The Pharma Data

AUGUST 5, 2020

. Novartis has revealed positive results from a phase 2 study of its CAR-T therapy Kymriah (tisagenlecleucel) in patients with relapsed or refractory follicular lymphoma. The therapy is a one-time treatment created individually for each patient using their own T cells.

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The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc.

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Drug Target Review

SEPTEMBER 22, 2023

Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. 5 Organoids are recognised as New Alternative Methods (NAMs) in drug development.

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Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Creative Biolabs

JUNE 29, 2023

At present, a large number of G protein-coupled receptor-targeted drugs have been marketed, and a large number of drugs are under development. GPCR is the most valuable target for drug development, and more drugs will be used to treat various diseases in the future.

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Drug Target Review

SEPTEMBER 26, 2023

Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. CNS-related issues account for nearly a quarter of failures during clinical development, a phase where consequences are high in terms of resources and patient impact. 2017 Jan;155(1):234–47.

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Advarra

AUGUST 16, 2022

In 2016, the FDA granted 333 (57%) of the 582 orphan drug designation requests it received – nearly 10 times the number of designations the agency awarded in 1986. The agency’s 2019 guidance, Rare Diseases: Common Issues in Drug Development , called attention to the need for natural history studies for rare diseases.

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Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

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Drug Target Review

OCTOBER 24, 2023

We also believe that drug development teams should routinely consider the potential modifying effect of the gut microbiome on drug potency and factor it into assessments of PK and PD. MB097, our drug with immune-oncology applications, is a consortium of nine bacterial strains. difficile and C. albicans.

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Vial

DECEMBER 5, 2023

It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development. Sponsors have used NGS to screen patients for clinical trial eligibility and patient stratification , expanded CDx development, and comprehensive genomic profiling (CGP).

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New Drug Approvals

SEPTEMBER 13, 2024

4] [6] Aticaprant was originally developed by Eli Lilly , was under development by Cerecor for a time, and is now under development by Janssen Pharmaceuticals. [2] 12] Positron emission tomography imaging revealed that brain KORs were almost completely saturated by the drug 2.5 Dhir A (January 2017). August 2017.

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The Pharma Data

JUNE 25, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (A?) Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions.

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Drug Target Review

JULY 10, 2023

This deeper understanding has enabled the development of numerous potentially breakthrough therapies, which are currently being tested in the clinic. As scientists continue to elucidate and advance novel drug targets and technology applications, the goal of bringing impactful treatments to patients becomes more of a reality.

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Drug Target Review

OCTOBER 25, 2024

Biomarkers are an essential advancement that will help determine risk for developing Alzheimer’s disease, diagnose the disease before symptoms have progressed significantly, predict how quickly patients may experience cognitive decline and evaluate how well new therapies are working. 2017, February 4). link] Cummings J.

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DrugBank

JUNE 27, 2023

The general notion is that patients should be viewed individually, rather than strictly as members of some larger general population, and that their specific genetic background, environment, and lifestyle choices should be considered throughout drug development to the point of treatment and continuing patient care. Drug Discov.

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The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Silverback Therapeutics

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NIH Director's Blog: Drug Development

JULY 20, 2017

Helping to fuel this optimism are preliminary results released this week from early-stage clinical trials of three next-generation, triple combination therapies that modulate the function of CFTR. Developing drugs that could rescue the function of mutant CFTR has been a long and difficult journey. 1989 Sep 8;245(4922):1059-1065.

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NIH Director's Blog: Drug Development

DECEMBER 4, 2018

This blood vessel-tissue interface can prevent certain molecules—including some potentially beneficial therapies—from entering the brain. It included a chip that models the blood-brain barrier. I’ll be anticipating some some really cool science results in the coming months!

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The Pharma Data

DECEMBER 9, 2020

333-222268) previously filed with the Securities and Exchange Commission (the “SEC”) on December 22, 2017 and declared effective by the SEC on January 5, 2018. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. SAN DIEGO, Dec.

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Alta Sciences

JUNE 27, 2023

They remind us of the immense benefits of drug development. For Michelle Newby, Study Coordinator II for preclinical services at Altasciences, research strikes a personal chord: In 2017, at the age of 25, Michelle was diagnosed with cancer. A: In December of 2017, I was diagnosed with two different types of stage III leukemia.

