SCIENCE THROUGH NETWORKING

News

In this section, is reported the full ATMP news database, sorted by area of interest

Replimune Group, a biotechnology company developing oncolytic immuno-gene therapies, has announced that the Phase 2 portion of the Company’s Phase 1/2 clinical trial of RP1 alone and in combination with nivolumab anti-PD1 therapy has initiated. In the Phase 2 portion of the clinical trial, RP1 in combination with nivolumab will be tested in four 30-patient cohorts of patients with melanoma, non-melanoma skin cancer (NMSC), metastatic bladder cancer or microsatellite instability high (MSI-H) tumors

Date: Jun 1, 2019

Kite, a Gilead Company, and Humanigen have announced the formation of a clinical collaboration to evaluate the combination between Yescarta, the Kite-Gilead CAR-T, and lenzilumab in relapsed/refractory diffuse large B cell lymphoma patients, with the aim to assess the effect of the monoclonal antibody on the safety of Yescarta. Toxicities associated with CAR T therapy include neurologic toxicity and cytokine release syndrome; pre-clinical evidence suggests that lenzilumab inhibition of GM-CSF may have the potential to disrupt CAR T cell mediated inflammation without disrupting CAR T cell anti-tumor efficacy. Kite will act as the sponsor of this study and will be responsible for its conduct

Date: May 31, 2019

A study of the Fred Hutchinson Cancer Research Center, published on Nature Materials, shows positive results about the use of gold nanoparticles like a CRISPR gene editing system’s carrier into the blood stem cells. The gene therapy technologies are inaccessible to millions of people, and the development of simpler systems for the gene editing key components’ delivery has therefore proved necessary. The gold nanoparticles were engineered to quickly cross the cell membrane, dodge cell organelles that seek to destroy them and go right to the cell nucleus to edit genes

Date: May 30, 2019

Starts in Padua Road map CAR-T, a series of regional conferences in order to create a communication link between medical system, politicians and stakeholders, to enable different Italian Regional Health Services to make the best operative decisions which will make the system efficient and sustainable, and at the same time to ensure a rapid patient access to the treatment. The CAR-T therapies should be read as complex and expensive treatment pathways, that require a careful services system organization, necessary to avoid the system’s crisis

Date: May 30, 2019

Cell therapies represent the largest number of active agents in development in the immuno-oncology field overall. The United States and China hold the leading positions in the global cancer cell therapy pipeline, although the majority of agents being developed in the United States are at the preclinical stage whereas the majority of agents being developed in China are in clinical stages. Using ClinicalTrials.gov data, it is possible to identify more than 1200 clinical trials with a substantial growth of new trials each year in the past decade, largely owing to the explosion of trials for CAR-T therapies. It is also interesting to note that while solid tumors are the more common, only around half of all cell therapy trials from 1993  have targeted solid tumours and ,in addition, the majority of cell therapy trials for these tumors focus on a handful of cancer types

Date: May 30, 2019

Fibrocell Science announced that the FDA has granted Regenerative Medicine Advanced Therapy designation to its gene therapy candidate, FCX-007, in development for the treatment of recessive dystrophic epidermolysis bullosa. This designation is granted to speed up the development and review of gene therapies that target serious or life-threatening conditions. To this gene therapy FDA has already granted Orphan, Rare Pediatric Disease and Fast Track designations. Currently there are no approved treatments for RDEB, so FCX-007 can be a potentially transformative treatment option for the given skin disorder if it wins approval

Date: May 30, 2019

A group of Stanford University and Tokyo University researchers claims it found a method for an efficient hematopoietic stem cells cultivation. The study, published on Nature, shows a set of conditions that enable this cells to expand themselves 900 times in one month. It result also possibile, during the cultivation, to use CRISPR technology to correct genetic defects in the original stem cells; the corrected cells can then be expanded for the transplant and this should allow to use a patient's own cells as gene therapy

Date: May 30, 2019

BioMarin Pharmaceutical has announced the updated data from a phase I/II clinical trial about its gene therapy valoctocogene roxaparvovec (Valrox) for hemophilia A that show, three years after drug administration, continuity in bleeds control but a factor VIII level decrease in data collected two years after administration. FDA has granted to the drug the Breakthrough Therapy Designation as a revolutionary therapy, and EMA has granted it PRIME designation giving a prioritary status in the UE. Also both Authorities have granted the orphan drug designation for the severe hemophilia A treatment

Date: May 29, 2019

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania has announced an expansion to their collaboration with rights to pursue collaborative research and development of novel gene therapies for lysosomal disorders and other rare diseases. This expanded relationship will continue to combine Amicus’ protein engineering and glycobiology expertise with Penn’s gene transfer technologies to develop novel gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability

Date: May 29, 2019

Orchard Therapeutics, Telethon and San Raffaele Hospital have announced that Orchard has been granted an exclusive license to intellectual property rights to research, develop, manufacture and commercialize the autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of Mucopolysaccharidosis Type I developed by the San Raffaele-Telethon Institute for Gene Therapy. The Company believes that the use of this innovative therapy in MPS-I has the potential to fundamentally change the lives of patients born with this devastating and rapidly progressive condition

