Harnessing the power of the immune system: from bispecific antibodies to CAR-Ts and beyond

by Diane Seimetz

The first class of therapeutic products harnessing the power of T cells for target-specific treatment of oncological diseases was bispecific antibodies. Eight years later, the first autologous chimeric antigen receptor (CAR)-T cells received regulatory approval. In this article, Diane Seimetz reviews the current landscape of CAR-Ts, recent approvals  and further important developments in the cell- and gene therapy space.

Let’s Celebrate! – Success Stories from Biopharma Excellence 🍾

Already in the beginning of 2019, the Biopharma Excellence has several good reasons to celebrate. The highlights with major contributions by the Biopharma Excellence team were:

  • An IND for a first-in-class product was approved by the US FDA.

  • Two products where Biopharma Excellence was making significant contributions during drug development, dossier preparation and the authorization process obtained marketing authorization in the EU.

 Together with our clients, we have been very delighted, and we congratulate the entire team to these great achievements. These are just the first success stories in this year, there are more to come! 🍾

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Pregnant women and vaccines against emerging epidemic threats

by Michael Pfleiderer

Vaccines have one of the highest live saving potentials amongst all medicinal products licensed around the globe. One commonality of all these vaccines is that they are developed for all age and risk categories except pregnant women. Although some vaccines are specifically recommended for use in pregnancy, access to novel vaccines is generally blocked for pregnant women. Michael Pfleiderer summarizes which advances have been made to overcome this dilemma.

Drug repurposing: how to protect your asset by an integrated regulatory strategy

by Michael Firgens

Drug repurposing is an alternative cost-effective approach to drug development which builds on already approved drugs and compounds. However, the actual commercial success of a repurposed drug depends on achieving effective protection against competitors such as generics and other products with the same active pharmaceutical ingredient. In this article, Michael Firgens explains the key strategies for the protection of a repurposed drug.

Season’s Greetings and a Happy New Year from Biopharma Excellence 🎅

2018 has had many great memories, such as the BIO Europe 2018 in Copenhagen, and this year is coming to an end soon.

We would like to express our sincerest gratitude to all our clients and cooperation partners, and in this past year we have grown with our clients’ projects. More colleagues have joined the Biopharma Excellence team and support biopharmaceutical development projects with passion.

We are confident that in 2019 many new adventures and successes lie ahead of us. We wish you happy and peaceful holidays and a great start in 2019.

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Cell and gene therapies are changing the face of modern medicine – But how to find the right dose?

by Diane Seimetz

Finding the right dose for the first in human study can be a challenging undertaking for biopharmaceuticals. For cell and gene therapy products (commonly referred to as ATMPs), this is even more challenging. In this article, Diane Seimetz reminds us what Paracelsus’ toxicology principle has taught us, and she explains what the key questions for ATMPs are to guide a tailored dose-finding exercise.

Cutting costs for cutting edge therapeutic products: ATMP process optimization

by Elena Meurer

The affordability of ATMPs for the health care system and patients lies among the important aspects that clearly require further improvement. The overall high price of cell-based therapeutics, as known today, is driven by specific factors involved in development, manufacturing and clinical logistics. In this article, Elena Meurer explains where to cut costs in the manufacturing process of ATMPs.

Four important considerations for the successful development of an in vitro or companion diagnostic test

by Linsey Reavie

In vitro diagnostic (IVD) and companion diagnostic (CDx) tests are crucial for the rapid diagnosis, response and treatment of diseases. IVD and CDx developers must satisfy a diverse set of evaluators to ensure commercial success, such as regulators, health care providers, physicians, and reimbursement organizations. While these sectors have recently seen significant growth, they have also been met with an increased demand from regulatory bodies to demonstrate the clinical utility for patient treatment. Linsey Reavie explains what to consider for a successful development of in vitro or companion diagnostic tests.

Lost in translation? – How you can smoothly progress to clinical development and beyond

by Diane Seimetz

For a smooth transition to clinical development stage, careful and intertwined upfront planning across important drug development disciplines is essential. Based on our experience, even mature research teams and start-up companies often underestimate the challenge to progress their product candidates as well as their teams from the R-stage (Research) to the D-stage (Development). In this article, Diane Seimetz tackles the issue and provides solutions.

What ATMP developers should know when looking for a CMO

by Elena Meurer

As ATMP start-ups mature, they will face the following challenging decision: whether to develop, test, and manufacture its products in-house or to outsource the activities to a contract manufacturer (CMO). ATMP developers who do not have their own GMP facility or plans to set-up their own plant, will need to look for a CMO. In this article, Elena Meurer provides a comprehensive and structured guide how to select a CMO for the manufacture of ATMPs.

