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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KIF1A

MAY 4, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. By talking to groups with these mutations, we may identify other diseases whose patients have KIF1A variants. She once worked at SACELLS Biotechnology Co.

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Drug Discovery World

DECEMBER 18, 2023

Digital biomarkers promise to transform preventive medicine and clinical trials, but also raise issues of security, regulation and potential disparities in access. However, this robust system can become alarmingly vulnerable when confronted with debilitating conditions like Alzheimer’s, cancer, and cardiovascular diseases.

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Drug Target Review

JULY 5, 2024

Since brain organoids can be derived from either diseased or healthy patients, they can recapitulate human (patho)physiology making them a better choice compared to mammalian cell systems that often fail to recapitulate the human phenotype. Interneuron migration is regulated by L-type calcium channels (LTCCs), encoded by the CACNA1C gene.

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Drug Discovery World

MARCH 29, 2023

UMNs have been defined in the context of the draft as diseases where there’s a lack of good treatments, and which have a high burden 3. For rare disease drugs, a variable RDP period of five, nine, or ten years, depending on the novelty of the drug. appeared first on Drug Discovery World (DDW).

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Broad Institute

JUNE 14, 2023

A trailblazer in cancer research, Glimcher's research identified key transcriptional regulators of protective immunity and the origin of pathophysiologic immune responses underlying autoimmune, infectious, and malignant diseases. She is also a member of the board of trustees of the Massachusetts Health & Hospital Association.

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Drug Target Review

OCTOBER 31, 2023

Regulatory affairs workers get to tell a story about a drug to the regulators and – keeping the end in mind, which is the drug label or prescribing information – is a good way to communicate with regulators. I work on a rare disease called primary mitochondrial myopathy, for which there is currently no approved treatment.

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The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Oral and nasal administration routes are both physiologic approaches to stimulate the mucosal immune system to induce disease modifying benefits.”.

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The Pharma Data

MARCH 3, 2022

Patients taking the oral blood pressure medication not only required less daily insulin two years after first diagnosis of the disease, but also showed evidence of surprising immunomodulatory benefits. Type 1 diabetes is an autoimmune disease that causes loss of pancreatic beta cells, which produce endogenous insulin.

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The Pharma Data

MARCH 1, 2022

The mechanism is currently being evaluated for the potential treatment of cognitive impairment and other symptoms associated with a range of neuropsychiatric and neurodegenerative disorders, such as Alzheimer’s disease and major depressive disorder. senior vice president, R&D, chief scientific officer, AbbVie.

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The Pharma Data

JUNE 18, 2021

1 Clinical remission was defined by Crohn’s Disease Activity Index (CDAI) in the U.S. “These results represent another step towards the development of risankizumab for these patients, many of whom do not find sufficient disease control with current treatments.” NORTH CHICAGO, Ill. vice chairman and president, AbbVie.

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The Pharma Data

OCTOBER 26, 2020

On 14 May 2018, the Company announced a collaboration with GlobalCo to work on its CDX antibody. ” Market Abuse Regulation (MAR) Disclosure. For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. [link]. .

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Agency IQ

JULY 5, 2023

What we expect the EPA to do in July 2023 In this returning feature, AgencyIQ looks at public data to determine what the Environmental Protection Agency (EPA) is planning to do in the month ahead, including deadlines, meetings, events, planned regulations, comment periods, and more.

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Codon

JANUARY 21, 2024

Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. How are disease targets selected for challenge trials?

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Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

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Agency IQ

MARCH 7, 2024

As stated by the FDA , the proposed HFP will allow the agency to truly fulfill the vision laid out in the FSMA, “elevate the importance of nutrition to help reduce diet-related diseases,” and leverage existing partnerships and new technologies so as to “more effectively regulate and uphold safety of the nation’s food supply.”

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The Pharma Data

JANUARY 12, 2021

LONDON, UK / ACCESSWIRE / January 13, 2021 / Hemogenyx Pharmaceuticals plc (LSE:HEMO), the biopharmaceutical group developing new therapies and treatments for blood diseases, is pleased to announce that it has successfully completed the development of its CDX antibody with a leading global pharmaceutical company (“GlobalCo”).

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The Pharma Data

NOVEMBER 24, 2020

Currently, mCRPC remains an incurable disease, and despite advancements in available therapies, the 5 year survival rate for men with metastatic prostate cancer is only 28 per cent. Merck is one of the top R&D investors in Canada , with investments totaling $69 million in 2018 and more than $1 billion since 2000.

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The Pharma Data

MARCH 23, 2022

Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase. But targeting Ebola virus polymerase has proven tough.

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Drug Target Review

APRIL 3, 2024

Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Everything started in school with an experiment on isolating DNA from bananas.

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NIH Director's Blog: Drug Discovery

MAY 9, 2019

Among those taking on this ambitious challenge is a recipient of a 2018 NIH Director’s New Innovator Award, Srivatsan Raman of the University of Wisconsin-Madison. In fact, he hopes the data generated—and there will be a massive amount—will reveal novel sites to control or exploit allosteric signaling. ’ Fenton AW.

