>FASTA_15
Prime Medicine dose, Synthego bankrupt, MeliBio acquired, Oxford Nanopore goes multiomic, and viruses secretly run the world + Techbros look to biotech, Blackmirror, and CRISPR real-life Mickey Mouse
>FASTA is a weekly newsletter sequencing the biotech ecosystem | Papers and patents, acquisitions and bankruptcies, and some biotech philosophy as dessert | Read in less than 5 min | Follow along on LinkedIn and X
#1 Prime Medicine dose
Last week, Prime Medicine announced their first patient dosed with a prime editing therapy against granulomatous disease, a dangerous condition that disables immune cells. The patient has experienced no serious side effects, and enzymatic function has been restored in two-thirds of his neutrophils1.
But launching gene therapies to the market takes more than just science. For all the media coverage, Nobel and Breakthrough Prizes, there’s still only a handful of FDA approved gene-editing therapies today, most cost more than $1M USD per dose (excluding IMLYGIC, ADSTILADRIN, and VYJUVEK), and some have been discontinued due to alleged “limited interest” from patients and doctors anyway.
Prime Medicine’s former CEO is now being replaced by their former CFO. The company will focus on in-vivo therapies to avoid the costs of cell harvest, gene modification in GMP labs, expansion, quality control, reinfusion, and logistics that ex-vivo therapies entail.
#2 Synthego’s bankruptcy
If gene editing companies are in trouble, you can only imagine how their suppliers are doing. Synthego was a “CRISPR solutions provider” specialized in the manufacturing of therapeutic and research-grade gRNAs. They estimate that at some point, ~25% of the ~1,000 global cell/gene therapy companies used their products.
This postmortem is not unlike other biotechs’: revenue growth between 2020 to 2023 and cost-cutting in the last year were not enough to catch up to their $100-500M in liabilities. After nearly a decade of operation and $400M of investment rounds — from Founders Fund, Jennifer Doudna, and the like — they filed for bankruptcy to be acquired by major biotech hedge fund Perceptive.
There is no logical reason why technologies like biomanufacturing and certainly gene therapies will not capture billions in value. Even beyond harsh economic environments, recent bankruptcies are a testament to the need for scalability breakthroughs and a good management team, in addition to the science — Time for the next wave.
#3 MeliBio acquired by FoodYoung Labs
Switching strands, our friends from MeliBio just got acquired by the Swiss food group FoodYoung Labs. The world’s first plant-based honey company developed a bee-free honey “Generation 1” technology, first commercialized it via the Mellody® brand to customers like 3-Michelin-Starred Eleven Madison Park, and recently scaled to sell to major retail partners such as Aldi.
It’s been a year since I interviewed Darko, CEO and cofounder of MeliBio on the Biofounders podcast. Perhaps the greatest lesson here is how crucial it was for him as a CEO to have worked in the honey industry before. Rather than going for the much-hyped precision fermentation approaches, the team focused on building a product people loved, and scaled it through manufacturing partners instead of building it in-house — Congrats guys!
#4 Oxford Nanopore goes multiomics
Today, most proteomics relies on mass spectrometry, which requires labor- and time-intensive and costly sample preparation that can introduce bias as some peptides ionize or fragment better than others. Nanopore wants to change that by identifying and sequencing native proteins directly (presumably through methods similar to those described in this paper).
For now, they have unveiled a simultaneous protein detection, quantification, and barcoding system in which the peptide of interest is captured between two DNA handles (barcodes), passes through a nanopore, and generates a distinct signal, at the same time that the DNA gets read at Q20+ accuracy.
This new era of proteomics could unlock biomarker detection at ultra-low concentrations. Just like an iPhone, an iPad, and an iMac are built out of similar hardware but use different chips for their specific use cases, Nanopore envisions creating a single multiomic device that can read DNA, RNA, and proteins by simply changing the nanopore.
#5 Yes, viruses secretly run the world.
8% of the human genome is made of Endogenous RetroViruses (ERVs, code for viral fossils that we got who knows when). You may have heard about the most famous one, syncytin, which is essential for placenta formation. Well, shit gets crazier when we talk about the brain.
LINE-1 (L1) retrotransposons, for instance, can become active during learning and result in impaired long-term memory formation in mice when inhibited. Arc, another viral gene, forms fucking viral-esque capsids to transfer mRNA between neurons like a virus (what the fuck?!), which now seems essential for memory consolidation and synaptic plasticity.
Other viral sequences are altered in neurodevelopmental disorders like schizophrenia. Upon infection, the maternal immune activation system can release cytokines that cross the placenta and influence fetal brain development — Could we find the answer to other deep questions about consciousness in viruses too?
Bonus recommendations
More techbros waking up to biotech. Coinbase CEO Brian Armstrong & ex-Google scientist Guillaume Verdon expressed their “merely intellectual” interest in gene editing on
this last month. I wouldn’t be surprised that such interest has actually been reflected in investments like Sam Altman’s in RetroBio already. I’m, in fact, very surprised we haven’t heard Elon invest in biotech to colonize Mars, or edit his children’s genomes, or grow them in artificial wombs. Who knows, 2025 is the year of cooking new weird things, so maybe he’s looking into it too.Black Mirror season 7. From my POV, all episodes strongly focus on AI and neurotech. My favorite questions were on whether: new biotech should adopt subscription models, online content creation will entail “selling your soul/dignity”, my loved ones and I will trust each other more than we trust AI, some people will actually prefer living in an AI fantasy than the real world. If you or anyone you know is connected to Black Mirror, we’ve really gotta freaking chat for a gene editing-focused season 8 😼.
CRISPR real-life Mickey Mouse. DNA is a language, but does it encode natural language? A crazy study published in February of this year genetically edited mice (yes, using CRISPR) to express the human version of the NOVA1 gene, which codes for a neuronal protein. Those mice presented significantly more complex vocalization patterns, suggesting the gene’s role in the development of spoken language during neuronal development. While biology is always more complex than a gene, these kinds of studies make me very optimistic that we will be able to create a real-life talking animal in less than 15 years from now.
This disease was the perfect target for prime editing since it a two-letter insertion was needed. Here, pegRNA (prime editing gRNA) not only targets the desired DNA site but also encodes the intended genetic change. Its fusion cuts only one strand of DNA and copy-pastes the desired sequence into the genome using a reverse-transcriptase.
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