Genotype vs Phenotype - The impact of new insights from human genetics on the future of drug development

by Maria Schacker

The genetics of disease inheritance is becoming more and more important for the pharmaceutical industry. Until the early 2000s, the only way to study genetic diseases was through family studies and linkage analysis in families with high occurrences of disease. However, these studies are extremely tedious as they can only be done on a small scale and they are by and large only applicable to diseases that follow mendelian inheritance, not multifactorial diseases. In this article, Maria Schacker provides an overview on how the insights from human genetics can be used for the development of new drugs. 

Drug Repurposing – A Smart Development Strategy to Make Drug Development Cheaper, Faster, Safer and More Successful

by Michael Firgens

Drug discovery and the translation of research findings into new therapies require significant investments and resources by biopharmaceutical companies. Analyses across all therapeutic areas indicate that the development of a new medicine, from target identification through marketing authorization, takes over 12 years and often much longer. Michael Firgens will tell you how the repurposing of known drugs, serves as a more attractive, alternate development strategy.

Hot Summer Topics from Biopharma Excellence

Not even the record high heats ever detected in Europe will scorch our fiery passion towards biopharmaceuticals. Biopharma Excellence has taken a full advantage of the summer and our team has been boiling some sweltering updates for you. We hope our current scientific and regulatory news will blaze your mind, sizzle your mood and ignite a flaming discussion on the future of these burning topics.

Also, the Biopharma Excellence Team is growing and has welcomed Karl Heller. The photo above shows us in August 2018. Left to right: Rajeswara Pannem, Luka Kotrikadze, Iris Mayer, Sabine Kistner, Michael Pfleiderer, Elena Meurer, Annegret Vaggelas, Karl Heller, Michael Firgens, Astrid Kremer, Diane Seimetz, Gabriele Dallmann.

Regulatory Science as an Important Principle of Good Consultancy Practices (“GCP”)

by Michael Pfleiderer

Regulatory science is the foundation of regulatory decision-making and is used to assess the quality, safety, and efficacy of human medicinal products throughout their life-span. The domains, covered by regulatory science, are considered to include both basic and applied biomedical sciences, clinical trial methodology and epidemiology, and social sciences. In the report by Michael Pfleiderer, you can read about the importance of regulatory sciences to contribute to the development of new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of regulated products.

The Devil is in the Details – Trouble Shooting for Biopharmaceutical Products

by Karl Heller

Harmonious flow of the industrial processes greatly depend on systematically complying, updating and maintaining the SOPs. However, sometimes the worst happens and the product of the industrial manufacturing is not in compliance with the quality standards, which underscores the deviance from the SOPs. Karl Heller will tell you how such cases are handled and how trouble shootig is conducted in the processes of industrial scale.

Advanced Therapies in Wound Management: What Needs to be Considered for an Integrated Development Strategy

by Diane Seimetz

The great potential of advanced therapies in wound management was recently demonstrated by a case study where the entire human epidermis of a boy with junctional epidermolysis bullosa (JEB) was regenerated using genetically modified autologous keratinocytes. Despite this, over the past 11 years, only 12 advanced therapies received a positive opinion for authorization in the EU, which underscores the necessity for an integrated development and regulatory strategy to realize the potential of advanced therapies, in particular for wound management. In this article, Diane Seimetz provides insights on the challenges companies are facing and what needs to be considered at each stage, from R&D up to authorization. Moreover, Diane Seimetz and Anne Vaggelas have recently published an article on this important topic in the Journal of Wound Care.

Biopharma Excellence Runs for Life! 🏃‍♀️🏃‍♂️

As one of the global charity events, Biopharma Excellence participated in the Wings for Life Run, 2018 in Munich (Germany). Aside from supporting leading biopharmaceutical and life science companies in tackling the biggest health challenges of our times, our team is excited to have contributed by participating in a charity event that donates to spinal-cord research projects and clinical trials at renowned universities and institutes worldwide. 🏃‍♀️🏃‍♂️

Expediting the Development of Advanced Therapies such as CAR-T cells by making use of FDA’s RMAT designation

by Annegret Vaggelas 

In 2017, the first CAR-T cell therapies (Kymriah, Yescarta) have been approved by FDA. End of June 2018, EMA followed by recommending marketing authorizations for both of them in the EU. These first-in-class medicines hold enormous potential to cure some of the deadliest and largely untreatable cancers and are expected to shift the focus of medicine from treating symptoms or delaying progression to curing diseases. Further CAR-T cell therapies are under development and are expected to reach the market not too far from now. Read the article below to learn how RMAT works and which products can be considered for designation.