Date: May 28, 2019

Published on the Official Journal the AIFA 2018 notice for independent researches carried out on specific themes, identified by the Agency, i.e. rare diseases, CAR-T and controlled comparative clinical studies. AIFA considers support for independent research an investment to build knowledge on effectiveness, safety and proper use of drugs, especially in areas that are out of the interest of for profit research.
Each project can collect a financing up to  €1,5 million for interventional studies, €500.000 for observational studies and €75.000 for meta-analysis

Date: May 28, 2019

FDA has approved Zolgensma, Novartis gene therapy against spinal muscular atrophy type 1, with a price of $2,125 million per patient. The treatment is the most expensive in history but, according to the swiss Company, the lifesaving benefit justifies the price, although a change in payment and reimbursement methods, in a system without lump sum payment capabilities, wil be needed. FDA uses a clinical trial data for its decision, that shows all of 15 infants treated with the drug were alive and without respiratory assistance at two years old

Date: May 27, 2019

The European Patent Office (EPO) grants the protection until 2035 for an innovative component, a “spacing structure” of CAR protein, already used in company’s CAR-T in development for different tumors. This “spacer” derives from the Low-affinity Nerve Growth Factor Receptor (LNGFR) allows to select the Car-expressing cells without the marker-gene inclusion, in-vivo cell tracking and to block the recognition by the innate immune system of Car, with an increased survival compared to Car with other spacers

Date: May 24, 2019

A study of Rutgers University, published on Cell Stem Cell, shows the potential of Olig2 gene inhibition in a possible Down syndrome gene therapy. Using the stem cells, the researchers have developed two experimental models of brain focusing on Olig2 gene, located on chromosome 21; the results suggest that this gene may be an excellent target for a prenatal gene therapy, able to prevent the abnormal brain development and to promote postnatal cognitive developement

Date: May 24, 2019

The study, developed by researchers of the Telethon Institute of Genetics and Medicine of Pozzuoli, has shown a way to solve a viral vectors’ limit, that is the ability to transport small amount of genetic material. The scientists have developed coding viral vectors for small portion of large proteins, then reassembled to complete proteins with a cyanobacteria-inspired process. The hereditary blindness diseases affect over 200.000 people in Europe and it is caused by altered gene expression for photoreceptors’ proteins

Date: May 24, 2019

Avrobio, a clinical stage company focused on developing lentiviral-based gene therapies, presented updated preclinical data for AVR-RD-02, its gene therapy candidate for Gaucher disease, and has announced its plans to begin the first clinical studies in patients with type 1 disease in the second half of 2019. Gaucher disease is a rare, inherited metabolic disorder caused by mutations in the GBA gene, coding for beta-glucocerebrosidase, an enzyme needed for a metabolic process of the lipid molecule glucocerebroside

Date: May 23, 2019

UniQure, a gene therapy focused biotech company, has announced the attainment of two patents that strengthen its intellectual property on the investigational candidate AMT-130, ahead of the clinical studies start, expected within the year. The therapy, focused on the gene silencing of mutant huntingtin, may be very successful for Huntington Disease, a condition without treatment so far

Date: May 23, 2019

Zolgensma, Novartis one-time gene therapy for the treatment of spinal muscular atrophy, is waiting the FDA market authorization within the month. The swiss company’s CEO said that the price for the gene therapy will be far lower than the rumors of $4-5 millions. The current treatment for SMA is Spinraza, a Biogen product, with a list price of $750000 for the initial year, and $375000 annually thereafter

Date: May 22, 2019

A study of San Raffaele Telethon Institute for Gene Therapy, published on Science Translational Medicine, shows the potential of integration of the protein CD47 into the surface of viral vectors, like Lentivirus, to promote gene therapy activity by increasing the vector’s retention in the body. The protein CD47 is known to inhibit the splenic and hepatic phagocytes, and the performed studies on monkeys with CD-47-Lentivirus have shown increased resistance against phagocytosis and less susceptibility to detection by Antigen-Presenting Cells than normal Lentivirus

Date: May 22, 2019

The american company, leader in gene and cell therapy, has announced the acceptance of investigational new drug application by FDA for ABO-202, gene therapy for the treatment of CLN1, rare neurodegenerative disease. The therapy is  is designed to deliver a functional copy of the PPT1 gene, critical enzyme for proper lysosomal function, to cells of the central nervous system and peripheral organs with a viral vector, using a combined intravenous and intrathecal administration. The Company will provide guidance on the timing of the clinical trial later this year

Date: May 22, 2019
Page 10 of 32

With the unconditional support of

 

Celgene logo P280
galta ris
novartis logo pos cmyk c

 

Sanofi genzymePNG
Takeda Symbol Color pos

We use cookies, just to track visits to our website, we store no personal details