Rare disease Archives

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

News and Trends 21 Feb 2023

FDA approves alpha-mannosidosis drug

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), says the U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder […]

February 21, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 17 Feb 2023

Positive results for INOVIO recurrent respiratory papillomatosis trial

INOVIO Pharmaceuticals has announced positive preliminary results from the second cohort of its phase 1/2 clinical trial evaluating INO-3107 for the treatment of HPV 6 and HPV 11-associated recurrent respiratory papillomatosis (RRP) in adults. In the second cohort of 11 patients who were administered INO-3107 via the exploratory side port needle, 10 of the 11 […]

February 17, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

Janssen reports positive topline phase 2 HDFN results

The Janssen Pharmaceutical Companies of Johnson & Johnson has announced positive topline results from the proof-of-concept phase 2 open-label UNITY clinical trial for the treatment of pregnant adults at high risk for severe hemolytic disease of the fetus and newborn (HDFN). HDFN is a serious and rare condition that can cause life-threatening anemia in the […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 19 Jan 2023

Hundreds of genetic discoveries from FinnGen study

New results from the FinnGen research consortium include previously unknown genetic risk factors for many debilitating diseases. These findings have potential to facilitate the development of new therapies. Since initiation in 2017, the FinnGen study has developed into one of the world’s leading biobank-based genomic research projects. Currently, FinnGen is completing the construction of a […]

January 19, 2023 - 4 minutesmins - By Jim Cornall

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Rare disease

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

FDA approves alpha-mannosidosis drug

Positive results for INOVIO recurrent respiratory papillomatosis trial

Janssen reports positive topline phase 2 HDFN results

Hundreds of genetic discoveries from FinnGen study

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10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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