The One about Investing in Biotechnology: A quick look at Abeona Therapeutics Inc. (NASDAQ Ticker Symbol: ABEO)

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I am not a financial advisor. This post is to provide information and not provide financial product advice. I discuss why I personally chose to invest in a stock, ETF, REIT, investment fund or cryptocurrency (which I have held for over a week) and also share information that is public about the following stock, ETF, REIT, investment fund or cryptocurrency and they are based on my own personal opinion.

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It is recommended that you should always consider visiting a financial advisor for independent financial advice before making investment decisions.

I do not work in the financial industry, so just because I write about it, doesn’t mean you should own it.  So, consult with a financial advisor and do your due diligence, RESEARCH!

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I think that for some people, they are surprised that while my portfolio is about positions that provide high dividends, I also own positions that currently provide no dividends but I do have high conviction for it beyond financial but for the benefits they provide towards society.

And some of these high convictions I do have, I have towards biotechnology companies.  If anything, having hope that these clinical trials will successfully help people who have been in pain and suffering from these disorders.  But if anything, to give hope.

One such company is Abeona Therapeutics (NASDAQ Ticker Symbol: ABEO), a fully-integrated gene and cell therapy company at the forefront of the rapid-advancing field of genetic medicine. While I have invested in ABEO, my high conviction is primarily towards a belief in that these clinical trials will become successful and help those who have been suffering from various disorders.

For one, Abeona Therapeutics do clinical trials for Phase 3 VIITAL of EB-101.  EB-101 is a gene-corrected cell therapy for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), a rare connective tissue disorder without an approved therapy.

RDEB is due to the lack of collagen protein in the skin and it makes skin fragile leading to blistering or skin loss at the slightest friction or knock and is progressive and painful.  The defect is caused by the COL7A1 gene, which can’t produce Type VII collagen, which is important in anchoring the dermal and epidermal layers of the skin.

In their study, according to the Abeona Therapeutics Inc., ” In this study, investigators will take two small biopsies of skin tissue, grow and multiply those cells, and then insert the correct type VII collagen gene. This may provide the cells with the ability to grow and develop normal skin. After cells’ gene transfer, the cells are further grown into a larger sheet that is then transplanted back onto the patient. After treatment, patients are followed for 26 weeks to assess safety and efficacy”.

The company has also done clinical trials for Sanfilippo Syndrome Type A (MPS IIIA) via a gene therapy of ABI-102 for younger, high functioning patients with MPS IIIA and “Sanfilippo Syndrome Type B (MPS IIIB) clinical trials of one-time therapy ABO-102 for patients with middle and advanced phases of MPS IIIA.

Sanfillipo Syndrome Type A (MPS IIIA) is a rare genetic metabolism disorder which prevents a childn’s body to break down carbs (sugars) which can lead to serious problems in the brain and nervous system.

While Sanfilippo Syndrom Type B (MPS IIIB) is a genetic disorder that makes the body unable to break down large sugar molecules called glycosaminoglycans (GAG). In this case, a GAG called heparan sulfate.  As the disease progresses, people can lose the ability to speak, walk and eat and the current life expectancy is 10 to 20 years.

The company even released a press release on new preclinical data supporting the potential of Cre-Mediated Dual AAV Vector Technology to enable delivery of large genes targeted for treatment of Stargardt Disease.

Both of these drug candidates for RDEB and for Sanfilippo Syndrome have received Orphan Drug Designation in the US and Europe, making governmental assistance available for their development. The company also received the FDA’s Rare Pediatric Disease Designation.

Stargardt Disease is a genetic eye disorder that causes progressive vision loss.  The disease causes vision loss during childhood or adolescence, although in some forms, vision loss may not be noticed until later in adulthood.  It is rare for people with the disease to become completely blind. While there is no cure or treatment for Stargardt Disease, the following findings were as follows:

“Autosomal recessive Stargardt disease is caused by mutations in the ABCA4 gene, preventing removal of toxic substances from photoreceptor cells that result in photoreceptor death and progressive vision loss,” said Linas Padegimas, Ph.D., Senior Director, Product Development at Abeona. “The preclinical data being presented at ARVO’s Annual Meeting provides compelling evidence that two independent AAV vectors utilizing Cre recombinase can efficiently reconstitute the ABCA4 gene, leading to full-size ABCA4 protein expression. Future studies that explore Cre-mediated ABCA4 gene reconstitution and lipofuscin clearance in vivo could provide additional findings to inform clinical development plans for a dual AAV vector approach as a potential treatment for Stargardt disease. In addition, our Cre-mediated dual AAV vector technology holds potential benefit for other indications that require delivery of larger genes that exceed the encapsidation capacity of AAV vectors.”

So, if anything, I have hope for Abeo Therapeutics Inc., in terms of its clinical trials and paving the way for hopefully something bigger and promising for people in the future.

Source: Schwab.com

As for the biotech stock investment, I look at the ABEO stock and while inexpensive, I know that the stock has been down but I believe it has potential. It’s best year was in 2017-2018 and the stock has hovered from $1.29-$3.00 for 2021.

I have read the bulls and the bears and I saw what Ram Selvaraju from H.C. Wainwright having reiterated a buy rating on Abeona Therapeutics (ABEO) with a price target of $8.00.

While I don’t know what the near future holds for ABEO, I did notice hedge fund manager Ken Griffin, chief of the investment firm Citadel, snapped up 1.846 million shares in Q4.

But for me, it’s pretty much having high conviction towards this company and its results on gene therapy but I believe in the company’s success, especially for the future. Also, as long as the company keeps its debt low and holding much more in net cash and strengthens its balance sheet over time, I’ll stay in for the long haul.

But as always, perform due diligence and do a lot of research before investing.  Talk to a financial advisor and find out if Abeona Therapeutics Inc. is a perfect investment for you.