BioTech 365

DECEMBER 6, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Investors punish Aeglea for missed rare disease secondaries, sending stock spiraling despite primary endpoint success.Investors punish Aeglea for missed rare disease secondaries, sending stock spiraling despite primary … (..)

Research 40

Research 40

BioTech 365

APRIL 13, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Recursion, targeting cancer, rare disease midstage tests, sets terms for $306M IPO.Recursion, targeting cancer, rare disease midstage tests, sets terms for $306M IPO badams Tue, 04/13/2021 – … Continue reading → (..)

Research 40

Research 40

BioTech 365

FEBRUARY 3, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: GSK tosses out cancer, rare disease meds amid Q4 clear-out.GSK tosses out cancer, rare disease meds amid Q4 clear-out badams Wed, 02/03/2021 – 08:10 from FierceBiotech: Biotech … Continue reading →

Research 40

Research 40

BioTech 365

JUNE 18, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: FDA rejects Orphazyme’s rare disease drug, continuing wild ride for Danish meme stock.FDA rejects Orphazyme’s rare disease drug, continuing wild ride for Danish meme stock ntaylor Fri, … Continue reading → (..)

Drugs 40

Drugs Research 40

BioTech 365

FEBRUARY 4, 2022

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: UCB races to regulators after $2.5B rare disease bet delivers data for attack on AstraZeneca and argenx.UCB races to regulators after $2.5B rare disease bet delivers data … Continue reading →

Regulation 40

Regulation Research 40

BioTech 365

DECEMBER 18, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Mereo BioPharma pens $50M Ultragenyx deal for rare bone disease drug.Mereo BioPharma pens $50M Ultragenyx deal for rare bone disease drug badams Fri, 12/18/2020 – 07:32 from … Continue reading →

Drugs 40

Drugs Research 40

BioTech 365

AUGUST 23, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Genevant pens $303M biobucks pact with Takeda, aiming at rare liver diseases using gene therapy.

Gene Therapy 40

Gene Therapy Gene Research 40

BioTech 365

OCTOBER 23, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Retrophin buys up Orphan Technologies, spending $90M on rare disease drug.Retrophin buys up Orphan Technologies, spending $90M on rare disease drug badams Fri, 10/23/2020 – 05:07 from … Continue reading →

Drugs 40

Drugs Research 40

Pharmaceutical Technology

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. The BGTC was launched in October 2021, as the first AMP initiative.

Gene Therapy 227

Gene Therapy Gene Research Clinical Trials 227

BioTech 365

DECEMBER 14, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Gene therapy player InnoSkel debuts with €20M for rare skeletal diseases.Gene therapy player InnoSkel debuts with €20M for rare skeletal diseases badams Mon, 12/14/2020 – 08:13 from … Continue reading →

Gene Therapy 40

Gene Therapy Gene Research 40

BioTech 365

JULY 15, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease.Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease … Continue reading → (..)

Gene Therapy 40

Gene Therapy Gene Research 40

BioTech 365

AUGUST 26, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: AstraZeneca’s $39B Alexion buyout bears fruit, yielding late-phase win for potential blockbuster rare disease drug.AstraZeneca’s $39B Alexion buyout bears fruit, yielding late-phase win for potential blockbuster rare (..)

Drugs 40

Drugs Research 40

BioTech 365

APRIL 23, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Sanofi starts off ‘pipeline-in-a-product’ rilzabrutinib in rare skin disease, but eyes bigger prize.Sanofi starts off ‘pipeline-in-a-product’ rilzabrutinib in rare skin disease, but eyes bigger prize aarmstrong (..)

Production 40

Production Research 40

BioTech 365

JANUARY 28, 2022

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Yet another delay at Amryt as EMA calls in experts to inform review of rare disease drug .Yet Yet another delay at Amryt as EMA calls in experts to … Continue reading →

Drugs 40

Drugs Research 40

BioTech 365

OCTOBER 25, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Intellia, LogicBio fuel hope for in vivo gene editing with new animal data in rare diseases: report.Intellia, LogicBio fuel hope for in vivo gene editing with new … Continue reading →

Gene Editing 40

Gene Editing In-Vivo Gene Research 40

BioTech 365

DECEMBER 9, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Albireo CMO Horn is out the door 5 months after US, EU approvals of rare liver disease drug.Albireo CMO Horn is out the door 5 months after … Continue reading →

Drugs 40

Drugs Research 40

BioTech 365

JANUARY 24, 2022

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: 1b, or not 1b: FDA hits Viking with clinical hold over mix-up in phase of rare disease trial.1b, 1b, or not 1b: FDA hits Viking with clinical hold … Continue reading →

Trials 40

Trials Research 40

BioTech 365

JANUARY 7, 2022

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: AstraZeneca picks up new weapon in rare disease fight with Alnylam and Pfizer, paying $30M for rights to an early-phase asset.AstraZeneca picks up new weapon in rare … Continue reading →

Research 40

Research 40

BioTech 365

DECEMBER 29, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: AstraZeneca taps Ionis to join crowd of companies gunning for rare heart disease in deal worth billions in biobucks.AstraZeneca taps Ionis to join crowd of companies gunning … Continue reading →

Research 40

Research 40

Pharma Times

NOVEMBER 10, 2023

A rare disease is defined as a condition that affects fewer than one in 2,000 people - News - PharmaTimes

118

118

BioTech 365

NOVEMBER 30, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: New DNA scanning method could lead to quicker diagnosis of cancer and rare disease.Understanding the sequence of human DNA gives scientists information about diseases, including potentially how … Continue reading → (..)

