Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:
So today I just want to do an initial assessment of a few of the top gene therapy companies and see what their fundamentals and technicals look like. I think that gene therapy is on the verge of introducing several blockbuster drugs over the next several years and those companies are expected to earn billions of dollars in profits. Obviously I want to be part of that as I intend to own shares in these companies.
- Replacing a mutated gene that causes disease with a healthy copy of the gene.
- Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
- Introducing a new gene into the body to help fight a disease.
So today I just want to do an initial assessment of a few of the top gene therapy companies and see what their fundamentals and technicals look like. I think that gene therapy is on the verge of introducing several blockbuster drugs over the next several years and those companies are expected to earn billions of dollars in profits. Obviously I want to be part of that as I intend to own shares in these companies.
Abeona Therapeutics Inc., a biopharmaceutical company, focuses on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. The company offers MuGard, a mucoadhesive oral wound rinse for mucositis, stomatitis, aphthous ulcers, and traumatic ulcers; and ProctiGard, a mucoadhesive oral wound rinse for the treatment of rectal mucositis and radiation proctitis. Its lead program is an adeno-associated virus (AAV)-based gene therapy for sanfilippo syndrome. The company develops ABO-101 for Mucopolysaccharidosis (MPS) III, a sanfilippo syndrome Type B; ABO-102 for MPS III, a sanfilippo syndrome Type A; ABO-201 for juvenile batten disease; ABO-301 for fanconi anemia; and clustered, regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated protein 9 AAV for rare blood diseases. It also develops PTB-101 salt diafiltration process (SDF) Alpha, a alpha1-proteinase inhibitor for chronic augmentation and maintenance therapy in adults with clinically evident emphysema due to severe deficiency of alpha1-proteinase inhibitor; and PTB-102 SDF IVIG, an intravenous immunoglobulin for autoimmune, infectious, and idiopathic diseases. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $17.05 1yr Target $20.00 Analysts 6 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 17.30% Yield 0.00% 1yr Tot Return 17.30% P/E --- PEG --- Beta 2.41 | EPS (ttm) $-0.60 EPS next yr $-0.78 Forward P/E --- EPS next 5yr --- 1yr Price Support --- Market Cap $682.85 Mil Revenues $0.80 Mil Earnings $-23.30 Mil Profit Margin 0.00% Quick Ratio 14.00 Current Ratio 14.00 Debt/Equity 0.00 | 1yr RevGR -14.42% 3yr RevGR -24.00% 5yr RevGR -13.54% 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -23.90% ROE -26.70% |
AveXis, Inc., a clinical-stage gene therapy company, engages in developing and commercializing treatments for patients suffering from rare and life-threatening neurological genetic diseases. Its initial product candidate is AVXS-101, a gene therapy product candidate that has completed Phase I clinical trial for the treatment of spinal muscular atrophy Type 1. The company also intends to identify, acquire, develop, and commercialize gene therapy product candidates for the treatment of other rare and life-threatening neurological genetic diseases. It has strategic collaboration and license agreements with Nationwide Children's Hospital; The Research Institute at Nationwide Children's Hospital; REGENXBIO Inc.; and Asklepios Biopharmaceutical, Inc. The company was formerly known as BioLife Cell Bank, Inc. and changed its name to AveXis, Inc. in January 2014. AveXis, Inc. was founded in 2010 and is headquartered in Bannockburn, Illinois.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $96.73 1yr Target $107.69 Analysts 13 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 11.33% Yield 0.00% 1yr Tot Return 11.33% P/E --- PEG --- Beta --- | EPS (ttm) $-4.93 EPS next yr $-5.46 Forward P/E --- EPS next 5yr 9.25% 1yr Price Support --- Market Cap $2.94 Bil Revenues --- Earnings $-134.20 Mil Profit Margin 0.00% Quick Ratio 15.60 Current Ratio 15.60 Debt/Equity 0.00 | 1yr RevGR --- 3yr RevGR --- 5yr RevGR --- 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -42.20% ROE -45.60% |
bluebird bio, Inc., a clinical-stage biotechnology company, focuses on developing transformative gene therapies for severe genetic diseases and cancer. Its product candidates include Lenti-D that is in phase II/III clinical studies for the treatment of cerebral adrenoleukodystrophy, a rare hereditary neurological disorder; and LentiGlobin, which is in four clinical studies for the treatment of transfusion-dependent beta-thalassemia, and severe sickle cell disease. The company’s lead product candidate is bb2121, a chimeric antigen receptor T cell product candidate that is in Phase I trial for the treatment of relapsed/refractory multiple myeloma. bluebird bio, Inc. has a strategic collaboration with Celgene Corporation to discover, develop, and commercialize disease-altering gene therapies in oncology; Kite Pharma, Inc. to develop and commercialize second generation T cell receptor (TCR) product candidates against an antigen related to certain cancers associated with the human papilloma virus; and Medigene AG for the research and development of TCR product candidates directed against approximately four antigens for the treatment of cancer indications. