5 Parasta kantasolu-yhtiötä sijoitettavaksi (elokuu 2026)

Kantasolun voima

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:

• Pluripotentit: kykenevät muuttumaan mihin tahansa mahdolliseen kudokseen.
• Multipotentit: kykenevät muuttumaan vain rajoitettuun määrään kudoksia ja solutyyppejä.

Lähde: 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.

Parantuminen kantasoluilla

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

1. Stem cells are rare in the body and require specific conditions to stay alive.
2. The host immune system might reject stem cells from someone else, as often happens with organ transplants.
3. Turning stem cells into the desired useful differentiated tissue is hard to control.

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.

Paras terapeuttinen alue kantasoluille

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:

• Neurologinen: monet neurologiset sairaudet johtuvat neuronien tai hermojen kuolemasta. Puuttuvan solun korvaaminen vaikuttaa suoravalta ratkaisulta, erityisesti neuronien kyvyn yhdistyä naapureihinsa, riippuen tarpeesta.
• Sydän- ja verisuonitautinen: useimmiten sydänkohtaus on yksinkertainen tapa kuvata osan sydänlihaksesta kuolevan. Kuolleen kudoksen korvaaminen uusilla soluilla voi auttaa korjaamaan sydämen järjestelmää.
• Tyypin 1 diabetes: tämä diabeteksen muoto johtuu kehon immuunijärjestelmän tuhoamista insuliinia tuottavista soluista. Näiden solujen korvaaminen voisi parantaa sen kokonaan.
• Munuaisten vajaatoiminta: jos munuaisten kudokset kuolevat, keho menettää kyvyn suodattaa myrkkyjä. Vajaatoimintaiset munuaiset korjaamalla voidaan pelastaa potilaiden henkiä ja välttää tuntikausisia päivittäisiä dialyysikertoja.
• Palovammat: syvälle palaneen ihon täytyy korvata, ja kantasoluista kasvatettu tuore iho on erittäin hyvä vaihtoehto palovammojen parantamiseen
• Artriitti: useimmat nivel- ja niveltaudit koskevat rustoa, joka on tuhoutunut tai kulunut. Sen uudelleenkasvu korjaisi ongelman.

Lue myös lisää kantasulahoidoista tässä NIH-artikkelissa.

Top 5 kantasolu-yhtiötä

The companies have been ordered by market capitalization at the time of the writing of this article. It does not represent a preference or financial advice.

1. Vertex

VRTX Hintakaavio

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 (CRSP ). 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.

Diabetes: Vertexin tulevaisuuden fokus

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.

(You can read more about this diabetes treatment technology in this detailed article.)

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 Hintakaavio

Vericelin hyväksytyt tuotteet are focused on sports medicine and skin burn care.

Kartonaan korjaushoito, MACI, is growing in use and is the leader in restorative cartilage products. More applications are being tested, including the development of new surgical tools for delivering the articulations of the company’s treatment. The total addressable market is estimated at $3B.

Ihon palovamahoidot have been approved in winter 2022 and will be commercialized by mid-2023. The total addressable market is estimated at $600M.

Lähde: 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%.

Lähde: 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 Hintakaavio

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

• Hemofilia A -geeniterapia, in partnership with Pfizer (PFE ), in clinical trial phase 3. The Biologics License Applications (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.’’
• Fabryn tautiin suunnattu geeniterapia, in phase 1/2.

Its 2 main stem cell therapies in development are:

• Kantasuluterapia, joka poistaa munuaisensiirron hylkimisriskin, in phase 1/2.
• Kantasuluterapia sirppisolutaudille, in phase 1/2.

Tutkimusputki also includes 5 more kantasuluterapioita and 9 genomin editointiterapiaa 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 companies.

4. BrainStorm Cell Therapeutics

BCLI Hintakaavio

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.

Lähde: Brainstorm Cell

Brainstorm Cell uses its autologinen soluteknologia-alusta (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. joulukuuta 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

Gamida technology is focused käyttämään nikotiiniamidia (NAM) kantasolujen terapeuttisen potentiaalin parantamiseen.

The company currently has 2 treatments in development:

• Omidubicel: a stem cell therapy replacing cancerous cells in bone marrow with healthy ones. NAM-enhanced stem cells have been shown to increase survival rate, with a lower risk of dangerous infections.
  • Omidubicelin PDUFA date is fixed at 1. toukokuuta 2023, which puts the possible acceptance by the FDA to 6-10 months later.
• GDA-201: a modified white cell therapy for other cancers, like non-Hodgkins lymphoma, myeloma, etc…

Lähde: 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.

Muut kantasoluja tutkaavat yritykset

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