The biotech industry has had a good start in 2019. The Zacks Biomed and Genetics industry has gained 16.1%, outperforming the broader S&P 500, which is up 15.3%, so far in 2019. The rise in merger and acquisitions (M&A) activity this year is one of the major driving forces behind this rally. The biotech industry is ranked 86 among the 256 Zacks industries.
Several large-cap pharma companies are facing patent expirations of their major drugs and pricing challenges in the U.S. market. This has led to consolidation through M&A in the pharma space. In a landmark deal, Bristol-Myers has offered to acquire big biotech, Celgene, for $74 billion. The large-cap companies are looking to acquire or partner with smaller biotech companies to boost their pipeline with newer technologies. Acquisitions, which are done generally by giving a premium on current price, and partnerships, which provide funds to smaller companies to support continued development of pipeline candidates, boost the stock price of the target companies.
A few of the M&A deals so far in 2019 involve companies with promising gene therapy candidates. Gene therapy, a new wave of innovation in pharma space, enables to mitigate the adverse effects of a malfunctioning disease-causing gene. These therapies have a different approach from traditional drug therapies as the underlying cause can be treated instead of the symptoms. These therapies generally target rare indications including hemophilia, Duchenne muscular dystrophy (DMD) and Parkinson's disease among others, which will likely lead to higher price realization. The gene therapy segment is expected to attract a lot more attention from pharma players going forward. Better efficacy achieved by gene therapies in clinical studies and likely rise in involvement of pharma companies may lead to higher M&A activity in the gene therapy segment.
In 2018, Novartis acquired AveXis for its gene therapy candidates and Celgene acquired Juno Therapeutics to add CAR T-cell therapy for cancer. In 2019, Roche and Biogen offered to buy Spark Therapeutics ONCE and Nightstar Therapeutics NITE, respectively. Both Spark and Nightstar are developing gene therapies targeting rare indications. Pfizer collaborated with innovative gene therapy developer Vivet Therapeutics, a privately-held company. There are several companies in the biotech industry, which are developing gene therapy candidates or shifting their focus to developing similar therapies. Looking at this trend, we expect some more deals targeting gene therapy makers.
We present four companies with promising gene therapy candidates in their pipelines. These companies may become acquisition targets going forward.
uniQure N.V. QURE)
The company is a promising player in the space. It is engaged in creating a pipeline of innovative gene therapies that have been developed both internally and through its collaboration, focused on cardiovascular diseases, with Bristol Myers-Squibb.
The companys lead candidate AMT-061, an experimental AAV5-based gene therapy incorporating the FIX-Padua variant, is being evaluated in the phase III HOPE-B pivotal study for the treatment of patients with severe and moderately severe hemophilia B. In January 2019, the company received clearance from the FDA to initiate clinical study for AMT-130 for the treatment of Huntingtons disease.
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uniQures stock has moved up 115.4% so far this year against the industrys decline. The company currently carries a Zacks Rank #3 (Hold). You can see the complete list of todays Zacks #1 Rank (Strong Buy) stocks here.
Audentes Therapeutics BOLD)
It is another biotechnology company with a pipeline of innovative gene therapy products for patients living with rare diseases. The company is currently conducting phase I/II studies for its lead product candidates, AT132,for the treatment of X-linked myotubular myopathy (XLMTM), and AT342 for the treatment of Crigler-Najjar syndrome. The company currently has two additional product candidates in its pipeline, AT845 for the treatment of Pompe disease and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT), which are likely to enter clinical development.
Audentes currently carries a Zacks Rank #3. Shares have gained 82.9% year to date.
REGENXBIO Inc. RGNX)
The company is a clinical-stage biotechnology company that focuses on the development, commercialization and licensing of recombinant adeno-associated virus gene therapy. The companys most advanced candidate, RGX-314, is being evaluated in a phase I/IIa for treating wet age-related macular degeneration (AMD). The company is planning to initiate a mid-stage study on the candidate for treating an additional chronic retinal condition in the second half of 2019. Apart from RGX-314, the company is also developing three other gene therapy candidates for treating certain rare neurological symptoms.
REGENXBIO currently carries a Zacks Rank #3. So far this year, shares have gained 41.3%.
