Rare disease Archives

News and Trends 29 Mar 2023

Pharming immunodeficiency drug given FDA approval

Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Pharming’s Joenja, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the […]

March 29, 2023 - 4 minutesmins - By Jim Cornall

Interview 3 Mar 2023

Interview: how has the first gene therapy for hemophilia B transformed research?

Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective clotting protein factor IX often due to a spontaneous mutation in the F9 gene, leading to excessive bleeding. Until recently, routine prophylactic infusions of factor IX replacement therapy to maintain enough clotting factor to prevent […]

March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter

In Depth 3 Mar 2023

Let’s Take a Closer Look at 7 Autoimmune Diseases Biotechs are Fighting to Treat

While we mostly think of disease as being caused by bacteria or viruses, an illness can often occur when the body’s own defenses turn against itself. Here we’ll take a look at seven rare autoimmune diseases you may not have heard of and what biotechs are doing to treat them. The body’s immune system can […]

March 3, 2023 - 8 minutesmins - By Alexander Burik

News and Trends 28 Feb 2023

What’s happening on Rare Disease Day?

Rare Disease Day 2023 is on February 28. The day is dedicated to raising awareness for the approximately 300 million people around the world living with a rare disease. More than 6,000 rare diseases are characterized by a diversity of disorders and symptoms that vary not only from disease to disease, but also from patient […]

February 28, 2023 - 8 minutesmins - By Jim Cornall

News and Trends 28 Feb 2023

European Commission approves first gene therapy for hemophilia B

The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […]

February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter

News and Trends 28 Feb 2023

GC Biopharma acquiring hemophilia rare disease pipeline

GC Biopharma Corp., a South Korean provider of biopharmaceutical products, has signed an asset purchase agreement with Catalyst Biosciences to acquire three programs related to the orphan hematology disorders. The agreement includes Marzeptacog alfa (MarzAA), an engineered factor VIIa, which is ready for phase 3 clinical stage development. In its previous clinical development trials, MarzAA […]

February 28, 2023 - 2 minutesmins - By Jim Cornall

Opinion 28 Feb 2023

Life changing treatments for rare disease – a beacon of hope

By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease. This number does not even take into account the impact on these individuals’ support units that includes family, friends and caregivers. A rare disease is defined as a disease that affects no more than 1 […]

February 28, 2023 - 6 minutesmins - By External Contributor

Best in Biotech 28 Feb 2023

5 advancements in rare disease research over the past year

There are more than 7,000 rare diseases that affect hundreds of millions of people worldwide. As Rare Disease Day is observed on February 28, here are five recent advancements that have transformed research and boosted the scope for the development of novel therapies. FDA approves tissue-agnostic therapies for rare cancers Tissue-agnostic drug development has been […]

February 28, 2023 - 6 minutesmins - By Roohi Mariam Peter

Best in Biotech 28 Feb 2023

10 biotech companies making a difference in rare diseases

February 28 is Rare Disease Day, which looks to raise awareness of rare diseases and bring about change for patients and families living with them. The event also brings attention to the need for improved access to diagnosis, as well as the need to develop more treatments to tackle a broader range of diseases. There […]

February 28, 2023 - 11 minutesmins - By Willow Shah-Neville

News and Trends 24 Feb 2023

Beyond Biotech podcast 35: Rare Disease Day

As February 28 is Rare Disease Day, we have three guests on the podcast today all relevant to rare diseases. And they are Dan Mandell, CEO and co-founder of GRO Biosciences; Stefano Portolano, chief executive officer, Azafaros; and from Replay, Adrian Woolfson, executive chairman, president, and co-founder, and Lachlan MacKinnon, CEO and co-founder. Rare Disease […]

February 24, 2023

News and Trends 23 Feb 2023

Moderna and Life Edit Therapeutics look to speed up gene editing therapies

Moderna, Inc., a biotechnology company working on messenger RNA (mRNA) therapeutics and vaccines, and Life Edit Therapeutics Inc., an ElevateBio company focused on gene editing technologies and therapeutics, have announced a strategic research and development collaboration to discover and develop in vivo mRNA gene editing therapies. The partnership will combine Life Edit’s suite of proprietary […]

February 23, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 21 Feb 2023

First patient enrolled in Pharming APSD trial

Pharming Group N.V. says the first patient has been enrolled in its phase III clinical trial evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children with activated phosphoinositide 3-kinase delta syndrome (APDS). There is currently no approved treatment for this complex and progressive disease caused by genetic variants. At […]

February 21, 2023 - 6 minutesmins - By Jim Cornall

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T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

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Rare disease

Pharming immunodeficiency drug given FDA approval

Interview: how has the first gene therapy for hemophilia B transformed research?

Let’s Take a Closer Look at 7 Autoimmune Diseases Biotechs are Fighting to Treat

What’s happening on Rare Disease Day?

European Commission approves first gene therapy for hemophilia B

GC Biopharma acquiring hemophilia rare disease pipeline

Life changing treatments for rare disease – a beacon of hope

5 advancements in rare disease research over the past year

10 biotech companies making a difference in rare diseases

Beyond Biotech podcast 35: Rare Disease Day

Moderna and Life Edit Therapeutics look to speed up gene editing therapies

First patient enrolled in Pharming APSD trial

Subscribe to our newsletter to get the latest biotech news!

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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