SYNTHETIC BIOLOGY FUTURE DIRECTIONS
SYNTHETIC BIOLOGY FUTURE DIRECTIONS / Synthetic Biology offers new ways for engineering new biological systems or re-designing existing ones for useful purposes. It is a disruptive technology at the heart of the so-called Bioeconomy, capable of delivering new solutions to global healthcare, but also in many other fields like agriculture, manufacturing, and environmental challenges. Not long ago, in May 2010, a team of scientists led by Craig Venter achieved a synthetic biology milestone by designing the genes for a new type of bacteria and implanting them in a bacterial shell that had its genome removed. The empty cell behaved like a factory programmed with human-designed genetic code. It was the first living being to escape the evolutionary tree, with a computer-programmed genome synthesized by chemical compounds, implanted in a cell, and subsequently multiplied under the control of that artificial genetic code. It opened the way to extraordinary developments in biology and medicine that are starting to blossom now. But we have a lot of work ahead. This quick_note tries to systematize the progressive steps in our quest to design “therapeutic life” #quick_notes_lp
LAUNCHING A BIOTECH STARTUP
Solving real problems requires commitment and focus. Here’s some advice for scientists willing to become entrepreneurs, six things you should think about before embarking on your journey:
(1) UNDERSTAND BIOTECH ECOSYSTEM/ Be aware of what creates and what destroys value, and understand why VCs prefer good problems to solve, big clinical needs, accelerated regulatory pathways and a big delta in what the drug will do for patients: USE IT TO IMPROVE YOUR POSITIONING
(2) UNDERSTAND YOUR COMPANY/ Ask yourself what open questions still remain unsolved in your field (they’ll lead you to clinical needs), and see the landscape of strategies to solve your particular clinical need: are you competitive enough? STAND OUT FROM THE CROWD
(3) FROM SOLOIST TO ORCHESTRA/ Find the right CEO (maybe yourself, but some other times someone else more experienced) and the right team that will make it possible… At first the team will be small but always bear in mind entrepreneurship is not an individual game
(4) CONTACT YOUR TECHNOLOGY TRANSFER OFFICE (research center, hospital…) or alternatively experts and consultants to think carefully about the whole process, the intellectual property, some initial understanding of the regulatory pathway, license negotiations to spin out, etc.
(5) FINE TUNE PoC/ Find your WOW slide, the experimental setting and results that will convince everyone that this is worth doing and that your way of solving the problem actually works… You’ll probably need to further improve it after visiting VCs, yes, but always aim for top quality experiments
(6) FUNDRAISE / Go find capital (non-dilutive from grants first, if possible), understand how VCs will analyze your project and try to capture their attention. The process iterates non-stop, go back to (1) to further understand your sector and start again to perfect your pitch
RETINA (DIFFICULT INDICATIONS):
Retinal diseases remain a difficult problem to solve because (1) the retina is in a sense a biological computer that needs to be properly “rewired”, (2) animal models are poor because they have no fovea, (3) the internal limiting membrane (ILM) limits delivery, and (4) regeneration of mammalian retinal is absent. However, very promising strategies are emerging, here is a conceptual map of the most interesting approaches to solve the problem. #quick_notes_lp
Glioblastoma remains one of the most pressing clinical needs in medicine. Its prognosis is dismal and current standard of care involves maximal safe surgical resection, radiotherapy and chemotherapy. News drugs are on the way though: many groups across the world are trying to solve the challenge. It is indeed a difficult challenge because (1) the blood brain barrier limits PK/PD, (2) new studies confirm glioblastoma stem cells confer resistance, (3) clinical trials are usually done in recurrent patients, and (4) no monotherapy strategy will probably eradicate it completely. It is a especially dear field to me because as some of you may know I used to operate them as a former neurosurgeon and I’ve seen first-hand how aggressive they are.
All big unsolved clinical needs may be a good investment opportunity. Here’s a conceptual map with some of the leading-edge alternatives and targets that are being moved forward.
You can find all my quick notes at #quick_notes_LP
DIFFICULT INDICATIONS: PANCREATIC CANCER
The higher the clinical need and the prevalence, the higher the effort from scientists around the world in trying to solve a problem. Pancreatic ductal adenocarcinoma (PDAC) remains one of the highest unsolved clinical needs today. It is a devastating disease with an unfavorable outcome and is projected to become the second deadliest cancer by 2030. Currently the overall 5-year survival rate is less than 10% despite all efforts. But new therapies are being pushed forward every month, the rhythm of innovation here is extraordinary.
All big unsolved clinical needs may be a good investment opportunity. Here’s a conceptual map (by no means complete) with some of the leading-edge alternatives and targets that are being moved forward. A map just to navigate the landscape and avoid getting lost amid all the different categories and approaches.
You can find all my quick notes at #quick_notes_LP
LAUNCHING A BIOTECH STARTUP
Some advice for scientists willing to become entrepreneurs, here are six things you should think carefully before embarking on your journey… (OPENING THREAD: some slides I used in a lecture @EU_Commission last month)
ONCOLOGY FUTURE DIRECTIONS
Here’s a map of some of the most promising research fields in oncology today, plus a video —below— with some advice for scientists willing to become entrepreneurs (from a debate on the future of oncology and how to make it happen)
CANCER BIOLOGY OPEN PROBLEMS
One of the most important things every investor looks for in a startup is a good problem to solve… Unanswered questions always signal what’s next. Here’s a map of some of the most pressing questions in the oncology field today.
RNAs are very active players in cell biology… they carry instructions for making proteins, turn genes on and off, aid chemical reactions, slice and dice other RNAs, and build proteins. Here’s a simple map to understand how can we use them as drugs
COVID19 VACCINES LANDSCAPE
A different view on phase II – III #COVID_19 vaccines, organized on a spectrum ranging from less innovative technology (older, slower, less uncertain development) to more leading edge approaches (faster, riskier)
THE ART OF CHOOSING INDICATIONS
You already have a lead candidate for a first specific indication, here’s a simplified conceptual map on how VCs think to choose the most interesting new indications (pipeline strategy).
INVESTMENTS IN THE TIMES OF #COVID19
Every crisis brings risks and opportunities to the table… here’s a mind map on how SARS-CoV-2 can impact the activities and value chain of the life sciences VC industry
Most rare diseases still lack approved treatments despite major advances in the knowledge of their molecular basis. Here’s a map of the different therapeutic modalities plus an update on some of the most interesting new developments in each field.
The vaccine effort for #COVID19 is unprecedented in scale & speed. To get it right and fast we need new tools and strategies including DNA/RNA vaccines, virus-like particles, T-cell inducing peptides & viral vectors… here’s a map of the leading edge
Emerging Fields: PROTAC (2)
These protein-slaying drugs can be used to go after targets that drug developers have long considered ‘undruggable’ and may become the next blockbuster therapies… The gold rush is on! here are some areas of research.
Fighting rare diseases
A strategic map to fight rare diseases (resulting from a defective gene)… in medicine there are no outliers, no circumstances rare enough to be ignored; every disease, no matter how rare, needs our attention.
Cell & Gene Therapies
Hundreds of trials are underway with some major therapeutic breakthroughs… Will their use become ubiquitous? Or will its adoption be slow? Here’s a map of the things that matter when discussing CMC issues to develop an advanced therapy.