Rare disease Archives

News and Trends 14 Nov 2022

InfanDx tackles early detection of hypoxic ischemic encephalopathy

InfanDx AG, a privately held diagnostics company focusing on the development and commercialization of novel diagnostic solutions for newborns, has completed the validation of metabolic biomarker panels for the early detection of hypoxic ischemic encephalopathy (HIE) in newborns. The validated biomarkers of such panels will be the target of the company’s HypoxE blood test for […]

November 14, 2022 - 8 minutesmins - By Jim Cornall

News and Trends 14 Nov 2022

A biotech boom manifesting real results in rare pediatric diseases

By Bob Coughlin, managing director, life sciences, JLL For the last two years, the pandemic has resulted in the explosive growth in life sciences. The world watched in awe and anticipation as biotech successfully raced to develop vaccines, therapeutics and diagnostics for Covid-19. It has been a good moment for an industry that deserves such […]

November 14, 2022 - 5 minutesmins - By External Contributor

centogene rare diseases diagnostic ipo nasdaq

News and Trends 4 Nov 2022

Centogene launches rare and neurodegenerative disease Biodata Network

German company, Centogene N.V., today (November 4) announced its launch of Biodata Network, a portfolio of data-driven partnering solutions including licenses and insight report for biopharma and research institutions. The company, which works to find data-driven answers in rare and neurodegenerative diseases, wants to offer increased access to its insights. To do this, it has […]

November 4, 2022 - 3 minutesmins - By Liza Laws

In Depth 3 Nov 2022

What’s on the horizon for gene editing-based therapies?

Gene editing has come far in the past decade. However, the field is still finding its feet regarding challenges such as drug delivery, regulation and competition with first-generation gene therapies. Gene editing is a sizzling hot topic in the biotech industry, powered by advances in genetic tools such as CRISPR, TALENs and zinc-finger nucleases. Gene […]

November 3, 2022 - 8 minutesmins - By Jonathan Smith

News and Trends 3 Nov 2022

Servier and OSE Immunotherapeutics complete enrollment in Sjögren’s syndrome trial

Servier and French biotech company OSE Immunotherapeutics SA have completed patient enrollment in a phase 2a clinical trial evaluating the efficacy and safety of monoclonal antibody OSE-127/S95011 in primary Sjögren’s syndrome. The international, randomized, double-blind, placebo-controlled, phase 2a study is designed to evaluate the efficacy and tolerance of the monoclonal antibody OSE-127/S95011 in primary Sjögren’s […]

November 3, 2022 - 2 minutesmins - By Jim Cornall

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

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Rare disease

InfanDx tackles early detection of hypoxic ischemic encephalopathy

A biotech boom manifesting real results in rare pediatric diseases

Centogene launches rare and neurodegenerative disease Biodata Network

What’s on the horizon for gene editing-based therapies?

Servier and OSE Immunotherapeutics complete enrollment in Sjögren’s syndrome trial

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

Subscribe to our newsletter to get the latest biotech news!

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

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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