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The Pharma Data

JANUARY 31, 2021

Trimer-Tag© is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. Dynavax is a commercial stage biopharmaceutical company developing and commercializing novel vaccines. CEPI has initiated 11 partnerships to develop vaccines against the novel coronavirus.

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A Dose of Drug Development

SEPTEMBER 17, 2018

How Integrium as a company plays a role in advanced technology such as these is that Integrium has the advantage of having 20 years of experience with variety of therapies within its portfolio. Though 2017 may have had more IPOs in 9 months of the year, but the capital raise is considerably different.

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Agency IQ

AUGUST 16, 2024

By Amanda Conti | Aug 13, 2024 10:00 PM CDT Regulatory context: Psychedelic regulation and drug development A growing body of evidence suggests that psychedelics may provide clinical benefit for certain purposes, especially mental health conditions. Importantly, midomafetamine was proposed for use in conjunction with psychotherapy.

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The Pharma Data

DECEMBER 3, 2020

Trimer-Tag © is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. Additionally, Clover is leveraging its in-house GMP biomanufacturing capabilities to support large-scale production of its biologic therapies. S-Trimer is intended to be adjuvanted. About CEPI.

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The Pharma Data

JULY 29, 2021

today announced results of a longitudinal preliminary assessment of the clinical effects of lecanemab (development code: BAN2401) — granted Breakthrough Therapy designation by the U.S. and slowed clinical decline in an 18-month Phase 2b proof of concept study (Study 201, n=856) in early AD ( Alz Res Therapy 13; 2021 ).

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The Pharma Data

JANUARY 31, 2021

Trimer-Tag© is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. CEPI is an innovative partnership between public, private, philanthropic, and civil organisations, launched at Davos in 2017, to develop vaccines to stop future epidemics. About CpG 1018.

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Agency IQ

AUGUST 2, 2024

September 30, 2024 PDUFA Novel Approaches to Development of Cell and Gene Therapy Convene a public meeting to solicit input on methods and approaches (e.g., use of RWE, registries) for capturing post-approval safety and efficacy data for cell and gene therapy products.

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Vial

DECEMBER 11, 2023

According to a study by the Tufts Center for the Study of Drug Development, the average time from site identification to study start-up completion is approximately 31.4 Starting up a new site can be a challenging and time-consuming process, often filled with unforeseen hurdles and delays. weeks for Phase II and Phase III studies.

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The Pharma Data

NOVEMBER 15, 2020

This prestigious group of professionals will help lead the Company as it prepares for potential commercialization of its investigational cell therapy, MultiStem ®. Most recently, she was a senior executive at Celgene Corporation, where she led corporate strategy from 2012 to 2017. drug development initiative from 2012 to February 2019.

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The Pharma Data

DECEMBER 15, 2020

A total of 43 patients initially diagnosed with unresectable stage IV metastatic PDAC, who had progressed following first-line gemcitabine-based therapy, were enrolled in the triple combination arm. Disease control rate (DCR). 63.2%. 29-52% 2,4.

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Agency IQ

FEBRUARY 23, 2024

Workshop addresses oncology dose optimization across full span of development In a series of broad-ranging, frank discussions, attendees at a joint FDA-American Association of Cancer Research (AACR) workshop worked through opportunities and challenges for dose optimization across the span of cancer drug development activities.

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The Pharma Data

OCTOBER 28, 2020

There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. About Ultragenyx.

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Agency IQ

JUNE 9, 2023

According to a study of healthcare expenditure on orphan drugs versus non-orphan drugs, the market share of expenditure on orphan drugs experienced an average annual growth of 16% in the eight E.U. countries examined between 2010 and 2017. million euros in Italy and 2.325 million euros in Germany.

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Drug Target Review

JUNE 21, 2023

Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies. Genetic-based therapeutics hold significant promise in the clinic Despite the growing number of genes linked to human disease, the proportion of genes targeted by approved therapies remains low.

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Broad Institute

OCTOBER 11, 2023

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.

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Drug Target Review

MARCH 15, 2024

Transposon technologies are gaining traction due to their accuracy in integrating the gene of interest into the host genome, making them an important tool for accelerating large molecule drug development. 2017 Nov;33(6):1468-1475. Epub 2017 Sep 7. Cell Gene Therapy Insights 2017; 3(2), 131-158, 10.18609/cgti.2017.014.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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