Fierce BioTech

JULY 25, 2023

How combining datasets from multiple sources can inform and improve commercial strategies Hundreds of rare disease treatments have entered the market over the past decade—thanks largely to a combination of government incentives, strong urging from patient advocacy groups and advances in cell and gene therapies.

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DrugBank

AUGUST 20, 2024

The world of drug discovery is in a state of transformation, with advancements in biotechnology opening doors to new possibilities for targeting previously "undruggable" disease mechanisms. We also explore why some disease-associated targets remain elusive and how recent innovations are changing that narrative.

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Plenge Gen

APRIL 7, 2020

You can download a pdf copy of the blog here.) Pharmacologic intervention has the opportunity to impact disease progression in the SARS-CoV-2 / COVID-19 crisis. … The post Drug repurposing in SARS-CoV-2 / COVID-19: preventing the maladaptive immune response leading to critical disease requiring ICU care.

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The Pharma Data

NOVEMBER 1, 2020

Corlieve’s lead program employs a novel AAV gene therapy approach for the treatment of refractory temporal lobe epilepsy (TLE), the most commonly diagnosed focal epilepsy in humans. Maryland , US), a leading gene therapy company. Maryland , US), a leading gene therapy company. PARIS , Nov.

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The Pharma Data

OCTOBER 27, 2020

Stargardt disease is a devastating genetic disorder that affects central vision with no approved treatment options. . “This designation permits reVision to further accelerate the development of our Stargardt disease treatment. . RIDGEWOOD, N.J. , 28, 2020 /PRNewswire/ — ReVision Therapeutics, Inc.,

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The Pharma Data

JANUARY 27, 2021

HK), announced today that China Center for Drug Evaluation (CDE) has granted Breakthrough Therapy designation to Batoclimab (HBM 9161), a fully human anti-FcRn monoclonal antibody (mAb), for the treatment of adult patients with Myasthenia Gravis (MG). CAMBRIDGE, Mass. ROTTERDAM, Netherlands and SUZHOU, China , Jan. ” he added.

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Drug Channels

FEBRUARY 6, 2024

Access USA will unite PAP – Patient Assistance & Access Programs, Hub and Specialty Pharmacy Models East and the Rare Disease Summit three influential access conferences under one roof for one week of collaborative discussions and opportunities to expand your network and establish powerful partnerships. What is Access USA?

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The Pharma Data

OCTOBER 14, 2020

“We look forward to continuing to advance apraglutide and to preparing our organization to bring a potential best-in-class therapy to people living with SBS.” . BASEL, Switzerland , Oct. chief executive officer of VectivBio. managing director at Versant Ventures and chairman of the board of directors at VectivBio.

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PPD

NOVEMBER 4, 2024

Large companies are particularly impacted by competition with other trials or sites , especially given low patient numbers for rare diseases and diversity requirements. Download the full report The post Global Survey Highlights Drug Development Challenges and Opportunities appeared first on PPD.

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The Pharma Data

NOVEMBER 26, 2020

(NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A.,

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The Pharma Data

SEPTEMBER 17, 2020

17, 2020 /PRNewswire/ — Eli Lilly and Company (NYSE:LLY) and Amgen (NASDAQ:AMGN) today announced a global antibody manufacturing collaboration to significantly increase the supply capacity available for Lilly ‘s potential COVID-19 therapies. INDIANAPOLIS and THOUSAND OAKS, Calif.

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PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes.

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The Pharma Data

DECEMBER 21, 2020

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. UNITY is a biotechnology company developing therapeutics to extend healthspan by slowing, halting or reversing diseases of aging. View original content to download multimedia: [link].

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KIF1A

AUGUST 28, 2024

KIF1A.ORG’s Chief Science Officer Dr. Dylan Verden will provide an overview of current areas of KAND therapeutic… Tuesday, September 24, 2024 5:00 pm – 6:00 pm Online via Zoom [link] ASO therapy: Positive Clinical Outcomes Observed We are thrilled to announce the publication of the **first-ever KAND ASO study** !

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The Pharma Data

MAY 4, 2021

INDIANAPOLIS , May 4, 2021 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) is donating COVID-19 therapies to Direct Relief, enabling the humanitarian organization to provide COVID-19 therapies at no cost to low- and lower-middle-income countries most heavily impacted by the pandemic.

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The Pharma Data

JANUARY 18, 2021

Potential first-in-class disease-modifying biologic in late-stage clinical studies. a clinical-stage biotechnology company focused on developing treatments for immunological and inflammatory diseases, today announced its launch backed by $80 million Series C financing led by Abingworth LLP.

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The Pharma Data

AUGUST 7, 2020

As the pharma industry stands firm in its commitment to advance the sector to fight Covid-19, news has emerged from the European Commission who intend to streamline the development of therapies using genetically modified organisms to treat Covid-19. Regenerative medicine and advanced therapies thriving despite Covid-19 disruption.

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The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

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The Pharma Data

MAY 16, 2021

Description: John Coleman, ND, was diagnosed with Stage IV Parkinson’s Disease in 1995. John Coleman ND talks about how he recovered from Parkinson’s Disease in his pioneering book Stop Parkin’ and Start Livin’ He is the first to say there is no cure for Parkinson’s. No magic bullet exists. Others were.

