List of questions

2222: What are the main safety challenges to overcome for developing cellular therapies for clinical use

2223: What are the main challenges other than safety which prevent reg med or cellular porducts reaching the clinic and how can SME's support this transition

2222: Alcyomics has developed useful in vitro safety assays which could predict adverse reactions to cellular products

2223: Challenges include lack of potency and efficacy assays - should SME's divert funds to this area of R&D

2237: What analytical methods are being most widely used for characterising advanced therapies, both in process and for product release? Where should improvements be made in making these more applicable to manufacturing ATMPs at commercial scale

2238: What steps in the ATMP manufacturing process are still having to be done in an open manner? Are there any materials needed for the process you would like to see reformulated to work better with current/future manufacturing methods?

2237: Bio-Techne empowers researchers in Life Sciences and Clinical Diagnostics by providing high-quality reagents, instruments, custom manufacturing, and testing services. Our family of brands creates a unique portfolio of products and services.

We have recently launched our Cell and Gene Therapy Initiative to put our expertise into creating new products and next generation technologies that will help bring novel therapies to the clinic.


2238: Bio-Techne empowers researchers in Life Sciences and Clinical Diagnostics by providing high-quality reagents, instruments, custom manufacturing, and testing services. Our family of brands creates a unique portfolio of products and services.


We have recently launched our Cell and Gene Therapy Initiative to put our expertise into creating new products and next generation technologies that will help bring novel therapies to the clinic.

Are there biologically validated GPCR drug targets that are poorly addressed by small molecule drug discovery?

We have developed a versatile and highly robust platform for discovering antagonist monoclonal antibody drugs against GPCR targets. The technology has been tested against 3 separate GPCRs from different subfamilies, delivering an unprecedented rate of functional antibodies against all three targets - for one of the GPCRs yielding the first antagonist mAbs ever described. The reason for success appears to be the platform's ability to elicit a very strong antibody response in an immunised animal, while preserving the three-dimensional fold of the target antigen GPCR.

The ability to reliably produce functional mAbs can overcome many of the problems with small molecule drug discovery such as poor selectivity, unpredictable pharmacokinetics and off-target effects. We are now seeking biologically validated GPCR drug targets to prosecute with our technology and develop mAb drugs. Validation can range from small clinical studies, to human genetic evidence, to animal models and in vitro assays.

What are the emerging tools for assessing drugs for chronic kidney disease (pre-clinical and clinical readouts)

Assessment of drugs for chronic kidney disease (pre-clinical and clinical readouts)

We are developing a mAb therapy to treat chronic kidney disease. The target of the mAb is directly involved in the integrity of the glomerular structures as well as the development of fibrosis.

A real challenge in the development of drugs to chronic kidney disease is a lack of translational research tools to allow early go/no-go program decision-making and candidate prioritisation. On the preclinical side there is a lack of widely-accepted, robust predictive disease models. On the clinical side, there are few early clinical pharmacodynamic readouts that can demonstrate drug-target engagement and desired pharmacodynamic effects in a reasonable time frame (e.g. than 12 weeks of clinical dosing).

We are keen to understand the state-of-the-art of thinking in this area, and work collaboratively to validate preclinical models and clinical biomarkers.

Inflammatory diseases driven by myeloid cells?

We have developed mAbs capable of blocking the infiltration of myeloid cells into tissues and are currently selecting disease indications for clinical testing of our mAbs. We are particularly interested to learn about diseases where at least a subset of pathology is associated with accumulation of myeloid cells/neutrophils in the affected tissue, and where patients do not satisfactorily respond to existing drugs.

What are the main challenges in using bioreactors for stem cell growth?

A number of bioreactors are available in the market to cater the growth of adherent cells, however, many labs do not adopt this system. What is the reason for it and what improvements to the bioreactor system and sector would enable the transition quicker?

What characteristics of stem cells would be affected if they are grown as suspension culture?

Conventional adherent culture system poses challenges for mass production of stem cells. Hence, a lot of research is focused on developing systems to grow stem cells as suspension culture. What characteristics of the cells would be affected by this and would the process be applicable for both autologous and allogenic stem cell growth?

How might we best manage and control the freezing and thawing of stem cell-derived immature neutrophils to maintain their selective cancer killing?

• LIfT BioSciences is a biotech bringing to market a first in class immuno-oncology cell therapy called N-LIfT that has the game-changing potential to destroy all solid tumours irrespective of mutation or strain. N-LIfT (Neutrophil-only Leukocyte Infusion Therapy) builds on two decades of ground-breaking research into innate cancer immunity by the renown co-founder Prof Zheng Cui. Our vision is to develop the world’s first cell bank of mass produced, cancer-killing neutrophils to deliver a portfolio of immuno-oncology cell therapies for curing all solid tumours.

