GBReport - September 2024

GBReport - September 2024

??News From the GBR Team

?? Biotechnology News

?? M&A in BIotechnology - Top 5 Deals

?? Tech Trends

?? Did you know?

?? Learning About AI, Bioinformatics and Genomics

?? Tips for Entrepreneurs

?? What is it like to be a Research Associate?


??News From the GBR Team

The Story of GLP-1 - Workshop

As told by the woman who pioneered this class of drugs with:

  • Lotte Bjerre Knudsen, PhD (Chief Scientific Advisor Novo Nordisk)
  • Walther Traberg, PhD (GBR Ambassador, Moderator)

Workshop - September 13th - Register Here!


Bioinformatics and Big Data in Biotechnology

Opportunities and Challenges in Africa

Discover how big data in biotechnology enables advanced analytics to optimize sectors like healthcare and agriculture. Dive in into how genomics offer personalized treatments for African populations.

  • Dr. Oladipo Kolawole (CEO & Founder, Helix Biogen Institute)
  • Zainab Ogunjimi (Medical Student at University of Lagos, GBR Ambassador)

Watch it here!


Are you a GBR Alumni? - Networking Opportunity

Join the new GBReach Virtual Networking Program!

Register, match with other GBR Alumni based on personal and professinal interests, and meet at your convenience!

  • Registration opens 26th September!


?? News

New Framework on Gene Transfer Medicinal Products (GTMPs)

The European Pharmacopoeia has updated its guidelines for gene therapy medicinal products (GTMPs) to reflect the growing number of approved therapies and ongoing clinical trials in Europe.

The new general monograph and accompanying chapter, adopted in March 2024, replace the earlier standards and provide a more standardized yet flexible framework for the production and regulation of GTMPs. This marks a step towards ensuring the safe and effective development of gene therapies, contributing to public health improvements across Europe.

If you want to know more, there will be a webinar on the 3rd of December, link here.

March, 2024


The Sound of the Future: Gene Therapy for Children's Genetic Hearing Loss

Recent advancements in gene therapy for genetic hearing loss have sparked significant optimism.

  • Regeneron's DB-OTO therapy has shown "dramatic" improvements in hearing for 2 young children with genetic deafness
  • Akouos's AK-OTOF therapy has improved from profound deafness to mild-to-moderate loss in an 11-year-old
  • Fudan University and Mass Eye and Ear have reported that 5/6 children treated with their AAV1-hOTOF therapy regained hearing and improved speech and sound recognition

The therapies target otoferlin deficiencies, a protein critical for hearing, by delivering a functional gene copy to the inner ear.

August, 2024


?? M&A in Biotechnology - Top 5 Deals in 2024

  • February, 2024 - Novo Holdings, the investment firm for Novo Nordisk Foundation, is to acquire Catalent, a global CDMO for pharma and biotech for $16.5B.
  • February, 2024 - Bristol Myers Squibb has completed the acquisition of RayzeBio, Inc., a company focused on Defeating cancers with radiopharmaceutical medicines.
  • March, 2024 - Bristol Myers Squibb acquired Karuna Therapeutics for an equity value of $14B. Karuna brings KarXT (xanomeline-trospium), an antipsychotic with the Prescription Drug User Fee Act (PDUFA) (Sept. 2024) for the treatment of schizophrenia in adults.
  • March, 2024 - Gilead Sciences announces the acquisition of CymaBay ($4.3B in total equity value). This clinical stage biopharmaceutical company is focused on the liver and other chronic diseases like Seladelpar, an orally active PPARδ agonist for autoimmune liver disease.
  • April, 2024 - Vertex Pharmaceuticals to acquire Alpine Immune Sciences, a clinical stage biopharmaceutical company developing protein-based immunotherapies, for approximately $4.9B.


?? Tech Trends

Organ-on-Chips

An organ-on-chip is a model composed by the cell types found in a certain organ that can realistically emulate it.

Researchers use them to investigate mechanisms of interest and evaluate the efficacy of drug candidates in various diseases.

But Why?

The goal is to:

  • Speed up drug development
  • Identify toxicities
  • Promote personalized medicine

These chips have been designed to replace animal testing in the processes above.


