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Nohla Therapeutics bags $56 million in Series B financing to fund cell therapy platform

In News, News by editor

Nohla Therapuetics, a Seattle based developer of universal, off-the-shelf cell therapies for patients with hematologic malignancies and other critical diseases, have announced that an additional $11 million has been raised after the second closing of its Series B financing, bringing the total raised to $56 million. The funding will support the ongoing clinical development of dilanubicel, the company’s lead cell therapy product.

The investors who participated in the second closing include the University of Tokyo Edge Capital, Schroder Adveq and Premier Partners, joining the initial Series B investors made up of Fidelity Management and Research Company, Celgene Corporation, ARCH Venture Partners, 5AM Ventures, Alexandria Venture Investments and AML Biotech Partners.

The additional funding will financially support Nohla’s ongoing clinical development of dilanubicel, a universal, off-the-shelf stem and progenitor cell therapy designed to provide patients with short-term bone marrow function, while also providing long term immunologic benefits with the potential for improved survival. Unlike autologous or patient-specific allogeneic cell therapies, dilanubicel does not require Human Leukocyte Antigen (HLA) tissue matching. Last month, Nohla received Priority Medicine (PRIME) status for Haematological malignancies in the European Union for the product. Dilanubicel is currently under evaluation in two ongoing Phase II trials for patients with hematologic malignancies undergoing an allogeneic transplant, and acute myeloid leukemia (AML) patients with chemotherapy-induced myelosuppression following high-dose chemotherapy. Results from the study will be published later this year.

Katie Fanning, President and CEO of Nohla Therapeutics commented “We have achieved a number of significant milestones in the first half of 2018 including further advancement of our lead product, dilanubicel. This additional funding will support our ongoing clinical development of dilanubicel in allogeneic transplant and acute myeloid leukemia, as well as support the advancement of discovery programs from our platform.”

Source: Nohla Therapeutics Announces Second Closing of $56 Million Series B Financing Press Release

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Pluristem and Thermo Fisher to collaborate on cell therapy industrialization

In News, News, Uncategorised by editor

Pluristem Therapeutics, a developer of placenta-based cell therapy products, has entered into a strategic collaboration with Thermo Fisher Scientific with the aim to advance fundamental knowledge of cell therapy industrialization and to improve quality control across the end-to-end supply chain.

This agreement will combine Pluristem’s cell therapy manufacturing, clinical development and quality control expertise with Thermo Fisher’s cell therapy development and bioproduction scaleup experience. The goal of the collaboration is to enable mass production of millions in therapeutic dose of regenerative medicines through the transformation of cell therapy manufacturing into a large scale, high capacity industry. At present there are over 850 companies working on the development of regenerative medicines and advanced therapies, and approximately 900 clinical trials being conducted, with several approved and marketed products worldwide. In order to address unmet medical needs, change is required in the supply chain of equipment, consumables, reagents and storage systems.

“From early on, our strategy was to build an in-house manufacturing facility. Having achieved that, we are now in a unique position within the industry, and have a broad understanding of its needs. Thermo Fisher, known for its biopharmaceutical infrastructure, materials supply, logistics, and manufacturing expertise is a perfect partner for Pluristem. This initiative has the potential to advance the industry significantly, allowing regenerative medicine to realize its potential, treating millions of patients globally while reducing healthcare spending. We look forward to a long-term, mutually beneficial partnership,” commented Pluristem President and Co-CEO Yaky Yanay.

Source: Pluristem Signs Collaboration Agreement With Thermo Fisher Scientific
Press Release

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Axovant Announces Global Licensing Agreement for Oculopharyngeal Muscular Dystrophy Gene Therapy

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Axovant Sciences have announced an agreement giving them the exclusive global rights to ACO-AAV-OPMD, part of the investigational ‘Silence-and-Replace’ gene therapy program for the treatment of oculopharyngeal muscular dystrophy (OPMD) from Benitec Biopharma.

Silence-and-Replace gene therapy technology suppresses mutant protein production while restoring expression of functional protein through the delivery of a combination of DNA-directed RNA interference (silence) along with a functional copy of the gene (replace) in a single vector construct. This method can be applied to various genetic diseases including autosomal dominant disorders caused by nucleotide repeat expansion.