DNA 40

DNA Scientist Research 40

Pharmaceutical Technology

DECEMBER 27, 2022

Tracking the news. Alzheimer’s disease drugs were constantly in the news, and not always for the right reasons. Irena Maragkou kickstarted an exclusive Rare Disease Spotlight series, with the first edition tracking how the popularity of the FDA’s Orphan Drug designation program, has increased over almost 40 years.

Clinical Trials 306

Clinical Trials Clinical Trials Trials Vaccination 306

STAT News

AUGUST 15, 2022

In the United States, the circumstances around monkeypox vaccine are a good news, bad news kind of story. The good news is that there is a licensed vaccine. At the start of many outbreaks of new or rare diseases, there are no vaccines. But the bad news quickly overtakes the good. In the U.S.,

Licensing 98

Licensing Licensing Vaccination Vaccine 98

XTalks

AUGUST 21, 2023

The US Food and Drug Administration (FDA) has approved Ipsen’s groundbreaking Sohonos (palovarotene) capsules for the treatment of fibrodysplasia ossificans progressive (FOP), an ultra-rare bone disease. FOP is an extremely rare disease, occurring in an estimated one out of every two million individuals.

Drugs 98

Drugs FDA Approval Genetics Clinical Trials 98

Worldwide Clinical Trials

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. He found out he had brain cancer — a rare, aggressive brain cancer with only a 15% chance of living even another five years. “To Uplifting Athletes (UA) is on a mission to change that. To say it was devastating is an understatement.

Clinical Trials 130

Clinical Trials Clinical Trials Research Trials 130

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

Drug Discovery World

NOVEMBER 15, 2023

“We are pleased that eligible patients in England and Wales will now be able to access velmanase alfa on the NHS for the first time,” said Dr Kamran Iqbal, Head of Medical Affairs, Global Rare Diseases, Chiesi UK&I. Alpha-mannosidosis is an extremely rare, genetic condition, thought to affect around 25 people in England. “On

Genetics 52

Genetics Drugs Genetic Disease 52

STAT News

SEPTEMBER 29, 2022

The news Tuesday night that a clinical trial of an experimental Alzheimer’s therapy had succeeded hit like a blast — at last, a rare win in a disease devastating nearly 6 million Americans and countless more caregivers.

Clinical Trials 128

Clinical Trials Clinical Trials Trials Drugs 128

ACRP blog

SEPTEMBER 27, 2023

Food and Drug Administration’s (FDA’s) Rare Disease Endpoint Advancement (RDEA) Pilot Program , the clinical research enterprise gains new support for efforts aimed at efficacy endpoint (or clinical outcome) development and the timely approval of drugs and biological products that treat rare diseases, including rare diseases in children.

Clinical Trials 64

Clinical Trials Clinical Trials Trials Gene Therapy 64

pharmaphorum

FEBRUARY 28, 2022

Amryt Pharma has been handed a major blow by the FDA, after the US regulator rejected its marketing application for Oleogel-S10, its drug for rare and debilitating skin disease epidermolysis bullosa (EB). 21-year-old Faith from Wisconsin lives with #EpidermolysisBullosa , one of over 6000 rare diseases worldwide.

Drugs 96

Drugs Marketing Regulation FDA Approval 96

Drug Discovery World

DECEMBER 9, 2022

News round-up for 5-9 December by DDW Digital Content Editor Diana Spencer. Teamwork makes the dream work’ this week, with the launch of new partnerships to further the development of precision drugs in neurodevelopmental disorders, lung cancer and rare paediatric diseases. The top stories: .

Drugs 52

Drugs Vaccination Vaccine Trials 52

Drug Discovery World

SEPTEMBER 23, 2022

News round-up for 19-23 September by DDW Editor Reece Armstrong. An interesting week for clinical research and drug approvals, with rare diseases being a key focus for both the FDA and European Commission. EC approves first treatment for rare kidney disease. This week’s top stories: .

Drugs 52

Drugs FDA Approval Gene Therapy Genetics 52

Drug Discovery World

JANUARY 12, 2024

News round-up for 8-12 January by DDW Digital Content Editor Diana Spencer. Perhaps most importantly, a new antibiotic was found that targets a Gram-negative pathogen, mRNA was used as an effective therapy for a rare liver disease, and a common immune cell was found to attack cancer.

Drugs 52

Drugs Immune Response Allergies Bacteria 52

Fierce Pharma

JULY 17, 2023

The news sent Apellis' share price down 32% to $52.62 by Monday afternoon.

FDA Approval 92

FDA Approval Doctors Doctor 92

Pharmaceutical Technology

APRIL 19, 2023

Bayer has announced that it will shift the focus of its drug research away from women’s health to four core therapeutic areas: oncology, cardiovascular, neurology and rare diseases/immunology.

Gene Therapy 130

Gene Therapy Hormones Production Sales 130

XTalks

NOVEMBER 30, 2023

Pfizer spinout SpringWorks Therapeutics’ Ogsiveo (nirogacestat) has received US Food and Drug Administration (FDA) approval for the treatment of desmoid tumors, an ultra-rare subtype of non-cancerous soft tissue sarcomas that can cause severe pain and disfigurement.

FDA Approval 101

FDA Approval Trials Clinical Trials Clinical Trials 101

pharmaphorum

JANUARY 9, 2023

per ADS, representing an approximate additional $225 million of potential consideration, based on certain milestones being achieved with Amryt’s Filsuvez, that will see the Italian company expand its rare disease medicine portfolio. The post Chiesi expands rare disease portfolio with Amryt Pharma acquisition appeared first on.

Sales 86

Sales Medicine Marketing Development 86