Bioprinting

Bioprinting involves 3D printing with “Bio-inks” (active human cells). The market was valued at $2.13B in 2022 and is expected to hit $8.3B by 20301.

While the end goal of this technique is to print tissue for non-rejected transplants, it is also being used to create lab-grown organs that emulate the blood-brain barrier or the alveolar substance exchange, among others.

The obtained tissues aim to replicate the behavior and structure of the original ones.

Although bioprinting is a promising technique, it is necessary to decrease its complexity and make it more available and cost-effective.

1 Precedence Research


?? Did You Know...


Fabian et al., Nature Communications, 2023

?? Learning About...

Bioinformatics and Big Data in Biotechnology

With the exponential growth of big data in biotechnology now, more than ever, is the time to leverage advanced data analytics to identify patterns, predict outcomes and optimize processes across various sectors such as healthcare, pharmaceuticals and agriculture.

One of the applications of big data in biotechnology is genomics. By analyzing large datasets of genetic information, we can identify biomarkers that improve disease diagnosis and develop personalized treatments that cater to the unique genetic makeup of Africans. This approach not only enhances treatment effectiveness but also offers a deeper understanding of disease progression and response to therapies of the African populations.

Despite the potential, challenges such as inadequate funding, limited research, data quality issues, and a shortage of experts persist. Addressing these challenges requires collaboration with investors, partnerships in research, and building capacity through training and retention of skilled professionals.

By Zainab Ogunjimi - Medical Student at University of Lagos & GBR Ambassador


AI in Genomics

Artificial intelligence (AI) holds immense potential in genomics, especially in enabling early diagnosis and predictive care, which aligns with the global trend toward personalized medicine. AI is poised to revolutionize treatment options by analyzing vast amounts of genomic data, identifying patterns, and making associations that would be difficult for humans to detect.

AI can significantly reduce the time and cost of genome sequencing, accelerating discoveries in genetic research.

In drug development, AI can help identify drug targets by simulating preclinical outcomes, and streamline the process of patient matching for clinical trials based on the omic profiles. This approach ensures that patients receive the most effective treatments, increasing the likelihood of success in clinical trials and personalized therapies.

However, AI algorithms are only as good as the data they are trained on. If the data is incomplete or biased, the results may be inaccurate, potentially affecting outcomes. Ensuring the quality and diversity of training data is critical to improving the precision and reliability of AI models in genomics.


?? Tips For Entrepreneurs

The Team

  • Teams with more than 1 founder are more likely to succeed, as having a diverse team often leads to addressing a wider range of needs. This diversity also tends to bring in more capital and a stronger network (McCarthy P. X. et al., Scientific Reports, 2023).
  • It’s crucial that your startup eventually has an expert for each key area. For example, if you are developing an AI platform to detect genomic anomalies, you'll need a CTO or a Head of Engineering to fulfill that role.
  • If you don't have prior experience in entrepreneurship or business, it's advisable to bring in a business expert who can help define the business model and guide your investment strategy.
  • Growing the team sustainably is also essential. Financial planning should be a priority from day one.


?? Being a Research Associate With...

Miriam López Ferreiro - GBR Ambassador

What is a Research Associate’s Role?

I am currently working with rAAVs for their use as delivery platforms in Gene Therapy. Within this role, you are expected to work with cells, perform purification methods, and implement analytical techniques for the determination of critical quality attributes, as well as other general tasks such as method optimization or process development.

"Focus on the right direction, work hard and smart, keep connected to the world and what is needed out there, pay attention to the little details, be resilient to embrace the challenges that you will find on the way, work on your ability to understand other people’s feelings and points of view, be authentic. But, above all, enjoy the process, learn from everything and everyone and be always grateful for what you have”.

What Does the Progression Look Like?

A first experience working independently in an academic lab would be a good first step to gaining knowledge and confidence. Later on, an internship in industry could help you understand how it differs from academia while gaining responsibilities that could be relevant for your next role.

The research associate position is mainly focused on lab work, which can be more or less research-based depending on the department where the group is located. With years of experience, you can get into more senior positions that can later derive to more managerial roles or, in most cases, keep it more related to lab work.



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