Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

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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.

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Drug Discovery World

DECEMBER 18, 2023

Casgevy The committee recommended granting a conditional marketing authorisation for Casgevy (exagamglogene autotemcel), an advanced therapy medicinal product (ATMP) for the treatment of transfusion dependent beta-thalassemia and severe sickle cell disease.

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Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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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.

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Drug Discovery World

AUGUST 8, 2024

DDW Editor Reece Armstrong speaks to Roger Levy , Global Medical Expert Immunology & Specialty Medicine at GSK about the company’s work into treating systemic sclerosis-associated interstitial lung disease and its recent BLISSc-ILD study evaluating its belimumab treatment. RA: Can you give us an overview of the BLISSc-ILD study?

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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.

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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.,

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Drug Discovery World

SEPTEMBER 1, 2023

metaLead, which is developing therapies to treat metal-related diseases, currently focused on Wilson disease, lead poisoning, and neurodegeneration with brain iron accumulation. Amalus Therapeutics, which is reshaping the lives of people affected by cancer and fibrotic diseases.

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Drug Discovery World

JULY 14, 2022

Pharmaceutical company SIGA Technologies has announced that its oral therapy tecovirimat has been approved in the UK for the treatment of smallpox, monkeypox, cowpox. . The therapy has also been approved for the treatment of complications following vaccination against smallpox in adults and children with a body weight of at least 13kg. .

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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.

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Drug Discovery World

SEPTEMBER 15, 2022

A new combination therapy could represent a significant advancement in the treatment of metastatic colorectal cancer. CRC is the second most common cause of cancer mortality, accounting for 881,000 deaths globally in 2018, and patients with metastatic disease have a five-year survival rate of just 11%.

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Drug Discovery World

MARCH 6, 2023

Since its inception in 2018, BII has supported 80 start-ups and projects with €65 million alongside the venture capital, industry and business expertise it provides to help them accelerate to the next level.

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Drug Discovery World

APRIL 11, 2023

2018; 10 (1): 34‐45 Duchesne M, et al. Elevated Expression of IL-33 and TSLP in the Airways of Human Asthmatics In Vivo: A Potential Biomarker of Severe Refractory Disease. 2018; 200: 2253–2262. There is also a significant healthcare burden, with these patients accounting for approximately 50% of NHS asthma-related costs.

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Drug Discovery World

MAY 22, 2023

Cell therapy, RNA technology, antibody drug conjugates and AI/machine learning are among the key areas of investment for pharma, according to a new report. While the reported bio partnering deal values for 2022 were the second highest recorded, the number of transactions was the lowest since 2018. Biotech companies raised $16.9

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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.

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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.

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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.

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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.

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Drug Discovery World

SEPTEMBER 26, 2022

The approval by the European Commission was based on results from the Pathfinder clinical trial programme, which included the pivotal Navigator Phase III trial in which Tezspire demonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.

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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. .

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Drug Discovery World

JUNE 1, 2023

According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1. A market research report published in late June 2018 by HTF market Intelligence Consulting suggested that the ADC market in China would grow exponentially by 2023 4.

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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.

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Common Sense for Drug Policy Blog

JANUARY 5, 2024

of the US adult population, injected drugs in 2018. Much of this increase is likely attributable to increases in IDU, but it is important to consider methodological differences in the creation of this 2018 estimate vs the 2011 estimate. million PWID in 2011, which suggest the 2018 estimate is closer to 3 times higher than in 2011.

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43 If the body fails to make the right protein, diseases such as cancer, type II diabetes, neurological conditions, and metabolic diseases can occur.

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New Drug Approvals

SEPTEMBER 6, 2024

2 History Efficacy was evaluated in MARIPOSA (NCT04487080), a randomized, active-controlled, multicenter trial of 1074 participants with exon 19 deletion or exon 21 L858R substitution mutation-positive locally advanced or metastatic non-small cell lung cancer and no prior systemic therapy for advanced disease. [2] hdl : 10665/330941.

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Drug Discovery World

JULY 22, 2022

Although immunotherapy has revolutionised cancer therapy, the effectiveness of currently available immunotherapies varies significantly across patients and treatment types. To mitigate these risks, clinicians are turning to other immunomodulators, such as cell-based therapies, bispecific antibodies, and cytokine treatments.

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Drug Discovery World

APRIL 25, 2023

Alba Health which is on a mission to reduce chronic disease risks from childhood, leveraging data science and the latest microbiome science. Amplify Therapeutics boasts disease-modifying therapies for Gaucher disease and genetic subgroups of Parkinson’s disease. billion by 2030.

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Drug Discovery World

AUGUST 10, 2022

But decades of research led to improvements in cancer therapy, centred around the idea that one’s immune system could aid in treatment. Next, the advent of antibody treatments in 1997 created the first targeted cancer therapy 2. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic.

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New Drug Approvals

SEPTEMBER 10, 2024

2] [3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation. [2] April 2018). mol), (R)-1,1,1-trifluoropropan-2-amine hydrochloride (0.39 6 August 2024.

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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.

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New Drug Approvals

SEPTEMBER 8, 2024

2222514-07-8 ] Palopegteriparatide , sold under the brand name Yorvipath , is a hormone replacement therapy used for the treatment of hypoparathyroidism. [1] 5] Study drug and conventional therapy were subsequently adjusted according to the albumin-corrected serum calcium levels. [5] 48,000) consisting of 34 amino acid residues.

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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.

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The Pharma Data

JANUARY 6, 2021

“IPF is a devastating condition with an estimated mean survival of 2-5 years from time of diagnosis, and currently there are no available therapies that stop the progression of fibrosis or treat the underlying causes of the disease,” said John Hood, Ph.D., co-founder, CEO and chairman of Endeavor. Miguel de los Rios, Ph.D.,

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Drug Discovery World

OCTOBER 6, 2022

This growth is due to factors including a rise in speciality medicines, an increase in lifestyle-driven diseases and a growing population 1. France, Switzerland, and the United Kingdom have seen the fastest growth, accounting for 63% of the biotechs founded between 2018 and 2020.” Valued at $14.8 until 2030. Hotspots . References: .

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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.

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