Healthcare industry

Review - 09/02/2023

5th Gene Technology Report – a critical observation of a cutting-edge technology

The societal importance of genetic technologies was demonstrated during the coronavirus pandemic, when it was possible to rapidly develop suitable vaccines thanks to genetic engineering methods. As a result, the Fifth Gene Technology Report published in autumn 2021 reads like a validation of many years of work, as well as making it clear that the will to continue the detailed long-term monitoring is very much present.

With its "Proof of Concept" grants, the European Research Council ERC supports scientists in further developing the commercial potential of their research results. Nina Papavasiliou from the DKFZ is now receiving the prestigious grant for the second time: she wants to advance the development of a "molecular delivery service" that ensures that therapeutic genes reach the right address in the body in a targeted manner.

Today, genome editing is almost as easy as programming software. However, the generation of viral vectors as initial material is still associated with many expensive and error-prone handling procedures. Viruses are generated via complex biological processes that have to be optimised virus-specifically in order to produce high-quality therapeutics. A new method is needed that simplifies and optimises these processes.

AaviGen GmbH: Hope for heart failure - 14/07/2022

Gene therapy for weakened hearts

Treating a weak heart – for example after a heart attack – at the GP’s surgery with a single intravenous injection, without causing serious side effects: what sounds almost too good to be true is actually already under development. The Heidelberg-based biotech company AaviGen is working on a platform technology based on adeno-associated viruses that can help introduce therapeutic genes in a highly specific way into diseased heart muscle cells.

The nuclease Cas13b associated with the CRISPR gene scissors, which is an enzyme that degrades nucleic acids, has the potential to be used in the future in hereditary diseases to switch off unwanted genes. In the fight against infections, this nuclease is also being researched as an antiviral agent, as Cas13b can specifically intervene in the genetic material of viruses and render them harmless.

SolidCAR-T project - 15/03/2022

Modular ‘mini-factories’ for decentralised production of CAR T cells

Novel CAR T-cell therapies have proved to be promising therapeutic options for the treatment of acute leukaemias and lymphomas. Researchers from the Fraunhofer IPA in Stuttgart, the University Hospital Tübingen and the NMI in Reutlingen have joined forces in the SolidCAR-T project that aims to generate CAR T cells to combat solid tumours and produce these cells directly on site in the clinic using automated 'mini-factories'.

Dossier - 16/12/2021

Advanced therapy medicinal products: gene and cell therapies

Novel gene and cell therapies for treating incurable and hereditary diseases have raised high expectations. However, success has so far been limited to the long-established bone marrow transplants involving the administration of haematopoietic stem cells used to treat blood cancer. CAR T-cell therapies have recently emerged as a major new hope in cancer treatment.

Article - 29/01/2019

Targeted RNA editing with the body’s own enzyme activity

Completely new possibilities for research and gene therapy became available following the development of the CRISPR/Cas method for targeted modification of the genome. However, treatment with molecular scissors is not without risk as potential errors are stored in the genome forever. Scientists from Tübingen have developed an alternative method in which the intervention takes place at the RNA level using the body's own enzymes and is thus…

Article - 10/01/2019

Stocktaking and recommendations for action: the BBAW’s fourth gene technology report

In the new gene technology report, the interdisciplinary working group of the Berlin-Brandenburg Academy of Sciences (BBAW) takes stock of gene technology developments in Germany during the past few decades, and discusses the societal, legal and ethical challenges associated with these technologies in the future. The report is highly topical due to the controversy surrounding the ruling of the European Court of Justice on CRISPR/Cas9 genome…

Expert interview - 17/10/2018

Biologicals are becoming increasingly important to Boehringer Ingelheim

Biopharmaceuticals are playing an increasingly important role in Boehringer Ingelheim’s development pipeline. Visible evidence of this is the company’s Biologicals Development Centre (BDC) that is currently being constructed in Boehringer Ingelheim’s Biberach plant and will bring the company’s process development under one roof by early 2020. Walter Pytlik from BIOPRO talked with Dr. Ing. Ralf Schumacher about the new centre.

Dossier - 28/08/2018

With molecular diagnostics to biomarker-based personalised therapy

Diagnosing suitable biomarkers is a prerequisite for tailoring personalised therapies to patient heterogeneity. Genetic tests and genome sequencing play a key role in these diagnoses. Up until now, personalised therapy has achieved the greatest success in the field of oncology. However, personalised treatments are also gaining in importance for treating other diseases.

Expert interview - 08/08/2018

Ludolph: diagnosing and treating neurodegenerative disorders

Ulm has long been a world leader in diagnosing and treating rare neurological disorders, notably amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and Huntington's disease (HD). We spoke with Professor Albert C. Ludolph, spokesperson for the Ulm DZNE site, medical director of the Clinic for Neurology at the RKU (University and Rehabilitation Clinics of Ulm) and world-renowned ALS researcher.

