5 migliori aziende di cellule staminali in cui investire (giugno 2026)

Il potere della cellula staminale

Our bodies are made of hundreds of different cells. Neurons, muscles, blood vessels, liver, kidney, etc., each are made of very different cell types. They differ in many respects: in biochemistry, size, shape, capability, and more.

But this was not always so. We all come from just one cell that later turns into an embryo and then a fetus. Every cell in our body contains the entire genetic program to become any cell type. Such cells are called “stem cells”. But once turned into a muscle cell, it can never evolve into a neuron. So, we distinguish 2 types of stem cells:

• Pluripotenti: in grado di trasformarsi in qualsiasi tessuto possibile.
• Multipotenti: in grado di trasformarsi solo in un numero limitato di tessuti e tipi cellulari.

Fonte: BioRad

Because stem cells can become anything, they also have massive therapeutic potential. They could replace damaged tissue after a heart attack. Or neurons degenerating from Alzheimer’s disease. Or replace several nerve tissues in a broken spine.

Guarire con le cellule staminali

This potential has long been known, but a few problems stand in the way:

1. Le cellule staminali sono rare nel corpo e richiedono condizioni specifiche per rimanere vive.
2. Il sistema immunitario dell’ospite potrebbe rigettare le cellule staminali di un’altra persona, come spesso accade con i trapianti d’organo.
3. Trasformare le cellule staminali nel tessuto differenziato desiderato è difficile da controllare.

The first effort tried to replicate the chemical environment of stem cells and their differentiation. It had some limited successes but was not enough.

Like so many things in biotech, the deeper understanding of DNA & RNA allowed real progress.

By understanding what happens in real-time during differentiation, researchers can now control the differentiation process much better. They can also often take tissue cells from a patient and turn them back into un-differentiated stem cells.

Miglior campo terapeutico per le cellule staminali

Using stem cells for therapy makes the most sense, where healthy tissues are destroyed or made non-functional. In that context, bringing new cells will restore the failing organ to its normal capabilities. So, a few medical conditions are especially pertinent for stem cell therapies:

• Neurologico: molte malattie neurologiche derivano dalla morte di neuroni o nervi. Sostituire le cellule mancanti sembra una soluzione diretta, soprattutto grazie alla capacità dei neuroni di riconnettersi ai loro vicini, a seconda di ciò che è necessario.
• Cardiovascolare: la maggior parte delle volte, un infarto è un modo semplice per descrivere una parte del muscolo cardiaco che muore. Sostituire il tessuto morto con nuove cellule può aiutare a riparare il sistema cardiaco.
• Diabete di tipo 1: questa forma di diabete è causata dal sistema immunitario del corpo che distrugge le cellule produttrici di insulina. Sostituire queste cellule potrebbe curarla completamente.
• Insufficienza renale: se i tessuti renali stanno morendo, il corpo perde la capacità di filtrare le tossine. Riparare i reni in difficoltà può salvare la vita dei pazienti ed evitare sessioni di dialisi giornaliere di ore.
• Ustioni: la pelle bruciata in profondità deve essere sostituita, e la pelle appena cresciuta dalle cellule staminali è un’ottima candidata per guarire le ustioni
• Artrite: la maggior parte delle malattie articolari e delle articolazioni coinvolge parte della cartilagine che è stata distrutta o usurata. Rigenerarla riparerebbe ciò che è sbagliato.

Puoi anche leggere di più sulle terapie con cellule staminali in questo articolo NIH.

Le 5 migliori aziende di cellule staminali

Le aziende sono state ordinate per capitalizzazione di mercato al momento della stesura di questo articolo. Non rappresenta una preferenza o un consiglio finanziario.

1. Vertex

(VRTX )

Vertex is a leader in treating cystic fibrosis (4 approved medicines), which is now actively diversifying in new segments. The company is very R&D focused, with 70% of its operating expenses and 3/5^th of its employees working on R&D.

It is working closely with one of the leaders of gene editing, CRISPR Therapeutics. They are working on Sickle cell disease therapy, beta-thalassemia, and a Type-1 diabetes treatment.

Finally, they also have a treatment for pain in phase 3, a $4B market now dominated by opioids. Non-opioid alternatives would command premium pricing and be of interest to the medical profession at large.

Diabete: Il futuro focus di Vertex

Diabetes is by far the largest potential market for Vertex treatment (2.5 million people just in Europe and North America), and one without any treatment currently. The story of the diabetes treatment development is interesting, as this is clearly one major focus for Vertex. First, they acquired Semma in 2019. Then, they acquired ViaCyte, the competitor of Semma, in 2022. With ViaCyte already in a deal to co-develop their treatment with CRISPR therapeutic, this plays right with the strengths of Vertex. This was followed by an extra agreement between CRISPR and Vertex to keep improving the product and the approval for new clinical trials.

(Puoi leggere di più su questa tecnologia di trattamento del diabete in questo articolo dettagliato.)

Cystic fibrosis revenue is still growing steadily, from $3B in 2018 to $8.9B in 2022. The company registered an operating income of $635M in 2018, growing to $3.3B in 2022.

Vertex is by far the largest company in this list, but not so large that its stem cell therapy for diabetes would not completely change the company’s profile. The painkiller therapy in phase 3 could also move the company forward. So this might be the safest bet in the segment, as even if the stem cell therapies fail, the ongoing cystic fibrosis revenue stream would still make the company valuation reasonable.