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was founded in 1992 and is headquartered in Cambridge, Massachusetts.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $137.35 1yr Target $116.47 Analysts 15 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain -15.21% Yield 0.00% 1yr Tot Return -15.21% P/E --- PEG --- Beta 2.04 | EPS (ttm) $-7.36 EPS next yr $-6.96 Forward P/E --- EPS next 5yr --- 1yr Price Support --- Market Cap $6.43 Bil Revenues $26.70 Mil Earnings $-288.00 Mil Profit Margin --- Quick Ratio 14.40 Current Ratio 14.40 Debt/Equity 0.13 | 1yr RevGR -56.29% 3yr RevGR -32.43% 5yr RevGR --- 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -25.20% ROE -32.20% |
BioMarin Pharmaceutical Inc. develops and commercializes pharmaceuticals for serious diseases and medical conditions in the United States, Europe, Latin America, and internationally. Its commercial products include Aldurazyme used for the treatment of patients with mucopolysaccharidosis I, a genetic disease; Kuvan, a proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU), an inherited metabolic disease; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with mucopolysaccharidosis VI; and Vimizim, an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder. The company also conducts clinical trials on several investigational product candidates for the treatment of various diseases, including Brineura for the treatment of late infantile neuronal ceroid lipofuscinosis, a form of Batten disease; pegvaliase, an enzyme substitution therapy for the treatment of PKU; vosoritide, a peptide therapeutic for the treatment of achondroplasia; BMN 270, an AAV VIII vector and Factor VIII gene therapy drug development candidate, for the treatment of hemophilia A; and BMN 250, a novel fusion of alpha-N-acetyglucosaminidase for the treatment of Sanfilippo B syndrome, or mucopolysaccharidosis type IIIB. The company serves specialty pharmacies and end-users, such as hospitals and foreign government agencies; and distributors and pharmaceutical wholesalers. BioMarin Pharmaceutical Inc. has a collaboration agreement with Genzyme Corporation. The company was founded in 1996 and is headquartered in San Rafael, California.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $93.07 1yr Target $111.57 Analysts 21 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 19.87% Yield 0.00% 1yr Tot Return 19.87% P/E --- PEG --- Beta 1.84 | EPS (ttm) $-1.02 EPS next yr $-0.25 Forward P/E --- EPS next 5yr 25.00% 1yr Price Support --- Market Cap $16.29 Bil Revenues $1.20 Bil Earnings $-174.60 Mil Profit Margin --- Quick Ratio 2.70 Current Ratio 3.90 Debt/Equity 0.24 | 1yr RevGR 25.53% 3yr RevGR 23.78% 5yr RevGR 20.40% 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -4.40% ROE -6.40% |
Spark Therapeutics, Inc. focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases. Its products include voretigene neparvovec, which is in Phase III clinical trial for the treatment of genetic blinding conditions called inherited retinal diseases caused by non sex-linked, autosomal recessive, or biallelic mutations in the RPE65 gene; and SPK-CHM that is in Phase I/II clinical trial for the treatment of choroideremia. The company’s products also comprise SPK-7001 that is in Phase 1/2 trial for choroideremia; SPK-9001, which is in Phase 1/2 trial for hemophilia B; and SPK-8011 that is in Phase 1/2 trial hemophilia A. In addition, its product candidates and development programs include SPK-FVIII program to treat hemophilia A; SPK-TPP1 program for the treatment of a form of Batten disease and Huntington's disease; RhoNova for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa; and SPK-LHON for treating Leber hereditary optic neuropathy, as well as preclinical programs in development for the treatment of rare genetic blinding conditions, hematologic disorders, and other neurodegenerative diseases. The company has collaboration agreement with Pfizer, Inc. for the development and commercialization of SPK-FIX product candidates in its gene therapy program for the treatment of hemophilia B. Spark Therapeutics, Inc. was founded in 2013 and is headquartered in Philadelphia, Pennsylvania.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $89.16 1yr Target $91.00 Analysts 14 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 2.06% Yield 0.00% 1yr Tot Return 2.06% P/E --- PEG --- Beta --- | EPS (ttm) $-6.38 EPS next yr $-5.01 Forward P/E --- EPS next 5yr 18.00% 1yr Price Support --- Market Cap $3.20 Bil Revenues $20.40 Mil Earnings $-196.00 Mil Profit Margin --- Quick Ratio 6.10 Current Ratio 6.10 Debt/Equity 0.01 | 1yr RevGR -8.53% 3yr RevGR --- 5yr RevGR --- 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -56.60% ROE -64.90% |
REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform. The company’s lead product candidate is RGX-314 for the treatment of wet age-related macular degeneration. It is also developing RGX-501, a product candidate for the treatment of homozygous familial hypercholesterolemia, which uses the AAV8 vector to deliver the human low-density lipoprotein receptor gene to liver cells; and RGX-111, a product candidate to treat the neurological symptoms of Mucopolysaccharidosis Type I that uses the AAV9 vector to deliver the human a-l-iduronidase gene to the central nervous system (CNS). In addition, the company engages in the development of RGX-121, a product candidate for the treatment of Mucopolysaccharidosis Type II, which uses the AAV9 vector to deliver the human iduronate-2-sulfatase gene to the CNS. Further, it licenses its NAV vectors to various other biotechnology companies. The company was formerly known as REGENX Biosciences, LLC and changed its name to REGENXBIO Inc. in September 2014. REGENXBIO Inc. was founded in 2008 and is headquartered in Rockville, Maryland.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $32.95 1yr Target $44.33 Analysts 3 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 34.53% Yield 0.00% 1yr Tot Return 34.53% P/E --- PEG --- Beta --- | EPS (ttm) $-2.72 EPS next yr $-3.06 Forward P/E --- EPS next 5yr --- 1yr Price Support --- Market Cap $991.80 Mil Revenues $8.80 Mil Earnings $-74.20 Mil Profit Margin --- Quick Ratio 14.50 Current Ratio 14.50 Debt/Equity 0.00 | 1yr RevGR -17.78% 3yr RevGR --- 5yr RevGR --- 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -36.30% ROE -38.70% |
uniQure N.V., a biopharmaceutical company, engages in the discovery, development, and commercialization of gene therapies in the Netherlands. The company’s principle programs include AMT-060, a gene therapy that has completed Phase I/II clinical trial for the treatment of hemophilia B; S100A1, a preclinical product candidate for the treatment of congestive heart failure; and AMT-130 for the treatment of huntington's disease. It also provides Glybera, a gene therapy product that has approved for the treatment of patients with lipoprotein lipase deficiency. The company has collaboration and license agreements with Bristol-Myers Squibb Company, 4D Molecular Therapeutics, Synpromics, and Chiesi Farmaceutici S.p.A. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $9.60 1yr Target $14.36 Analysts 5 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 49.58% Yield 0.00% 1yr Tot Return 49.58% P/E --- PEG --- Beta 0.45 | EPS (ttm) $-3.01 EPS next yr $-2.60 Forward P/E --- EPS next 5yr --- 1yr Price Support --- Market Cap $245.38 Mil Revenues $19.20 Mil Earnings $-76.10 Mil Profit Margin --- Quick Ratio --- Current Ratio 4.80 Debt/Equity 0.75 | 1yr RevGR --- 3yr RevGR --- 5yr RevGR --- 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -41.90% ROE -132.00% |
Voyager Therapeutics, Inc., a clinical-stage gene therapy company, focuses on the development of treatments for patients suffering from severe diseases of the central nervous system. The company’s lead clinical candidate is the VY-AADC01, which is in Phase Ib clinical trial for the treatment of advanced Parkinson's disease. Its preclinical programs comprise VY-SOD101 for a monogenic form of amyotrophic lateral sclerosis; VY-HTT01 for Huntington’s disease; VY-FXN01 for Friedreich's ataxia; VY-TAU01, Alzheimer’s disease; and VY-NAV01 for severe chronic pain. The company has strategic collaboration agreements with Genzyme Corporation and the University of Massachusetts. Voyager Therapeutics, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 1 October 2017 Price $20.59 1yr Target $28.00 Analysts 6 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 35.98% Yield 0.00% 1yr Tot Return 35.98% P/E --- PEG --- Beta --- | EPS (ttm) $-2.30 EPS next yr $-2.59 Forward P/E --- EPS next 5yr --- 1yr Price Support --- Market Cap $525.46 Mil Revenues $8.30 Mil Earnings $-59.20 Mil Profit Margin --- Quick Ratio 12.20 Current Ratio 12.20 Debt/Equity 0.00 | 1yr RevGR 44.01% 3yr RevGR --- 5yr RevGR --- 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -32.70% ROE -46,20% |
The Path Forward
Gene Therapy is an area of medicine that has become extremely interesting to me. In an area of genetic manipulation it may not be as sexy as gene editing but it may just be a treatment that is ahead of editing. It may also be complementary to gene editing and in some cases more effective. As an alternative, it will most likely produce a lot of effective treatments and cures for genetically damaged diseases.
As a result, I need to be involved in at least some of these companies. Today, however, I'm still trying to get my head wrapped around which of these companies is close to developing an approved product and which ones are simply in Phase 11/111 trials. So obviously additional research is needed.
At first glance BioMarin Pharmaceuticals seems the obvious one to own simply because it is increasing revenues and appears to be close to producing a profit within the next two years. That may be a great place to start but I'm sure there's more to these companies stories.
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