PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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The Pharma Data

JUNE 11, 2025

Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.

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The Pharma Data

JUNE 12, 2025

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

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Drug Target Review

JUNE 30, 2025

“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. From theory to therapy This approach is already delivering promising insights across key therapeutic areas.

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Disease Medical Schools Science Therapies 59

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system.

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Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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DrugBank

JUNE 13, 2024

Similarly, for cardiovascular diseases, implantable drug-eluting stents can deliver controlled doses of anti-proliferative drugs directly to the site of arterial blockage, preventing restenosis (re-narrowing) and improving long-term integrity of the vessel. 

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Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Furthermore, the diagnosis of the autoimmune diseases could be a challenge and requires several kinds of evidence.

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Research Clinical Trials Therapies Immune Response 52

The Pharma Data

JUNE 12, 2025

The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.

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The Pharma Data

JUNE 9, 2025

This new monoclonal antibody therapy is designed to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in neonates and infants born during or entering their first RSV season. Octavio Ramilo, Chair of the Department of Infectious Diseases at St. Key results from the CLEVER trial include: A 60.5%

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The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.

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The Pharma Data

JULY 3, 2025

The results, recently published in the International Journal of Molecular Sciences , come from a collaborative effort between researchers at Erlangen University Hospital and Berlin-based biotech firm AudioCure. The potential of AC102 to treat both hearing loss and tinnitus sets it apart from other investigational therapies.

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Clinical Trials Hospitals Treatment Trials 52

Drug Target Review

MAY 23, 2025

In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. Existing therapies, such as tocilizumab, work by blocking specific inflammatory pathways but often come with limitations.

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Immune Response Therapies Treatment Clinical Trials 52

SCIENMAG: Medicine & Health

JULY 21, 2023

The preclinical study showed that injecting ovarian tissue-derived differentiated induced pluripotent stem cells can restore hormone production and fertility in mice with premature ovarian failure related to genetic diseases and cancer treatment A new study by investigators from Brigham and Women’s Hospital, a founding members of the Mass General Brigham (..)

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Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

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Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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Clinical Trials Disease Trials Therapies 52

Drug Target Review

MAY 18, 2023

In sickle cell disease, however, this function is impaired, leading to a range of potential health consequences. The irregularly shaped and sticky red blood cells produced by the disease can obstruct small blood vessels, causing recurring painful attacks. Kutlar says the possibilities of this additional therapy are exciting.

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Disease Clinical Trials Drugs Trials 80

Drug Target Review

APRIL 14, 2025

By preventing infections before they occur, these drugs reduce the need for hospital treatments, including the costly intensive care unit (ICU) care that can burden healthcare systems. Traditional biologics manufacturing can be complex and expensive, making it difficult to offer affordable therapies for widespread diseases.

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Broad Institute

OCTOBER 23, 2024

They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.

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DNA RNA Disease Hospitals 128

Broad Institute

MARCH 8, 2024

Type 2 diabetes is a disease of elevated blood sugar impacting approximately one in 10 people in the United States that can cause devastating health complications and is usually not cured. Adapted from an MGH press release. Paper cited: Smith K, Deutsch AJ, McGrail C., Multi-ancestry polygenic mechanisms of type 2 diabetes.

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Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Most drugs do not work in all people.

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Doctors Clinical Trials Treatment Therapies 111

Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Furthermore, the diagnosis of the autoimmune diseases could be a challenge and requires several kinds of evidence.

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Research Clinical Trials Therapies Immune Response 59

Broad Institute

AUGUST 19, 2024

Jude Children’s Research Hospital have discovered that oleic acid, one of the most abundant fatty acids in the body, restores a healthy balance of vaginal microbes in a laboratory model of BV. Jude Children’s Research Hospital, the Bill & Melinda Gates Foundation, and other sources. Paper cited Zhu M et al.

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Hospitals Treatment Medical Schools Science 121

biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies.

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Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes.

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Treatment RNA Hospitals Disease 96

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.

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Therapies Treatment Engineering Molecular Biology 59

Drug Target Review

JULY 15, 2025

The latest technologies are empowering researchers with faster, more efficient and more accessible genomic tools, leading to richer data generation and accelerating progress in areas like cancer genomics, rare disease research and infectious disease surveillance.​

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RNA DNA Laboratories Science 59

The Pharma Data

JUNE 18, 2025

AMVUTTRA’s approval has not only underscored the therapeutic potential of RNAi-based medicines but also signaled Alnylam’s readiness to scale its capabilities and deepen its impact across a wider range of disease areas. I believe we are just beginning to scratch the surface of what RNAi therapeutics can do to improve human health.”

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RNA Clinical Development Science Disease 40

Conversations in Drug Development Trends

JULY 3, 2025

Approximately two in 10 patients with hematologic malignancies are estimated to be eligible and able to receive cell therapy. The REMS Burden Explained Once deemed necessary for managing the risks associated with CAR T therapies, the REMS requirements imposed significant operational burdens on treatment centers.

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The Pharma Data

JUNE 11, 2025

argenx Unveils Positive Phase 2 Results for Efgartigimod in Myositis and Sjogren’s Disease at EULAR 2025 argenx SE a global immunology company dedicated to advancing treatments for severe autoimmune diseases, presented encouraging new clinical data at the 2025 European Congress of Rheumatology (EULAR), held June 11–14 in Barcelona, Spain.

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Broad Institute

OCTOBER 3, 2024

The findings point to factors involved in how neurons communicate and fire, suggesting potential targets for new therapies. Epilepsy also has several subtypes, and while one group called developmental encephalopathies have been connected to several genes, other forms of the disease are less well understood.

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The Pharma Data

OCTOBER 20, 2020

Kidney Disease, Injury Linked to Higher Mortality With COVID-19. The researchers found that 58 percent of patients presented with COVID-19 and renal impairment (45 percent developed acute kidney injury [AKI] during their ICU stay and 13 percent had preexisting chronic kidney disease [CKD]). Professional. TUESDAY, Oct.

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The Pharma Data

JUNE 27, 2025

SMA, a rare and often devastating genetic neuromuscular disease, primarily affects motor neurons in the spinal cord, resulting in progressive muscle wasting and weakness. Jude Children’s Research Hospital. At the conclusion of the study, all 25 participants were alive, and none had required permanent ventilation. Source link

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PPD

JUNE 7, 2023

Adults aren’t the only patients affected by the increasing prevalence of liver disease. The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations.

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Trials Clinical Trials Clinical Research Disease 98

The Pharma Data

JUNE 12, 2025

The positive results from APPULSE-PNH further reinforce Fabhalta’s ability to provide clinically meaningful improvements in Hemoglobin in PNH patients who previously may not have adequately responded to standard C5 inhibitor therapy — or who may wish to discontinue infusion therapy in favor of a convenient, oral option.” Source link

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The Pharma Data

DECEMBER 17, 2020

(NASDAQ: REGN ) today announced that the New England Journal of Medicine (NEJM) has published initial clinical data from an ongoing seamless Phase 1/2/3 trial of the antibody cocktail casirivimab and imdevimab in non-hospitalized patients with COVID-19. who require oxygen therapy due to COVID-19, OR. Yancopoulos , M.D.,

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Hospitals Production Clinical Trials Virus 52

Plenge Gen

APRIL 7, 2020

Pharmacologic intervention has the opportunity to impact disease progression in the SARS-CoV-2 / COVID-19 crisis. Repurposing of approved therapies is the fastest way to impact patients today, as these medicines have regulatory approval to enable investigator-initiated trials and have a manufacturing process to ensure drug supply.

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