiple genes for optimal results.

DNA 105

DNA Engineering Therapies Regulations 105

The Pharma Data

MAY 5, 2022

Validated Measurable Residual Disease Assays are Critical for the Rapid Development of Future Novel Therapeutics for Patients with Acute Myeloid Leukemia. 1 Clinical trials for new therapies in AML are becoming increasingly lengthy to complete based on the traditional clinical endpoint of survival. About Acute Myeloid Leukemia.

Disease 52

Disease Clinical Trials Drug Development Pharmaceutical Companies 52

Broad Institute

JANUARY 22, 2024

Since 2018, the Cancer Dependency Map (DepMap) Consortium , an academic-industrial partnership launched by the Broad, has uncovered several potential drug targets by systematically screening cancer models in search of genetic dependencies. By Allessandra DiCorato January 22, 2024 Bill Sellers is the head of Broad's Cancer Program.

Drugs 128

Drugs Therapies Regulations RNA 128

Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. 2018 Mar;17(3):167–81.

Drugs 111

Drugs FDA Disease Animal Testing 111

Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. However, if the therapy targets only the subclonal (branch) mutations, this will result in the mere ‘pruning’ of specific branches rather than elimination of the whole cancer.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., SCHEME PATENT PTC Therapeutics Inc.,

Small Molecule 57

Small Molecule RNA DNA Disease 57

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

Production 141

Production Clinical Trials Trials Therapies 141

Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution. 1 , 33–43 (2018).

Small Molecule 126

Small Molecule Drugs Immune Response Targeted Protein Degradation 126

The Pharma Data

JULY 15, 2021

The access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). The browser can be accessed via [link]. About AbbVie.

Disease 52

Disease Biosimilars Government Treatment 52

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. When the company launched an IPO in late 2018, its valuation was $7 billion, which at the time was a record biotech IPO.

Marketing 52

Marketing Engineering Therapies Disease 52

DrugBank

OCTOBER 4, 2024

For example, Takeda Pharmaceutical's $62 billion acquisition of Shire plc in 2018 expanded its global footprint and tapped into Shire's strong presence in emerging markets. The Evolving Regulatory System Regulatory frameworks play a pivotal role in shaping the pharmaceutical M&A landscape.

Pharmaceuticals 98

Pharmaceuticals Pharmaceutical Companies Therapies Marketing 98

NIH Director's Blog: Drug Development

NOVEMBER 15, 2018

Wimley is working hard to change that, since peptides have shown promise as therapies against some microbes, dubbed “superbugs,” that have developed resistance to multiple drugs and present a serious health threat, especially to people with weakened immune systems. 2018 Jul 2;9(1):2568. [2] Kauffman WB, Guha S, Wimley WC. Nat Commun.

Science 52

Science Drugs Disease Therapies 52

Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

Disease 52

Disease Clinical Trials Trials Treatment 52

The Pharma Data

MARCH 16, 2021

Viela (Nasdaq: VIE), headquartered in Gaithersburg, Maryland, is a biotechnology company dedicated to the discovery, development and commercialisation of novel treatments for autoimmune and severe inflammatory diseases. AstraZeneca.

Treatment 52

Treatment Science Disease Therapies 52

The Pharma Data

JULY 8, 2021

Today, access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). The browser can be accessed via [link]. About AbbVie. About Biogen.

Disease 52

Disease Clinical Trials Government Research 52

The Pharma Data

SEPTEMBER 8, 2020

Takeda has revealed it has agreed to divest its non-core portfolio of prescription therapies to Cheplapharm in exchange for an upfront payment of $562 million. The moves represent Takeda’s attempts to balance the books following its mammoth acquisition of Shire in 2018 for $62 billion. Matt Fellows. Source link.

Therapies 52

Therapies Production Disease Drugs 52

Drug Target Review

MAY 23, 2023

Inflammatory bowel disease (IBD) is an umbrella term that describes complex disorders that cause chronic inflammation in the digestive tract with alternating periods of relapse and remission. Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. More than 6.8

Drugs 75

Drugs Small Molecule Production Disease 75

Broad Institute

MAY 2, 2024

In 2018, she graduated with a bachelor’s degree in computer science and biology and joined the lab of Gad Getz at the Broad Institute of MIT and Harvard to complete her master’s. If we can understand how those processes work, we can better develop drugs to target these multifaceted diseases. What do you like about rowing?

Science 124

Science Medical Schools Disease Hospitals 124

Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo.

Treatment 98

Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

JANUARY 14, 2021

For the full year of 2019, Xalkoria brought in $530 million, an increase of 1% from 2018. “We We are proud to deliver the first biomarker-driven therapy for children and young adults with ALCL,” said Chris Boshoff, chief development officer, Oncology, Pfizer Global Product Development. Most Read Today. Source link.

FDA Approval 52

FDA Approval FDA Therapies Treatment 52

The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. About Targeted Alpha Therapies (TAT) at Bayer Targeted Alpha Therapies (TAT) are an emerging class of radionuclide therapy for various difficult to treat tumors.

Small Molecule 52

Small Molecule Licensing Licensing Therapies 52

Agency IQ

MAY 17, 2024

The Creating Hope Reauthorization Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program. The markup hearing comes three months after the E&C Committee last held a hearing on 18 bills related to rare diseases, including all four of the above-mentioned bills. DORIS MATSUI (D-Calif.)

FDA 40

FDA Disease FDA Approval Production 40