pharmaphorum

OCTOBER 26, 2022

But Giacomo Chiesi, head of Chiesi Global Rare Diseases, thinks of his 6,000-person company as a family business. For example, at the SSIEM Annual Symposium earlier this month, the company shared data on a new candidate for the treatment of Fabry disease, a rare genetic blood disorder. Caring for a global community. “We

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Pharmaceutical Technology

AUGUST 12, 2022

In the lead-up to a critical meeting that will influence the approval of a new drug for amyotrophic lateral sclerosis (ALS), the US Food and Drug Administration (FDA) has detailed its plans to advance rare neurodegenerative disease research. There are only two marketed treatments for ALS in the US.

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Pharmaceutical Technology

MAY 8, 2023

110 innovations will shape the pharmaceutical industry According to GlobalData’s Technology Foresights, which plots the S-curve for the pharmaceutical industry using innovation intensity models built on over 756,000 patents, there are 110 innovation areas that will shape the future of the industry.

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pharmaphorum

NOVEMBER 27, 2020

Amanda Barrell explores how a perfect storm of changing economics, advances in technology, and the increasing volume of the patient voice is stoking change in the rare disease space. That was among the discussion points during Fighting Rare Diseases – The Science, Economics and the Patients , a webinar hosted by o2h Group.

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pharmaphorum

SEPTEMBER 22, 2021

Family businesses across industries including the life sciences sector contribute significantly to the global economy – they represent about half of the world gross domestic product and global employment. In the life sciences sector, the overall goal is to develop and commercialise innovative pharmaceutical solutions to improve health.

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pharmaphorum

DECEMBER 21, 2021

There are currently an estimated 300 million people living with one or more Rare Diseases across the world. There are over 7,000 Rare Diseases that have been identified, yet only 5% have treatments. One factor is the lack of substantive data about Rare Diseases, which makes understanding specific diseases very difficult.

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pharmaphorum

MARCH 10, 2022

The Netherlands-based biotech, which is putting together a pipeline of genetic modifier drugs for cancer and rare diseases, said the new funding would also allow it to double its headcount to add clinical capabilities and move to a larger facility. Others working in this area include Maze Therapeutics in the US.

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Pfizer

AUGUST 3, 2022

“This development confirms the complexity of advancing new treatments for rare cardiovascular diseases and the need to further increase knowledge in this space. Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development. About Pfizer Rare Disease.

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pharmaphorum

MARCH 10, 2022

Biologics have revolutionised treatments for several serious diseases, some of which had no therapeutic alternatives until now,” says Antonio Magnelli, head of the Chiesi Group’s Global Manufacturing Division. “As Yet, despite the immense potential of the field, biologics have remained largely untapped.

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XTalks

JUNE 29, 2022

The FDA’s action plan will encourage the development of drugs for rare neurodegenerative diseases and help streamline clinical trial operations. Neurodegenerative diseases involve the progressive degeneration of the function and structure of the central nervous system (CNS) or peripheral nervous system.

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pharmaphorum

FEBRUARY 11, 2022

Komodo Health’s president and co-founder, Web Sun, tells us about his company’s new partnership with the Chan Zuckerberg Initiative (CZI) to improve early diagnosis and advance research of rare diseases by providing CZI’s Rare as One Network healthcare data and software. The benefits of accessibility.

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FDA Law Blog

JUNE 2, 2022

This meeting brings together industry, regulators, academics, and patients to discuss global and local challenges in the life sciences industry. FDA Rare Disease Town Hall with CDER & CBER Center Directors (June 22, 2-3pm Central). Hyman, Phelps & McNamara, P.C.’s More information can be found here.

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pharmaphorum

APRIL 8, 2022

With more than 6,000 different diseases comprising the rare disease (RD) landscape, connecting patients and practitioners with the expertise needed to achieve the correct diagnosis can be a significant barrier to treatment. . Optimising and exploiting data for use in rare disease-specific research and studies.

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pharmaphorum

OCTOBER 22, 2020

Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences investors. He has previously worked as vice president of sales at Vertex Pharma, and had various commercial roles at Gilead Sciences.

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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.

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pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. At the moment it’s mostly being applied to ultra-rare diseases. The exact mechanism depends on the disease in question. First of all it will be used for ultra-rare monogenic disease.

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Drug Discovery World

NOVEMBER 10, 2023

With the first DDW Turning Science into Business Summit taking place next week on the theme of Cancer Research, this dynamic area of drug discovery is my round-up focus again. News round-up for 6-10 November by DDW Digital Content Editor Diana Spencer.

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Drug Discovery World

APRIL 5, 2023

Two key regenerative medicines in late stages are beremagene geperpavec, a gene therapy by Krystal Biotech for the treatment of epidermolysis bullosa, a rare dermatological condition, and Yescarta, a gene-modified cell therapy by Gilead Sciences indicated for nodal marginal zone B-cell lymphoma.

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pharmaphorum

MAY 18, 2022

Considerable scientific progress across neurological diseases and disorders was reported at the recent 74th American Academy of Neurology (AAN) Annual Meeting. One research-driven company with a strong patient focus in neurology is UCB. “In In everything we do, our guiding question is: What difference will this make for patients?”