• LIfT BioSciences’ N-LIfT therapy platform is an off-the-shelf allogeneic patented product that we mass-produce from exceptional stem cells from donors with exceptional immunity. LIfT BioSciences are pioneering neutrophil ATMPs, with a team of experienced industry leaders. LIfT has already proven to be 100% curative in mice and with promising human data in an FDA approved safety trial showing up to 80% tumour necrosis in a mix of late stage carcinoma patients even at low dose.

• Our vision is to develop the world’s first cell bank of ‘cancer killing neutrophils’ to deliver a portfolio of immuno-oncology cell therapies for all solid tumours, with an initial focus on high unmet need orphan tumours such as those found in the lung, liver and pancreas.

• We have cornerstone investors including Jonathan Milner (Meltwind & Abcam) and lead institutional investors for our forthcoming PoC trial and bioreactor scale-up. We are now seeking other institutional investors to join the syndicate. We are also seeking co-development partners and are in talks with several major pharmaceutical companies.

How might we best select-out mature CD10+ and retain immature CD10− neutrophils during the GMP scale-out/scale-up of our final neutrophil-only leukocyte infusion therapy (N-LIfT) product?

• LIfT BioSciences is a biotech bringing to market a first in class immuno-oncology cell therapy called N-LIfT that has the game-changing potential to destroy all solid tumours irrespective of mutation or strain. N-LIfT (Neutrophil-only Leukocyte Infusion Therapy) builds on two decades of ground-breaking research into innate cancer immunity by the renown co-founder Prof Zheng Cui. Our vision is to develop the world’s first cell bank of mass produced, cancer-killing neutrophils to deliver a portfolio of immuno-oncology cell therapies for curing all solid tumours.

• LIfT BioSciences’ N-LIfT therapy platform is an off-the-shelf allogeneic patented product that we mass-produce from exceptional stem cells from donors with exceptional immunity. LIfT BioSciences are pioneering neutrophil ATMPs, with a team of experienced industry leaders. LIfT has already proven to be 100% curative in mice and with promising human data in an FDA approved safety trial showing up to 80% tumour necrosis in a mix of late stage carcinoma patients even at low dose.

• Our vision is to develop the world’s first cell bank of ‘cancer killing neutrophils’ to deliver a portfolio of immuno-oncology cell therapies for all solid tumours, with an initial focus on high unmet need orphan tumours such as those found in the lung, liver and pancreas.

• We have cornerstone investors including Jonathan Milner (Meltwind & Abcam) and lead institutional investors for our forthcoming PoC trial and bioreactor scale-up. We are now seeking other institutional investors to join the syndicate. We are also seeking co-development partners and are in talks with several major pharmaceutical companies.

How far are we from printing human organs?

Current drug discovery efforts use in vitro cell culture and animal models to understand the pathology behind disease. However, their value in predicting good targets, the effectiveness of treatment options, and success rate of clinical trials remains inefficient. This may result from over simplified cell culture models and species-specific differences in biology. Thus there is an unmet need to develop a better and more complex human model system in a dish.

State-of-the-art 3D bio printing technologies have the potential to revolutionize drug discovery, toxicity testing, and tissue engineering/replacement therapy in both industry and academia. Although relatively simple tissues and structures can be printed routinely (e.g. sheets of skin or cardiac patches), printing entire organs or complex tissues remains a challenge. So what are the outstanding hurdles, and how many/in what order do we tackle them?

How can the production process for autologous cell therapies be made faster and more efficient?

Current production methods
(for instance for CAR-T cells) are currently expensive, slow and unreliable. How can each process - cell harvesting, selection, transduction, expansion, reformulation/preservation - be simplified and accelerated. Are there new technologies that can achieve this, or can the whole process be reinvented?

Which engineered tissues and/or organ products are closest to commercialisation?

There has been a lot of academic interest in engineered tissues and organs for therapeutic use. However, so far very few have been approved by regulators. What products or classes of product are in clinical or pre-clinical development and what are the hurdles to commercialisation (particularly related to scaled-up manufacturing).

What are the main problems in viral vector development and production?

Access to viral vectors has been identified as the major bottleneck in the development of ex-vivo gene therapies. What are the main difficulties in developing and scaling up production of lentivirus?

What are the most labour intensive aspects of academic cell therapy R&D?

Researchers spend a lot of time and effort culturing cells. Although this is an inefficient use of resources and several different systems are available to automate cell culture, none has been widely adopted in R&D. What are the reasons for this?