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The Pharma Data

AUGUST 19, 2020

(Momenta), a company that discovers and develops novel therapies for immune-mediated diseases, in an all cash transaction for approximately $6.5 Nipocalimab recently received a rare pediatric disease designation from the U.S. Food and Drug Administration. An estimated 2.5

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KIF1A

FEBRUARY 29, 2024

Dear KAND Community, Happy Rare Disease Day! Today we are celebrating our collective strength and numbers within our KAND community and with all rare diseases globally. Download and read the research flyer for more details and scan the QR code to fill out the interest form. If we all standup and be counted, we will be heard!

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The Pharma Data

MAY 4, 2021

Baricitinib is an oral medication currently registered in India for the treatment of moderate to severe active rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs. Test patients for latent TB before initiating Olumiant and during therapy.

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The Pharma Data

DECEMBER 9, 2020

The targeted indication is for treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. The following files are available for download: View original content: [link].

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The Pharma Data

NOVEMBER 25, 2020

CAN10 has been designed to block the signalling of the inflammatory cytokines IL-1, IL-33 and IL-36 resulting in unique properties for treatment of inflammatory diseases. 556791-6019, is a biotechnology company that develops antibody-based treatments for life-threatening diseases. About BioInvent. Cantargia AB (publ), reg.

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PPD

OCTOBER 31, 2024

These internal issues are compounded by external pressures like inflation, the development of innovative therapies that require complex protocols and the need to comply with shifting regulatory requirements. This issue is particularly pronounced for rare diseases and trials requiring diverse patient populations.

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Perficient: Drug Development

OCTOBER 5, 2023

These disabilities can be the result of various factors, including developmental disorders, acquired brain injuries, neurodegenerative diseases, and more. Sensory Processing Therapy : Offers a range of sensory-based activities and exercises to improve sensory processing skills.

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The Pharma Data

MAY 4, 2021

Amid the coronavirus pandemic, concern has grown over the potential neglect of other serious diseases. Multiple sclerosis (MS), for example, is a condition for which timely diagnosis, early treatment and the development of innovative therapies are particularly important for reducing the disabling progress of the disease.

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The ChEMBL-og

MAY 13, 2021

This paper on the 'Identification of the Core Chemical Structure in SureChEMBL Patents' has recently been published by Maria Falaguera and Jordi Mestres, and the resulting data set is available to download from the SureChEMBL FTP site: [link]. As mentioned above, this data set can be downloaded from SureChEMBL. of the total molecules).

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The Pharma Data

NOVEMBER 19, 2020

Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced that Andrew Robbins , Chief Executive Officer and President, will participate in a virtual fire side chat at the 3 rd Annual Evercore ISI HealthCONx Virtual Conference on Wednesday, December 2, 2020 at 10:55 a.m. .

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Perficient: Drug Development

SEPTEMBER 26, 2023

These disabilities can be the result of various factors, including developmental disorders, acquired brain injuries, neurodegenerative diseases, and more. While traditional therapies and interventions have proven beneficial, technology, particularly brain training apps, offers a new dimension of support.

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The Pharma Data

NOVEMBER 10, 2020

According to data published on the WHO website, more than 46 million people have been tested positive for COVID-19, with over 1 million patients in more than 200 counties died from the disease as of November 2 nd , 2020. SHANGHAI , Nov.

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The Pharma Data

JANUARY 22, 2021

Voclosporin follows the recent approval of belimumab by the FDA as new therapies indicated for lupus nephritis – both within the past month. Since lupus impacts people differently and can change over time, a multidisciplinary team approach is going to be key to ensuring better overall care and to minimizing end-stage kidney disease.”

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The Pharma Data

OCTOBER 20, 2020

CureLab’s partners in the region will test the efficacy of the company’s DNA-based products against cancers and non-cancerous diseases of chronic inflammation. “There are horrible diseases that are dominant in the GCC region and rarely seen in the West,” said Jihad Fakhreddine. BOSTON , Oct. Source link.

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The Pharma Data

JANUARY 13, 2021

Mr. Barrow has over a decade of experience leading drug development programs aimed at identifying and testing novel treatments in a wide range of disease conditions under FDA and EMA. . NEW YORK , Jan. MindMed Co-CEO J.R. ” Robert Barrow Bio : Mr. .”

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The Pharma Data

JANUARY 20, 2021

21, 2021 /PRNewswire/ — Dynacure , a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today the appointment of Jean M. View original content to download multimedia: [link].

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The Pharma Data

DECEMBER 1, 2020

IsoPlexis’ Single-Cell Intracellular Proteome solution has been recognized for its impact on the development of better targeted and edited therapies and its potential to innovate and accelerate medical research. View original content to download multimedia: [link]. BRANFORD, Conn. , BRANFORD, Conn. , SOURCE IsoPlexis.

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The Pharma Data

SEPTEMBER 11, 2020

While stories from news outlets indicate the peak of the infection has passed in most European countries, cases of the disease continue to rise in other parts of the world, especially Latin America and Africa. Covid-19 command center aims to accelerate clinical research by individual sponsors to combat disease.

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