Expert interview - 26/06/2018

Genome analyses: "Germany lags behind dramatically"

Around 6,000 genetic diseases can be diagnosed using genetic tests. Genetic testing enables the accurate identification of diseases especially when symptoms are unclear, and also allows statements to be made about disease progression. However, restrictive regulations considerably hinder the use of genetic diagnostics. BIOPRO spoke with Dr. Dr. Saskia Biskup, a human genetics specialist and co-founder of the Tübingen-based company CeGaT.

Article - 19/03/2018

Regenerative medicine: curing rather than simply treating diseases

Some scientists refer to the latest developments in the field of regenerative medicine as the "next revolution in medicine". With the help of gene therapies or stem cells, regenerative medicine aims not only to treat disease symptoms, but to cure them at source. Some approaches are already being used to treat patients and several others are close to application.

Article - 26/10/2016

GeneWerk: precision analyses for humans

Gene therapy approaches are increasingly being used for treating life-threatening diseases in humans. GeneWerk GmbH, a spin-off of the DKFZ and the NCT in Heidelberg, offers customised, high-resolution molecular and bioinformatic analyses that ensure the efficacy and safety of gene therapy and immunotherapy studies.

Article - 08/08/2016

First achromatopsia gene therapy clinical trial in Germany is going well

Around 3000 people suffer from achromatopsia in Germany. Achromatopsia is an inherited visual disorder characterised by the absence of full colour vision. The disease is caused by a genetic defect that makes the retina's cone photoreceptors, needed for daylight and colour vision, non-functional. There is currently no cure for achromatopsia. Scientists from Tübingen University Hospital and their colleagues from Munich and New York have now…

Article - 19/07/2016

Licence to kill – the enormous potential of CAR T cells

With 6 million euros of EU funding, the CARAT project aims to optimise a technology called CAR T that is used to equip T cells with antibody fragments and specifically direct them to destroy cancer cells. The CARAT consortium comprises a multinational team of experts from the Institute for Cell- and Gene Therapy at the Freiburg University Medical Center led by Prof. Dr. Toni Cathomen and seven partner institutions. Cathomen’s team is developing…

Dossier - 14/06/2016

CRISPR/Cas – genome editing is becoming increasingly popular

The number of publications and patents that involve the CRISPR/Cas system has been increasing exponentially since the technique was first described a few years ago. The increase in funding for projects involving CRISPR/Cas also demonstrates how powerful this new method is. The targeted modification of genomes (also called gene editing or genome editing) using CRISPR/Cas is extraordinarily accurate and also has the potential to cure hereditary…

Article - 07/10/2015

Call for a moratorium on germ line experiments in humans

Modern genetic engineering tools enable genes to be modified in a genomic context in living cells. Genome surgery unlocks enormous potential for the treatment of genetic diseases, but it could also be misused for the uncontrolled manipulation of the human genome. An interdisciplinary working group of German scientists is calling for a moratorium on human germ line experiments to provide a space to define the opportunities and risks of this new…

Dossier - 10/11/2014

Cell and gene therapies: from bench to bedside

While cell therapy has become standard treatment for a number of blood cancers, most cell and gene therapy approaches for the treatment of hereditary and metabolic diseases, neurodegenerative disorders and cancer are still in the experimental phases or early clinical trials. However, recent successes give rise to the hope that cell and gene therapies will in future make important contributions to previously incurable diseases.

Article - 20/10/2014

First ever eye gene therapy close to clinical testing

There are many different retinal diseases simply because many different steps of the visual process can be affected. However, they all have one thing in common: correction of the relevant defective gene currently provides the only possibility of permanent cure. Prof. Dr. Mathias Seeliger and his group of researchers at the Institute of Ophthalmic Research at the University Hospital in Tübingen is specifically focused on the development of gene…

Article - 15/09/2014

Gene therapies for pulmonary disease are close to final development

Gene therapy currently offers the only chance of curing genetic diseases such as cystic fibrosis and beta thalassaemia. Gene therapy is the replacement or correction of a mutated gene with DNA that encodes a functional gene. Intensive research has been going on in this field for many years however only a handful gene therapies are available at this point in time. Dr. Michael Kormann junior professor at the Childrens Hospital at the University of…

Heidelberg start-up develops gene therapy for the treatment of cardiac insufficiency. UniQure N.V. pays 3 million euros in cash and shares for the acquisition additional milestone payments and future revenue shares were agreed upon.

Article - 18/08/2014

The use of human genes as medical products

In contrast to medications used for treating disease, gene therapy does not use chemical agents to alleviate or cure disease symptoms. Instead, it involves the introduction of a normal copy of a mutated gene to restore the function of a protein. Gene therapy could also be described as a way of restoring the body’s self-healing process. It is an extremely smart idea that enables the sustainable treatment of diseases which cannot usually be…

Article - 04/08/2014

Europe’s first gene therapy

Scientists from the National Center for Tumour Diseases (NCT) have shown that adeno-associated viruses (AAV) do not pose a cancer risk. This conclusion is based on the analysis of several million patient cells treated with AAV and the finding that the viruses do not integrate into the patient genome. AAV is the first-ever gene therapy for clinical use in the Western world. AAV vectors could also potentially be used as prototypes for the treatment…