Drug Discovery World

MARCH 14, 2023

Teitur Trophics, a biotech company developing new treatments for patients suffering from neurodegenerative diseases, has complete a €28M Series A financing. The financing was co-led by Sunstone Life Science Ventures and Sound Bioventures, with participation from new investors, Industrifonden, Innovestor’s Life Science Fund and P53 Invest.

Disease 130

Disease Treatment Science Clinical Development 130

Drug Discovery World

NOVEMBER 24, 2022

DDW’s Diana Spencer asks what will be needed for Europe to compete in the growing global market for advanced therapies. . In terms of ATMPs, other countries are ramping up support for these innovative therapies. share of global clinical trials activity in 2020, a decrease of 6.3%, compared with a 25.6%

Therapies 245

Therapies Clinical Trials Pharmaceuticals Government 245

Drug Discovery World

SEPTEMBER 4, 2024

The drug was approved by the FDA for Expanded Access, allowing trial participants to continue CS1 treatment when no comparable or satisfactory alternative therapy options are available. The EU OMPD complements the orphan drug designation (ODD) granted by the US FDA in 2020, enhancing the protection and value of CS1 in these major markets.

Drugs 264

Drugs FDA Marketing Treatment 264

Drug Discovery World

OCTOBER 31, 2023

Professor Andy Whiting and Dr Tony Lockett discuss how specific levels of a protein could be used to advance drug development in neurodegenerative diseases. These parameters aim to measure the progression of disease over time by looking at the functional or observable change in a patient.

Drug Development 147

Drug Development Disease Drugs Clinical Trials 147

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

Therapies 130

Therapies Clinical Trials Vaccine Disease 130

Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? Curbishley explains that as the wave of new therapies in development continues to increase, global pressures on manufacturing availability will become more acute.

Therapies 130

Therapies Clinical Trials Marketing Production 130

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.

Therapies 130

Therapies Clinical Trials Production Trials 130

Eye on FDA

MARCH 7, 2024

Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC).

FDA 78

FDA Disease Government Therapies 78

Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

Disease 59

Disease Clinical Trials Clinical Pharmacology Trials 59

Drug Discovery World

MAY 3, 2024

TGCT is a type of tumour that affects the soft tissue lining of joints and tendons and is a highly debilitating disease often impacting large, important joints such as the knee, hip and ankle. It is a chronic disease, which often impacts patients throughout their lives.

Disease 130

Disease Therapies Trials Treatment 130

Drug Target Review

OCTOBER 5, 2023

The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020. Around three years following their aHSCT, 20 patients (11 percent) were administered disease-modifying drugs.

Therapies 116

Therapies Treatment Clinical Trials Disease 116

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America. SATURDAY, Dec.

Therapies 52

Therapies Disease Medical Schools Virus 52

The Pharma Data

JUNE 28, 2021

Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.

Therapies 52

Therapies Disease Clinical Trials Trials 52

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 Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

Production 52

Production Therapies DNA Virus 52

The Pharma Data

DECEMBER 8, 2020

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced. To date, a total of 19 trial participants have been dosed with the gene therapy.

Therapies 52

Therapies Clinical Trials Trials Treatment 52

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

The Pharma Data

OCTOBER 21, 2020

22, 2020 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the company will host a conference call and live webcast on Thursday, November 5, 2020 at 8:00 a.m. CAMBRIDGE, Mass.,

International 52

International Disease Pharmaceuticals Therapies 52

The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. ADUHELM is indicated for the treatment of Alzheimer’s disease. It may also limit coverage for any future approved treatment in the class.

Treatment 52

Treatment Therapies Disease FDA Approval 52

The Pharma Data

JUNE 28, 2021

Food and Drug Administration (FDA) granted Breakthrough Therapy designation for donanemab, Eli Lilly and Company’s (NYSE: LLY) investigational antibody therapy for Alzheimer’s disease (AD). Dementia due to Alzheimer’s disease is the most common form of dementia, accounting for 60 to 80 percent of all cases 1.

Therapies 40

Therapies Disease Treatment FDA Approval 40

Drug Discovery World

MAY 29, 2023

When it comes to published scientific research, the UK is ranked third in the world with nearly 200,000 citable publications in 2020. The new location is part of Edinburgh Technopole, a Pioneer Group in Midlothian Science Zone.

