Rare disease Archives

News and Trends 24 Oct 2022

Syncona to acquire Applied Genetic Technologies Corporation

Healthcare company Syncona Limited says its newly established portfolio company has agreed to acquire, by a tender offer, all outstanding shares of Applied Genetic Technologies Corporation (AGTC). AGTC is a clinical-stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an […]

October 24, 2022 - 2 minutesmins - By Jim Cornall

In Depth 20 Oct 2022

Opportunities and challenges for cell and gene therapy investments

Companies in the cell and gene therapy space are struggling to raise investments amid adverse market conditions. However, this could be an opportunity for the strongest companies and investors to shine. After several years of record-breaking investments, funding is hard to come by in the gene and cell therapy space. Part of this trend relates […]

October 20, 2022 - 9 minutesmins - By Jonathan Smith

News and Trends 13 Oct 2022

CMT Research Foundation funding boosts Samsara Therapeutics Charcot-Marie-Tooth treatment

The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, has invested in Samsara Therapeutics Inc. Samsara Therapeutics is a biotech company focusing on discovering and developing therapeutics to restore autophagy – the process by which cells recycle damaged or dysfunctional components. Samsara is developing several novel, […]

October 13, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 4 Oct 2022

ASPIRE set up to collaborate on amyloidosis

A new group of biotech and pharma companies has been assembled by the Amyloidosis Research Consortium (ARC). ASPIRE (amyloidosis stakeholder partnerships for impact reach and equity), is a collaboration between biotech and pharmaceutical companies committed to advancing solutions, improving diagnosis and systems of care, and addressing obstacles to health equity on behalf of amyloidosis patients. […]

October 4, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 4 Oct 2022

Aeglea BioTherapeutics updates homocystinuria program

Aeglea BioTherapeutics, Inc. says dosing in the third cohort of the phase 1/2 clinical trial of pegtarviliase for the treatment of classical homocystinuria is under way. Patients in this cohort are receiving 1.35 mg/kg of pegtarviliase once weekly administered via subcutaneous injection; patients in cohorts 1 and 2 received doses of 0.15 mg/kg and 0.45 […]

October 4, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 3 Oct 2022

FDA clears Kira Pharmaceuticals’ phase 2 trial for SLE-TMA treatment

Kira Pharmaceuticals says the US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for KP104, a first-in-class bifunctional biologic that selectively and synergistically targets the alternative and terminal complement pathways. A phase 2 trial will evaluate the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of KP104 in participants with […]

October 3, 2022 - 3 minutesmins - By Jim Cornall

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

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Rare disease

Syncona to acquire Applied Genetic Technologies Corporation

Opportunities and challenges for cell and gene therapy investments

CMT Research Foundation funding boosts Samsara Therapeutics Charcot-Marie-Tooth treatment

ASPIRE set up to collaborate on amyloidosis

Aeglea BioTherapeutics updates homocystinuria program

FDA clears Kira Pharmaceuticals’ phase 2 trial for SLE-TMA treatment

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

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