Rare disease Archives

News and Trends 3 Oct 2022

Alexion, AstraZeneca Rare Disease to acquire genomic medicine company LogicBio Therapeutics

LogicBio Therapeutics, Inc. has entered into a definitive agreement under which Alexion, AstraZeneca Rare Disease (Alexion) will acquire LogicBio. The proposed acquisition brings LogicBio’s technology, experienced rare disease R&D team, and expertise in pre-clinical development to support Alexion’s growth in genomic medicines. Fred Chereau, president and chief executive officer at LogicBio, said: “We are excited […]

October 3, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 3 Oct 2022

Poxel granted orphan drug designation for kidney disease treatment

French biopharma company Poxel SA says the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PXL770 for the treatment of patients with autosomal-dominant polycystic kidney disease (ADPKD). PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator – and is also a phase 2 ready ADPKD asset, subject […]

October 3, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 27 Sep 2022

Protocol for treatment of neurotrophic keratopathy submitted to FDA by ReGenTree

Protocol for a second phase 3 clinical trial for a treatment for neurotrophic keratopathy (NK) – a degenerative disease of the corneal epithelium resulting from impaired corneal innervation, has been submitted. RegeneRx Biopharmaceuticals reported that its U.S. joint venture partner and licensee, HLB Therapeutics, together called ReGenTree, made the submission to the U.S. Food and […]

September 27, 2022 - 3 minutesmins - By Liza Laws

News and Trends 23 Sep 2022

Grant awarded to help bring to market an innovative treatment for bone cancer

A grant has been awarded to look into the feasibility of bringing to market a new therapy to treat osteosarcoma that aids bone regeneration with fewer side-effects. The European Research Council gave the Proof of Concept grant to professor Cristina Canal from the Technical University of Catalunya’s (UPC) department of materials science and engineering who […]

September 23, 2022 - 3 minutesmins - By Liza Laws

News and Trends 21 Sep 2022

Poolbeg to investigate melioidosis vaccine candidate

Poolbeg Pharma has signed an exclusive agreement with University College Dublin (UCD) through NovaUCD, the university’s knowledge transfer office, for a late preclinical stage vaccine candidate for melioidosis, a disease for which there is no current approved vaccine available.The vaccine candidate, which is being developed by Poolbeg Pharma as POLB 003, was invented following many […]

September 21, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 20 Sep 2022

First treatment of rare kidney disease launched in Germany by STADA Arzneimittel

The first and only approved treatment for primary immunoglobin A (IgA) nephropathy, a rare, progressive disease of the kidneys, has been launched in Germany. European commercial partner of Calliditas Therapeutics AB, STADA Arzneimittel AG, says the treatment will launch in additional European countries later. The disease, also known as Berger’s disease, occurs when immunoglobin builds […]

September 20, 2022 - 3 minutesmins - By Liza Laws

News and Trends 13 Sep 2022

Aldatu gets grant to develop Crimean Congo hemorrhagic fever test

U.S. biotech company Aldatu Biosciences has been awarded a $3 million Direct-to-Phase II Small Business Innovation Research (SBIR) grant from the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH). The award will fund the advancement of the company’s proprietary PANDAA technology platform and its specific application to the […]

September 13, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 12 Sep 2022

Sensorion receives positive opinion from EMA for treatment of hearing loss

The European Medicines Agency (EMA) through its Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion for a treatment for otoferlin gene mediated hearing loss. Clinical-stage biotech, Sensorion, announced the news today (September 12) that its application for an Orphan Drug Designation (ODD) for its lead therapy gene candidate, OTOF-GT will now be […]

September 12, 2022 - 3 minutesmins - By Liza Laws

News and Trends 12 Sep 2022

Ayala Pharmaceuticals presents interim data on desmoid tumor trial

Clinical oncology company Ayala Pharmaceuticals, Inc. has announced updated, positive interim results from part A of its ongoing RINGSIDE pivotal phase 2/3 clinical trial evaluating investigational new drug AL102 in desmoid tumors. Ayala Pharmaceutical’s AL102 is a selective, oral gamma-secretase inhibitor. The data are being featured in an oral presentation at the European Society for […]

September 12, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 12 Sep 2022

SiSaf reveals positive preclinical data in siRNA program to treat osteopetrosis

SiSaf Ltd, an RNA delivery and therapeutics company, has announced positive data confirming safety and efficacy of its Bio-Courier next generation silicon stabilized hybrid lipid nanoparticles (sshLNP) as an effective non-viral delivery system for autosomal dominant osteopetrosis type 2 (ADO2) siRNA therapy. The data was presented at the American Society of Bone and Mineral Research […]

September 12, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 7 Sep 2022

Ionis presents positive study results at International Symposium on amyloidosis

Ionis Pharmaceuticals, Inc. has presented positive results from a planned 35-week interim analysis of the phase 3 NEURO-TTRansform study of Ionis and AstraZeneca’s eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). In the study, eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and secondary efficacy endpoints compared to […]

September 7, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 5 Sep 2022

Avacta’s drug targeting soft tissue sarcoma granted Orphan Drug Designation by FDA

A drug to treat soft tissue sarcoma has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). Clinical stage oncology drug company Avacta Group plc has produced a form of the generic chemotherapy, doxorubicin AVA6000. About Avacta’s drug The drug has been modified using the company’s pre|CISION technology and Affirmer […]

September 5, 2022 - 2 minutesmins - By Liza Laws

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Rare disease

Alexion, AstraZeneca Rare Disease to acquire genomic medicine company LogicBio Therapeutics

Poxel granted orphan drug designation for kidney disease treatment

Protocol for treatment of neurotrophic keratopathy submitted to FDA by ReGenTree

Grant awarded to help bring to market an innovative treatment for bone cancer

Poolbeg to investigate melioidosis vaccine candidate

First treatment of rare kidney disease launched in Germany by STADA Arzneimittel

Aldatu gets grant to develop Crimean Congo hemorrhagic fever test

Sensorion receives positive opinion from EMA for treatment of hearing loss

Ayala Pharmaceuticals presents interim data on desmoid tumor trial

SiSaf reveals positive preclinical data in siRNA program to treat osteopetrosis

Ionis presents positive study results at International Symposium on amyloidosis

Avacta’s drug targeting soft tissue sarcoma granted Orphan Drug Designation by FDA

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Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

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

These 7 biotechs are shaping the next generation of endometriosis care

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