April 2, 2026 Amir Saberi

Science Talks
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Science Talks: Dr Amir Saberi

Thursday April 2, 2026 at 10:30am EST

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Peek behind the curtain and learn how science and entrepreneurial spirit can lead to tech development and innovation! Join Dr Amir Saberi [LinkedIn], a Founder & CEO of biotech startup Ambryon as he talks about his Academia-to-Biotech path designing and building biomanufacturing infrastructure for accelerating cell and gene therapies.

Amir received his PhD in Cell and Developmental Biology from the University of Illinois Urbana-Champaign and did his postdoctoral work at the Johns Hopkins University School of Medicine. He then worked as a Senior and Principal Scientist at Domicell and Vita Therapeutics, transitioning to Scientific Operations at Vita before founding Ambryon. Stem Cells, CRISPR, plasmids, AAVs, gene therapy, biomanufacturing; postdoc, scientist, director, CEO; Industry, startups, biotech - Ask him anything!

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  1. When you are growing stem cells in a large bioreactor instead of a small lab dish, do the cells ever change or stop working correctly because the environment is different?

  2. When using tools like CRISPR or base editors to fix DNA, how do you make sure the “molecular scissors” only cut the specific spot you want and don’t accidentally damage other parts of the genome?

  3. What was the most surprising challenge you faced when you went from being a student and researcher to starting your own company?

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How are researchers currently improving the specificity and control of gene editing in vivo, and what advances do you think are most promising for making these therapies safer for clinical use?

Your work involves technologies like CRISPR and stem-cell biomanufacturing. What computational or AI-based tools are becoming most useful in designing gene-editing strategies?

You transitioned from academia to biotech and eventually got into the entrepreneurship field. What experiences helped prepare you the most for entrepreneurship?

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  1. Amir’s company focuses heavily on adenovirus associated virus (AAV) gene therapies. From what I’ve read, AAVs do not incorporate their cargo DNA into the host genome which is why AAV gene therapy is targeted at post mitotic tissues like neurons. My question is how AAVs are designed (by his company for example) to target specific tissue types? Are there AAV libraries and its as simple as loading the cargo DNA?
  2. How would you compare the gene therapy delivery systems lipid nanoparticles and AAVs to each other? What are their advantages and disadvantages respectively?
  3. Why did you choose to go into the private sector and eventually found a company? I think the current academic pipeline skews heavily towards academia or government research so I am curious as to how your post studies experience has been outside of academia. How is the private sector better or worse?
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  1. How do smaller base editors compare to traditional SpCas9 editors in terms of editing efficiency and off-target effects in vivo, especially for diseases requiring high precision, like sickle cell or DMD?
  2. What computational or experimental strategies do you think are most promising for designing compact base editors that still retain high specificity and efficiency?
  3. For an undergraduate student interested in gene therapy and genome editing, what are the most valuable skills or experiences to build early on, and how would you recommend finding a strong mentor in this field?
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  1. Why are the editing efficacies of compact nucleases vary across different tissues (i.e. 30% in skeletal muscle but 15% in cardiac muscle)? And what current approaches are researchers taking to increase the editing efficacy of compact nucleases for gene editing in different tissues?
  2. What are the clinical implications of compact base editors? How does this technology promise to advance gene therapy to treat human diseases which can’t be achieved by current means of gene editing?
  3. I would like to know more about the biotech startup space and how we can become more involved with this field post-graduation?
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  1. In the article about precardiac organoids forming the two heart fields, you provided key insight into potential new techniques to study heart field/chamber-specific cardiac disease, and I was curious to see if this ever did happen, and if not, I’m curious to see if there are any treatment applications of the results of this study.
  2. Throughout my time conducting research in this class, and throughout my undergraduate experience, there has been a growing discussion of the usage of AI, and I was curious to hear your inputs and how you have utilized AI successfully, as well as areas where you have seen it fail. Overall, what do you think the future of AI is in the sphere of biology?
  3. What drove you to pursue a private biotech startup rather than continuing in academia? Also what drove you to your own startup rather than the companies you previously worked for? If ever, when have you considered returning to academia?
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  1. Given your work on stem cell bioreactors and regenerative therapies, how do you see scalable stem cell expansion systems influencing tissue-specific regeneration (e.g., musculoskeletal vs. neural tissues), and what are the key biological bottlenecks that still limit clinical translation?
  2. In developing stem cell bioreactor platforms, how do you integrate computational modeling or multi-omics data (e.g., single-cell RNA-seq) to optimize cell fate decisions, and do you think these approaches are becoming essential for next-generation regenerative therapies?
  3. What early decisions (e.g., choosing labs, developing technical expertise, balancing clinical vs. research exposure) most strongly shape long-term success, and how would you recommend identifying the right mentor in this field?
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  1. Some scientific questions I have is how more on how AAV works. I have never heard of adeno-associated virus before and I am not familiar with related literature. I want to know how exactly this virus interacts with its environment and if there are limits to what part of the body AAV can deliver medication to. Would AAV be applicable for assisting in maladies such as Adenomyosis where endometrial tissue grows within muscle tissue?
  2. I am curious on how accurate and precise you think AI is in the context of your new technology. Do you think the AI that is available today is strong enough to handle this kind of data? Is there possibility for AI to “hallucinate” while processing the data?
  3. Where do you think you have found your most meaningful connections? Is there a pattern or do you have to be at the right place at the right time? When finding mentors do you ask them directly or is it more of an unofficial process?
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  1. What does it mean for a virus to need a helper virus to propagate? How does that work?
  2. Do you encounter any ethical issues doing your work with stem cells and gene editing? How do you resolve them if they arise?
  3. What was your favorite and least favorite thing about working in academia? What about in biotech?
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  1. I was fascinated by the use of an intravascular bioreactor to deliver anti-inflammatory factors for Acute Respiratory Distress Syndrome (ARDS). How do you determine the optimal dose/delivery of these paracrine factors coming from the bioreactor? Does the bioreactor have a way to sense the patient’s inflammatory levels and adjust its output in real-time (or does it just release it constantly at a steady rate?)
  2. When you are designing guide RNAs, are there specific computational tools or wet lab validation steps you consider non-negotiable before moving a design into a therapeutic model?
  3. I was interested in what pointed you more towards biotech startup rather than academia?
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  1. What are the biggest scientific hurdles that need to be overcome for therapies to become affordable to use widely?

  2. What computational methods will be the most important for gene therapy in the next 5 years and how can students prepare for that?

  3. What types of students would excel in academia vs biotech startups? What experiences or skills helped you? How would you recommend finding mentors?

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  1. When developing a new therapy or technology, how long does a project take and what factors are a part of that?
  2. Do you ever have to balance public concerns with your development of new treatments?
  3. What experiences aided you the most in getting you to the place you are now? How did they lead you to become more specialized in the field you work in?
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  1. Science – What would be the specifics for the optimization steps on the SCIB cellular component for the clinical-grade model?

  2. Method – How did the prototyping for these technologies work?

  3. Career – What allowed you to make the big leap into startups in the first place?

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  1. Why do you think the signaling in flatworms in their neuropeptides can be similar to vertebrate systems overall?
  2. How you think AI-based tools will change in the future for genomics and what kinds of new systems do you think can be developed?
  3. What kinds of things should people looking to go into biotech keep in mind (general lessons)?
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  1. How do Adeno-associated virus delivery systems affect how well CRISPR works in real treatments?
  2. What is the hardest part of scaling up cell and gene therapy production while keeping it high quality?
  3. What was the biggest mindset change when you moved from academia to leading Ambryon?