2. Vericel

(VCEL )

I prodotti approvati di Vericel are focused on sports medicine and skin burn care.

Il trattamento di riparazione della cartilagine, MACI, sta aumentando in utilizzo ed è il leader nei prodotti di cartilagine restaurativa. Vengono testate ulteriori applicazioni, inclusi lo sviluppo di nuovi strumenti chirurgici per la somministrazione delle articolazioni del trattamento dell’azienda. Il mercato totale indirizzabile è stimato a 3 miliardi di dollari.

I trattamenti per le ustioni cutanee sono stati approvati nell’inverno 2022 e saranno commercializzati entro metà 2023. Il mercato totale indirizzabile è stimato a 600 milioni di dollari.

Fonte: Vericel

Vericel has $140M in cash in Q4 2022 and no debt. The company has been free cash flow positive since 2020.  It also expects a strong revenue growth in the next 3 years, at a yearly +20%.

Fonte: Vericel

Vericel is an interesting play for conservative investors looking for positive free cash flow and already-approved products. It has a solid financial position and operates in small but underpenetrated markets with several billion TAM in total.

Current earnings do not yet include the approved skin product, guaranteeing revenue growth in the coming 2-3 years.

 3. Sangamo Therapeutics

(SGMO )

The company is both a gene therapy and a stem cell company. Its 2 flagship programs are:

• Una terapia genica per l’emofilia A, in partnership with Pfizer, in clinical trial phase 3. Le domande di licenza biologica (BLA) is anticipated for the end of 2024.
  • The first results have been very positive, with ”Pfizer announced at Q4 earnings that this remains one of their most promising assets.’
• Una terapia genica per la malattia di Fabry, in phase 1/2.

Its 2 main stem cell therapies in development are:

• Una terapia con cellule staminali per rimuovere il rischio di rigetto del trapianto renale, in phase 1/2.
• Una terapia con cellule staminali per la malattia falciforme, in phase 1/2.

Il pipeline di ricerca also includes 5 more terapie con cellule staminali and 9 terapie di ingegneria genomica therapies.

Genome engineering could be used to solve neurological issues like prions disease (like mad cow disease), Huntington’s, etc. These programs are developed in partnership with Pfizer, Novartis, Biogen, and Takeda.

The company has invested heavily in proprietary manufacturing capacities.  At the end of 2022, Sangamo had $308M in cash. It is also receiving money from its partners for achieving clinical research milestones, with to date $815 million received. There are up to $3.6B in future milestone payments for cell therapies and potentially more from genome engineering.

Overall, Sangamo is an investment opportunity providing exposition to gene and cell therapy technologies. Its financial position is relatively solid, thanks to non-dilutive partnerships with some of the largest pharmaceutical firms.

4. BrainStorm Cell Therapeutics

(BCLI )

Brainstorm is focused on neurological diseases like Multiple Sclerosis, Alzheimer’s, Parkinson’s, Huntington’s Disease, or Autism Spectrum Disorder. The ALS treatment is the only one in phase 3, with hopes to get it approved quickly and preliminary results seemingly indicating it could help if used preventively.

Fonte: Brainstorm Cell

Brainstorm Cell uses its piattaforma tecnologica cellulare autologa (NurOwn®): the company takes the patient’s bone marrow cells and turns them into neurons and other nerve cells. This is a rather short process, taking only 7 days.

The company seems tight on cash, with just $4M left in cash on 31 dicembre 2022. This is unsettling when compared to $19M in a net loss in the latest quarter. So it is pretty clear it will need to raise more money, from equity and/or debt, to reach commercialization of its ALS treatment and pursue its other R&D programs.

Overall, Brainstorm seems an interesting company from a technology point of view, but with serious financial risks. Risk-taking investors might want to monitor the situation closely and buy after a stock decline caused by these immediate financial concerns.

5. Gamida Cell

(GMDA )

Gamida technology is focused sull’uso della nicotinamide (NAM) per migliorare il potenziale terapeutico delle cellule staminali.

The company currently has 2 treatments in development:

• Omidubicel: una terapia con cellule staminali che sostituisce le cellule cancerose nel midollo osseo con cellule sane. Le cellule staminali potenziate con NAM hanno dimostrato di aumentare il tasso di sopravvivenza, con un rischio più basso di infezioni pericolose.
  • La data PDUFA di Omidubicel è fissata al 1 maggio 2023, il che porta l’eventuale accettazione da parte della FDA a 6‑10 mesi dopo.
• GDA-201: una terapia a cellule bianche modificata per altri tumori, come il linfoma non Hodgkin, il mieloma, ecc…

Fonte: Gamida

Gamida treatment could save thousands of patients who cannot find marrow donors. It also has an already built and inspected manufacturing facility.

In the last quarter (Q3 2022), the company had $62M in current assets. Net loss for the quarter was $17M.  The company issued $14M worth of shares in Q3 2022. Some additional dilution is likely due to $72M in convertible debt.

Gamida is a more speculative bet, as it has fewer products in its pipeline and more risk of diluting existing shareholders. Therefore, a cheap enough valuation will be important for good investing returns.

Altre aziende coinvolte nella ricerca con cellule staminali

Some other companies are working on stem cell therapies but are so large that it is unclear how important to the company profile these therapies are.

AstraZeneca and Sanofi have advanced stem cell therapy programs and purchased companies in that segment. But with such giant pharmaceutical companies, the core business of blockbuster drugs will likely stay the main driver of future income.