La Jolla

La Jolla Pharmaceutical Company has initiated a phase II clinical study of LJPC-401 in patients with transfusion-dependent beta thalassaemia [ Identifier: NCT03381833]. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin, the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in the heart and other vital organs, where it can cause potentially fatal complications. La Jolla is developing LJPC-401 for the potential treatment of iron overload, with promising results reported in preclinical and phase I clinical studies. The phase II clinical study is a multinational, multicentre, randomized, controlled study designed to involve 100 patients from 40 sites across 9 countries. The primary outcome measure in this study is the effect of LJPC-401 on myocardial iron overload in patients receiving chelation therapy. Secondary outcome measures include the effect of LJPC-401 on blood iron levels, on hematology laboratory parameters, and on iron laboratory parameters.

The first clinical trial site for the phase II study of LJPC-401 has now opened in San Diego, CA. Additional trial sites will open in the U.S. in the near future.

Eligibility criteria include:

  1. 18 years of age or older with transfusion-dependent beta thalassemia.
  2. Increased iron blood levels as measured by transferrin saturation (TSAT).
  3. Increased iron levels in the heart as measured by MRI.
  4. Received iron chelation therapy for a minimum of 1 year and be on a stable dose prior to enrollment in the study.


More information: La Jolla announcementCAF NewsLJPC , Clinical trial


GG2020 logo

Τhe HVP Global Globin 2020 Challenge is organising a fringe meeting at the 14th International Conference on Thalassaemia and Other Haemoglobinopathies. The meeting, entitled "Towards Comprehensive Global Epidemiology and Prevention of Haemoglobinopathies", will be held on 19th November 2017 at the Grand Hotel Palace – Ilida Meeting Room, Thessaloniki, Greece, between 14:00 and 16:30. 

The GG2020 Challenge  seeks to apply recent developments in human genomics, including the systematic collection and sharing of variation data, to fighting haemoglobinopathies (notably thalassaemias and sickle cell disease) in low- and middle-income countries. The GG2020 meeting, a great opportunity to forge new collaborations, will highlight the main goals and partnerships (including with WHO and UNESCO) of the Challenge and will provide an update on past achievements, ongoing projects and future plans. 

More information: Programme



The Cooley's Anemia Foundation (CAF) sponsors incentive awards for thalassaemia patients to further their education and career goals and to help them live positively with thalassaemia. Applicants must be U.S. residents who are currently pursuing education in a graduate, undergraduate, associate, certificate or vocational level.

The incentive awards are given as follows:

·$2,000 for Doctorate programmes with the possibility of receiving two awards during the course of study.

·$1,500 for Master degree programmes with the possibility of receiving two awards during the course of study.

·$1,000 for Baccalaureate degrees with the possibility of receiving four awards during the course of study.

·$750 for an Associates degree, limited to two awards per applicant.

·$500 for certificate programmes lasting for one year or longer, limited to two awards per applicant.

·$250 for six-month certificate programmes or vocational training programmes, limited to two awards per applicant.

The application deadline is February 15, 2018.


More information: CAF announcement 



ITHANET logo trans300bioRxiv logo


We are pleased to inform you that a preprint describing all sections of the ITHANET portal is now available online on bioRxiv (doi: This is the first article resulting from our partnership with the HVP Global Globin 2020 Challenge. Most importantly, we would like to thank everyone who supported the ITHANET portal, contributed data and provided suggestions for its improvement. 


Haemoglobinopathies are the commonest monogenic diseases, with millions of carriers and patients worldwide. Online resources for haemoglobinopathies are largely divided into specialised sites catering for patients, researchers and clinicians separately. However, the severity, ubiquity and surprising genetic complexity of the haemoglobinopathies call for an integrated website to serve as a free and comprehensive repository and tool for patients, scientists and health professionals alike. This paper presents the ITHANET community portal, an expanding resource for clinicians and researchers dealing with haemoglobinopathies. It integrates information on news, events, publications, clinical trials and haemoglobinopathy-related organisations and experts and, most importantly, databases of variations, epidemiology and diagnostic and clinical data. Specifically, ITHANET provides annotation for 2690 haemoglobinopathy-related variations, epidemiological data for more than 180 countries and information for more than 600 HPLC diagnostic reports. The ITHANET portal accepts and incorporates contributions to its content by local experts from any country in the world and is freely available for the public at


The Cooley’s Anemia Foundation is accepting applications for medical research grants and fellowships in areas related to thalassaemia. The awards are in 3 categories:

1. Support for Ongoing Clinical Research in Thalassaemia (Deadline: January 15, 2018 for a letter of intent and February 5, 2018 for invited full applications).

The goal of this initiative is to support investigators from all disciplines and backgrounds (MD, RN, PhD, MPH, MSW or other disciplines) with their ongoing clinical projects to address one or more of the following areas impacting patients with thalassaemia, including but not limited to: cardiac issues and iron overload; fertility, pregnancy and family planning; quality of life, psycho-social impact and/or burden of disease. Funding: $40,000 per year.

2. Clinical Trials in Thalassaemia Cell and Gene Therapy (Deadline: February 5, 2018).

This initiative aims to support clinicians and researchers involved with clinical applications of cell and gene therapy as to advance a cure for thalassaemia. Both phase I (safety) and phase II (efficacy) trials are eligible for support. Funding: $60,000 per year.

3. Research Fellowships (Deadline: February 5, 2018).

This initiative accepts applications from junior faculty and postdoctoral fellows involved with basic and translational research towards the understanding and/or treatment of thalassaemia and/or a complication that is related to thalassaemia. The areas of interest include, but are not limited to, studies of globin gene regulation, globin gene transfer and expression, fetal haemoglobin production, haematopoietic stem cell research, bone marrow transplantation, iron chelation and iron overload, endocrine and cardiac disorders in thalassaemia, and transfusion therapy and its complications.

More information: CAF announcement