INDUSTRIAL IMPACT, MEDICAL
prnewswire | April 28, 2023
Hansa Biopharma AB, "Hansa" a pioneer in enzyme technology for rare immunological conditions, and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, announced they have entered a research and development collaboration.
The collaboration will, in a clinical study, evaluate the safety and efficacy of Hansa's antibody cleaving enzyme imlifidase as a pre-treatment prior to the administration of Genethon's gene therapy product candidate GNT-0003 in Crigler-Najjar syndrome in patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus serotype 8 (AAV8). The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.
Søren Tulstrup, President and CEO, Hansa Biopharma stated: "Genethon is a pioneer at the cutting-edge of research and development of gene therapies for rare diseases and we are thrilled to be collaborating with them. This research collaboration further validates Hansa's commitment in gene therapy and underscores the important role that our antibody-cleaving enzyme technology can play in ensuring that even more patients can benefit from life-saving gene therapies".
GNT-0003 is currently being evaluated in a pivotal clinical study in France, Italy, and the Netherlands and has received PRIME (PRIority MEdicines) status from the EMA. Through the collaboration announced today, patients with Crigler-Najjar and pre-formed antibodies to AAV8 will be enrolled in a study with similar design where imlifidase is evaluated as a pre-treatment to enable gene therapy treatment with GNT-0003. The outcome of the ongoing clinical study of GNT-0003 could potentially form the basis for a MAA or BLA application in Europe or the US.
Crigler-Najjar is a genetic disease-causing bilirubin accumulation which leads to irreversible neurological damage manifested as muscle weakness, lethargy, deafness, mental retardation, and eye movement paralysis. Crigler-Najjar syndrome is an ultra-rare disease affecting less than one case per one million people per year.1
Frédéric Revah, CEO, Genethon added: "Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy. This collaboration with Hansa Biopharma is thus an important next step in the development of our gene therapy treatment for Crigler-Najjar syndrome, Hansa Biopharma's proven enzyme technology coupled with its scientific expertise will help us advance the critical research we are conducting in Crigler-Najjar and could enable gene therapy treatment for patients who are today not eligible because of their immunological status".
About Hansa Biopharma
Hansa Biopharma is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. Hansa has developed a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa has a rich and expanding research and development program based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The Company is listed on Nasdaq Stockholm under the ticker HNSA.
Premier Research | March 14, 2023
Premier Research has partnered with InSilicoTrials, a leader in using artificial intelligence (AI) and computational modeling and simulation (CM&S) to speed up the development of new therapies and medical devices. Premier Research helps biotech and device companies take their ideas from the idea stage to the commercialization stage. The aim of the partnership is to make it easier, faster, and safer for treatments for rare diseases to get approval from the government.
CM&S and AI have made it possible to use virtual patients in clinical trials in whole or in part, which the FDA approves of in some cases. In silico trials make it possible to create fake control or treatment arms, help figure out how to sign up patients, and predict the safety and effectiveness of new drugs and medical devices more accurately.
The use of synthetic control arms generated using in silico CM&S techniques represents a powerful tool for enabling non-feasible rare disease research, both preclinical and clinical research. In the last five years, Premier Research has been in charge of more than 240 rare disease studies for a wide range of indications. This shows that the company is committed to orphan drug development for a long time. Premier Research is continuing to put money into trials for rare diseases by partnering with InSilicoTrials.
One major advantage of in silico simulation is the ability to investigate 'what if' scenarios, particularly in rare diseases where a larger patient population does not exist. By using biological, molecular, or genetic data to describe rare diseases, in silico models can make synthetic control arms. This, along with Premier Research's expertise in clinical and preclinical development, execution, and regulatory aspects of small population trials, can shorten the time it takes to make effective treatments for rare diseases.
The partnership between Premier Research and InSilicoTrials will focus on smarter in silico study design, leading to a more effective pre-clinical review of trial design parameters and faster submission-ready studies. CEO of InSilicoTrials Luca Emili says that combining modeling and simulation with AI is the best way to improve the R&D process in drug development. This is because it allows sponsors to speed up the development of new treatments, make medical products safer, and lower R&D costs.
About Premier Research
Premier Research is a global company that does clinical research, product development, and consulting. It focuses on using new technologies to design and run smart studies and trials. With more than 2,500 employees in 75 countries, it has a lot of experience in certain therapeutic areas and uses. The company is committed to empowering its customers to meet the unmet needs of patients and offers great benefits and flexible working conditions.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
The Center for Breakthrough Medicines | March 13, 2023
Nucleus Biologics and Stoic Bio have teamed up to form a supply agreement with the Center for Breakthrough Medicines (CBM), a contract development and manufacturing organization that aims to accelerate the development and manufacture of advanced therapies. Under the agreement, Nucleus Biologics will become CBM's selected supplier of cell culture media and other biological solutions, ensuring a consistent supply of this critical material with tighter quality control measures.
Nucleus Biologics' approach to cell culture media, which sees it as a critical raw material that can drive desired attributes of cells crucial to the success of cell and gene therapy research, is complementary to CBM's focus on transparency, technology, and end-to-end solutions. By working together, they hope to expedite cell and gene therapy breakthroughs.
In addition, CBM will be an early adopter of Stoic Bio's innovative family of point-of-use media makers, Krakatoa™, which allows scientists to manufacture their own media with 65% fewer CO2 emissions than conventional media. By demonstrating that developing and manufacturing lifesaving therapies doesn't have to come with a high environmental cost, CBM hopes to set an example for the industry.
With over 2,200 cell and gene therapies in development, collaborations like this one are needed to speed up the process of getting these therapies to the clinic and ultimately approved. The CBM, Stoic Bio and Nucleus Biologics collaboration aim to create a unique service offering that introduces a paradigm shift in development and manufacturing, showing how technology and sustainability can converge to make better therapies sustainably.
About The Center for Breakthrough Medicines
The Center for Breakthrough Medicines (CBM) is a CDMO focused on accelerating the development and manufacture of advanced therapies to save lives. It offers end-to-end solutions such as process development, manufacturing, and quality control for cell and gene therapies, gene editing, and immunotherapies. In addition, CBM collaborates with pharmaceutical companies, academic institutions, and healthcare providers to make advanced therapies more accessible while ensuring compliance with regulatory standards. The company also prioritizes sustainability by implementing green initiatives and sustainable technology and strives to positively impact the communities it operates in.