AXO-AAV-OPMD is an investigational gene therapy that has been granted Orphan Drug Designation by the FDA and European Commission, and is currently in preclinical development, with a planned placebo-controlled clinical study due to begin in 2019. There are currently no approved products for the treatment of OPMD, which is caused by mutations in the gene coding for polyA-binding protein nuclear 1 (PABPN1), which can lead to formation of intranuclear inclusion bodies causing muscle cell pathology.. This investigational therapy is given as a single administration directly into target muscle tissue, utilizing an AAV vector to silence the mutant PABPN1 gene which causes OPMD and replacing it with a functional copy. Data from mouse models of OPMD showed AXO-AAV-OPMD provided up to 86% inhibition of PABPN1 expression, while restoring functional PABPN1 up to 63% of normal levels.

Axovant will pay Benitec an upfront payment of $10 million for rights to the AXO-AAV-OPMD program and five additional investigational gene therapy products, as well as payments tied to development, regulatory and commercial sales milestones. Benitec will also receive 30% of the net profits on worldwide sales of AXO-AAV-OPMD and tiered royalties on the other gene therapy products that result from this collaboration. “The Silence-and-Replace technology is a unique approach in gene therapy, using a single vector to suppress mutant protein production while also restoring expression of the functional protein, and could be an elegant solution to tackling autosomal dominant genetic disorders,” commented Fraser Wright, PhD, Chief Technology Officer of Axovant.

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Cold Chain Logistics – what have we learnt from Pharma?

In News, Spotlight by Elisa Manzotti

Date: October 2015

There are many lessons to be learned from the tortuous path taken by pharma and biopharma over the past 20 years. Many of these challenges and solutions are unknown in cell therapy as the majority of products have not reached the point of commercialization. However, it is a consideration that must be addressed long before Phase III clinical trials.


Kevin O’Donnell, BioLife Solutions, USA guides us through the key challenges faced in biologistics and discusses what lessons the cell & gene therapy sector can take from Pharma’s experience.

You will also have the opportunity to hear from and pose your questions to a panel of industry experts with our webinar program – featuring live presentations from Big Pharma and Biotech, sharing their experience and often creative solutions to optimise product delivery.

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Gene Editing and Potential Applications in Cell & Gene Therapy

In News, Spotlight by Elisa Manzotti

Date: December 2015

The rapidly advancing developments in gene editing have numerous implications and applications for the cell and gene therapy field – from disease modelling through to the potential editing of patient-derived cells to modify/correct disorders.

The Gene Editing spotlight features a lead review from Prof. Andrew Bassett, Oxford University, UK discussing the main gene-editing technologies in detail: Zinc Fingers, TALENs and CRISPR-Cas9, comparing mechanisms of action, limitations and opportunities for future development and applications.Gene Editing Spotlight

Building upon this, we present a series of Expert Insight articles discussing potential clinical applications in more detail including:
– Cystic fibrosis
– Duchenne Muscular Dystrophy
– Genetic blood disorders and hemoglobinopathies

Alongside the discussion of the incredible advances in gene editing applications, an ongoing patent dispute plays out in the media over the CRISPR-Cas9 technology. Dr Beatriz San Martin, Partner, FieldFisher, UK helps demystify and explain the IP situation and talks about the possible implications of the outcome of this dispute.

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The Cell & Gene Therapy Manufacturing Pathway

In News, Spotlight by Elisa Manzotti

Date: From January 2016

The sector has recently seen an influx of investment and interest from Big Pharma and VCs, in part due to the promising early clinical data from immunotherapies such as CAR T cells and engineered T cells. However, many questions and challenges remain on the path to successfully translating a cell-based therapy into a commercially viable product.

Our expert Guest Editors – Dr Peter Zandstra, University of Toronto; Dr Stephen Ward, Cell Therapy Catapult; and Dr Greg Russotti, Celgene Cellular Therapeutics – have identified the critical issues along the manufacturing pathway and these will be discussed across a 4-part Spotlight Series:

1. Commercial Attributes to Fit Reimbursement RealityCell therapy manufacturing
2. Integration of Manufacturing and Delivery into Healthcare Systems
3. New Approaches to Process Validation and Supply
4. Advances in Analytical Toolkit Development

This Series will help inform the essential discussion and debate concerning the issues at the heart of successful commercialisation – learning from success stories and failures, hearing from experts across industry, government and academic translation centers and helping identify the attributes required to achieve commercial success.