Therapies 130

Therapies Science Trials Production 130

The Pharma Data

NOVEMBER 18, 2020

Per the deal, Obsidian Therapeutics will provide its cytoDRiVE delivery platform to the university to speed research into TILs, which target tumors by reversing the expression of certain cancer-causing proteins via an oral therapy. Source link.

Therapies 52

Therapies Treatment Disease Research 52

The Pharma Data

NOVEMBER 8, 2020

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs. LONDON and RALEIGH, N.C., For more information, please visit www.veronapharma.com. Forward-Looking Statements.

Therapies 52

Therapies Treatment Disease Hospitals 52

The Pharma Data

NOVEMBER 18, 2020

Antimicrobial resistance occurs when microorganisms resist the effects of medications, making infections harder to treat and increasing the risk of disease spread, severe illness and death. Key definitions. Combatting antimicrobial resistance is a global. efforts to address antimicrobial resistance. Department information note. infants. .

Laboratories 52

Laboratories Virus Government Therapies 52

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Therapies 52

Therapies FDA Clinical Trials Production 52

The Pharma Data

DECEMBER 30, 2020

The year 2020 will certainly go down in history as the year of COVID-19. Gilead Sciences Makes Multiple Bets : When Gilead Sciences wasn’t working on remdesivir in COVID-19, the company seemed to be constantly flexing its M&A muscle over the course of 2020. It began in May with the $4.9

Science 52

Science Pharmaceuticals Disease FDA Approval 52

The Pharma Data

MARCH 24, 2021

billion EUR in 2020 (18.9% Boehringer Ingelheim stepped up its investments in R&D significantly in 2020 in pursuit of innovative medicines and therapies for diseases for which no satisfactory treatments are available. In particular, efforts to research potential COVID-19 related therapies were accelerated.

Vaccine 52

Vaccine Small Molecule Therapies Disease 52

The Pharma Data

OCTOBER 20, 2020

Kidney Disease, Injury Linked to Higher Mortality With COVID-19. 20, 2020 — Renal impairment in patients admitted to intensive care with COVID-19 is common and is associated with high mortality, according to a study published online Oct. © 2020 HealthDay. Posted: October 2020. Professional. TUESDAY, Oct.

Disease 52

Disease Hospitals Therapies Research 52

Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

Disease 52

Disease Clinical Trials FDA Approval FDA 52

The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

Clinical Development 52

Clinical Development Disease Therapies Treatment 52

The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

Disease 52

Disease Small Molecule Pharmacokinetics Licensing 52

The Pharma Data

DECEMBER 11, 2020

11, 2020 — Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. The researchers said the technique could break the cycle of disease passed from mother to baby through mutations in mitochondrial DNA (mtDNA). FRIDAY, Dec.

Therapies 52

Therapies Clinical Trials International Disease 52

Drug Discovery World

NOVEMBER 20, 2023

Biologics represents the fastest-growing sector of the pharmaceutical industry, with hundreds of therapies approved in recent years 3,4. mRNA vaccines and antibody therapies in particular are promising areas that are already having significant impact across the healthcare industry, with enormous potential to address unmet medical needs.

DNA 173

DNA Engineering Vaccine Therapies 173

The Pharma Data

OCTOBER 26, 2020

27, 2020 11:00 UTC. DALLAS–( BUSINESS WIRE )– Taysha Gene Therapies Inc. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S.

Therapies 52

Therapies Disease FDA Drugs 52

The Pharma Data

NOVEMBER 20, 2020

20, 2020 — Hormone therapy can be a lifesaver for men with prostate cancer , but it also appears to put some at increased risk of heart problems, a new study reports. At the start of the study, 4 out of 5 men had two or more risk factors for heart disease , the study says. FRIDAY, Nov.

Therapies 52

Therapies Doctors Disease Hospitals 52

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7

Drugs 130

Drugs Therapies Clinical Trials Cell Based Assays 130

The Pharma Data

SEPTEMBER 16, 2020

16, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) will host two webcast calls for the investment community in conjunction with the European Society for Medical Oncology (ESMO) 2020 Virtual Congress. 20, 2020, at 11:00 a.m. THOUSAND OAKS, Calif. On Sunday, Sept. PDT, David M. Reese , M.D., Reese , M.D.,

Clinical Development 52

Clinical Development Therapies Disease Research 52