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The Pharma Data

DECEMBER 3, 2020

2 However, serious disease may occur in individuals with compromised immune systems, which includes patients who received immunosuppressants associated with various types of transplants including hematopoietic stem cell transplants (HSCT) or solid organ transplants (SOT). Cytomegalovirus infection in transplant recipients.

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The Pharma Data

SEPTEMBER 9, 2020

Basel, 10 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. Applications are under review with numerous regulators, including in the EU and China.

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Drug Discovery World

JULY 11, 2022

As the field matures and the risks are better understood, regulators continue to streamline duplicative and burdensome oversight efforts 5. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. " December 20, 2018[link]. Conclusion. The promise of gene therapy is substantial.

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Drug Target Review

AUGUST 24, 2023

These findings were mirrored in the clinical results from an analysis of two metastatic melanoma patient cohorts who received anti-PD-1 treatment: patients with responses and disease control had significantly lower GDF-15 serum levels (measured before treatment start), while none of the patients with elevated GDF-15 levels showed a lasting response.

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NIH Director's Blog: Drug Development

SEPTEMBER 20, 2018

That includes those involved in regulating our appetites and our moods, via a class of G proteins known as inhibitory G proteins (G i ). 2018 Jun;558(7711):553-558. [2] 2018 Apr;93(4):251-258. [3] They share a common architecture and bind a relatively limited number of intracellular signaling proteins called G proteins.

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The Pharma Data

JANUARY 19, 2021

About Biophytis Biophytis SA is a clinical-stage biotechnology company specialized in the development of therapeutics that slow the degenerative processes associated with aging and improve functional outcomes for patients suffering from age-related diseases, including severe respiratory failure in patients suffering from COVID-19.

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Agency IQ

JULY 7, 2023

regulators have previously discussed , using RWE “for regulatory decision-making is already a reality, and there is increasing interest in using big data and advanced analytics as a complementary source of evidence.” Regulators have been investing in ways to understand better how RWE can be used and how RWD is generated.

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Agency IQ

JUNE 7, 2024

BY CHELSEY MCINTYRE, PHARMD | JUN 3, 2024 8:43 PM CDT Regulatory background: DSHEA and dietary supplements The Dietary Supplement Health and Education Act ( DSHEA ) of 1994 defines the FDA’s authority in the regulation of dietary supplement products and dietary ingredients. See AgencyIQ’s deep dive into kratom research and regulation here. ]

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The Pharma Data

JANUARY 24, 2021

On December 17, 2018, Toripalimab obtained a conditional approval from the National Medical Products Administration (the “NMPA”) for the second-line treatment of unresectable or metastatic melanoma. and a disease control rate (DCR) of 86.2% The results of the study were published in the Journal of Clinical Oncology in August 2019.

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Agency IQ

NOVEMBER 3, 2023

Synthetic biotics have been engineered to perform a variety of functions: “ Several classes of effectors have been engineered into bacteria, including enzyme pathways that metabolize disease-causing toxins, production of protein effectors, and production of small-molecule effectors,” according to a recent article on the topic.

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The Pharma Data

NOVEMBER 9, 2020

On August 20, 2018, the Company signed the Exclusive Dealing Agreement with Prof. Neither the CSE nor its Market Regulator (as that term is defined in the policies of the CSE) accepts responsibility for the adequacy or accuracy of this news release. XPhyto Therapeutics Corp. ON BEHALF OF THE BOARD.

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The Pharma Data

JUNE 18, 2021

4,5 Between 20 and 46 percent of adults with atopic dermatitis have moderate to severe disease. 6 The range of symptoms pose significant physical, psychological and economic burden on individuals impacted by the disease. Nat Rev Dis Primers 4, 1(2018). About RINVOQ ® (upadacitinib). 14 In August 2019 , RINVOQ received U.S.

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The Pharma Data

JANUARY 13, 2021

Structure 3:1031-1039 (1995) 3 Waters J, Biocentury ; October 26, (2018) 4 NCT02273960; ClinicalTrials.gov ; “Study to Evaluate Safety and Efficacy in Adult Subjects With ITP (ITP)”; results posted April 1, 2019, updated July 29, 2019 and accessed Jan 11, 2021 5 Ferrant JL et al., 1 Lederman, S. International Immunol. (11):1583

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The Pharma Data

JANUARY 24, 2021

Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Food and Drug Administration (“FDA”) approval.

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The Pharma Data

OCTOBER 15, 2020

Please be aware that Photocure does not take any responsibility for accessing such information which may not comply with any legal process, regulation, registration or usage in the country of your origin. About Bladder Cancer. 75% of all bladder cancer cases occur in men. 75% of all bladder cancer cases occur in men.

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Agency IQ

JANUARY 12, 2024

BY AMANDA CONTI | JAN 10, 2024 9:29 PM CST Quick background: Goal dates under the Prescription Drug User Fee Act (PDUFA) The FDA collects user fees as part of an essential bargain between regulators and industry. In fact, CDER received a perfect 100% for meeting PDUFA dates for novel drugs in 2013, 2017, 2018 and 2020.

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