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Pharmaceutical Technology

NOVEMBER 29, 2022

Now, efforts are underway to study ketamine’s effects in Parkinson’s disease, fibromyalgia, and Rett syndrome. On November 14, the Canadian developer PharmaTher announced positive data from a Phase I/II study where ketamine was used to treat levodopa-induced dyskinesia in Parkinson’s disease.

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pharmaphorum

DECEMBER 7, 2022

Having recently assessed the relationship between drug price and prevalence of non-oncology rare diseases, CRA’s Life Sciences Practice team explores in greater depth the situation for these orphan drugs in France in this final article of a three-part series. Rare diseases: a flawed system.

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Drug Discovery World

FEBRUARY 19, 2024

Financial landscape In 2023, life science companies experienced a significant decrease in funding, particularly seed funding for early-stage drug development. Throughout 2023, oncology and rare disease indications consistently dominated the pipeline for advanced therapies, and this trend is expected to continue in 2024.

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pharmaphorum

NOVEMBER 23, 2021

“The emerging mRNA technology has rapidly advanced the science of vaccine and drug development for infectious diseases like COVID-19,” said Tian Wu, Baidu’s corporate vice president. billion earlier this year. The post Chinese tech giant Baidu licenses mRNA algorithm to Sanofi appeared first on.

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pharmaphorum

JUNE 2, 2022

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. As covered in a previous article , the importance of R&D to develop treatments for rare diseases is high. of the global population are affected by a rare disease.

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pharmaphorum

JANUARY 13, 2023

Sanofi Ventures invests in innovators working in areas including immunology and inflammation, rare diseases, oncology, cell and gene therapy, vaccines, and digital health and data science. The team partners across all stages of the private company lifecycle, from Seed to Series B and beyond.

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Drug Discovery World

FEBRUARY 12, 2024

Cameron Ross, SVP, Generative AI, Elsevier “AI has the potential to dramatically shorten one of the most costly areas of early drug development – small molecule discovery. It’s no surprise that interest in AI for this area is surging, since early phase drug discovery normally accounts for 42% of the cost of the entire pipeline.

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Drug Discovery World

AUGUST 28, 2023

DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. But it’s now known that if it stops working this can lead to many different diseases. What is autophagy?

Genetics 52

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XTalks

DECEMBER 12, 2023

These components make plasma an invaluable resource for treating various medical conditions, especially those that are rare and chronic. The plasma sector, perhaps because our therapeutics treat diseases and conditions that affect smaller patient populations, doesn’t draw as much attention.

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The Pharma Data

JULY 28, 2021

As published in Science , the new target was found by sequencing nearly 650,000 people and identifying individuals with rare protective mutations. 1 Obesity and insulin resistance are key drivers in the development of type-2 diabetes and often lead to cardiorenal complications, as well as liver disease. Yancopoulos, M.D.,

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Pharma Marketing Network

MARCH 2, 2022

According to Murray Aitken, IQVIA’s Senior VP and Executive Director of the IQVIA Institute for Human Data Science, “the pandemic has accelerated biopharmaceutical innovation.” 3 therapeutic areas with the most growth are oncology, gastrointestinal disorders, and neurology. IQVIA’s Report Highlights.

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The Pharma Data

FEBRUARY 22, 2022

Next-generation respiratory inhalers Most patients with asthma and chronic obstructive pulmonary disease (COPD) need inhaled medicines 2 and many make use of pressurised metered dose inhalers (pMDIs) 2 which contain small quantities of a type of GHG which acts as the propellant to deliver the medicine into the lungs.

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Drug Discovery World

SEPTEMBER 20, 2022

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. Long-reads are often applied in complex areas, such as rare diseases and neurodegenerative diseases, which require deeper insights into more regions of the human genome that cannot be achieved with short read alone. .

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Drug Discovery World

DECEMBER 20, 2022

As 2022 draws to a close, life sciences market analysts attempt to correctly predict what the next 12 months might bring to the drug discovery sector. A focus on the representation of diverse patient populations in clinical trials will be a very good thing for the industry, but could be challenging.

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The Pharma Data

JANUARY 24, 2021

–( BUSINESS WIRE )– Faze Medicines , a biotechnology company pioneering therapeutics based on the groundbreaking new science of biomolecular condensates, today announced that it has appointed Philip Vickers, Ph.D., 25, 2021 12:00 UTC. CAMBRIDGE, Mass.–( as its chief executive officer (CEO). He earned his B.Sc.

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Pharma Marketing Network

MARCH 2, 2022

According to Murray Aitken, IQVIA’s Senior VP and Executive Director of the IQVIA Institute for Human Data Science, “the pandemic has accelerated biopharmaceutical innovation.” 3 therapeutic areas with the most growth are oncology, gastrointestinal disorders, and neurology. IQVIA’s Report Highlights.

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Pfizer

SEPTEMBER 27, 2022

We need YOU to design for science! We need YOU to design for science! More of us than ever need science to restore not just the health of our loved ones, but life as we remember it. The  The power of science is the key to unlocking the answers to the world’s most challenging medical questions. Calling All Creatives!

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The Pharma Data

MAY 31, 2022

In Japan, it is indicated for the prevention of coronavirus disease 2019 (COVID-19) caused by SARS-CoV-2. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both ground-breaking science and rapid scaling of manufacturing. About Takeda.

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