Solid Biosciences Inc. SLDB)
The companys lead product candidate is SGT-001, a microdystrophin gene therapy that is in phase I/II clinical studies, intended to restore functional dystrophin protein expression in DMD patients.
Solid Biosciences currently carries a Zacks Rank #3.
Solid Biosciences Inc. Price
Solid Biosciences Inc. Price | Solid Biosciences Inc. Quote
Some other companies with gene therapy candidates in their pipeline include Sarepta Therapeutics SRPT and Ophthotech OPHT, which are developing treatments for DMD and retinal disorder, respectively.
Whats Ahead?
Investors should keep an eye on this space as potential approvals and data read-outs from late-stage studies on several candidates are likely to drive the related stocks.
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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportOphthotech Corporation (OPHT) : Free Stock Analysis ReportuniQure N.V. (QURE) : Free Stock Analysis ReportSpark Therapeutics, Inc. (ONCE) : Free Stock Analysis ReportREGENXBIO Inc. (RGNX) : Free Stock Analysis ReportSarepta Therapeutics, Inc. (SRPT) : Free Stock Analysis ReportNightstar Therapeutics PLC Sponsored ADR (NITE) : Free Stock Analysis ReportAudentes Therapeutics, Inc. (BOLD) : Free Stock Analysis ReportSolid Biosciences Inc. (SLDB) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research
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4 Gene Therapy Players Likely to Become Buyout Targets in 2019
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- Exopharm identifies two new lead programs as exosomes gain prominence in gene therapy - Stockhead - October 13th, 2022
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- AMPLIFYBIO ACQUIRES PACT PHARMA ASSETS TO ENHANCE CELL AND GENE THERAPY CHARACTERIZATION CAPABILITIES - PR Newswire - October 4th, 2022
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- Emulate Announces New Gene Therapy Application Enabling Accelerated Development of Treatments for Genetic Diseases with Organ-on-a-Chip Technology -... - September 25th, 2022
- CSL flexes gene therapy muscle with latest drug - Sydney Morning Herald - September 25th, 2022
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- AVROBIO Receives Rare Pediatric Disease Designation From FDA For First Gene Therapy In Development For Cy - Benzinga - September 25th, 2022
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- KSQ Therapeutics Announces Leadership and Board Additions - Business Wire - September 25th, 2022
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- Hopkinton bioscience company forms advisory board for cell and gene therapy - Worcester Business Journal - September 16th, 2022
- Gene Therapy Restores Some Vision in Colorblind Children - Pharmacy Times - September 16th, 2022
- Coave Therapeutics partners with IMN on gene therapy - Labiotech.eu - September 16th, 2022
- MeiraGTx's Industry-Leading Gene Therapy Manufacturing Facility in Shannon, Ireland Formally Introduced by Head of Irish Government -... - September 16th, 2022
- Bluebird CFO resigns ahead of gene therapy launch - BioPharma Dive - September 16th, 2022
- Enrollment completed in STARLIGHT Phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease - Ophthalmology Times - September 16th, 2022
- Global Cancer Gene Therapy Market to Reach $11.35 Billion by 2030 at a CAGR of 23.3% - PR Newswire - September 16th, 2022
- How WhiteLab Genomics is using AI to aid gene and cell therapy development - TechCrunch - September 16th, 2022
- CAR-T Cell Therapy Market Segmentation and Forecast Analysis up to 2027 - BioSpace - September 16th, 2022
- Cluster-busters against Cancer and a Potential New Gene Therapy for LCA - BioSpace - September 16th, 2022
- Ori Biotech and CTMC team up on cell therapy delivery - BioPharma-Reporter.com - September 16th, 2022
- Adverum (ADVM) Doses First Patient in the Mid-Stage wAMD Study - Zacks Investment Research - September 16th, 2022
- Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |... - ALS News Today - September 8th, 2022
- A new gene therapy based on antibody cells is about to be tested in humans - MIT Technology Review - September 8th, 2022
- Myrtelles rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European... - September 8th, 2022
- Sangamo presses ahead with Fabry disease gene therapy - BioPharma Dive - September 8th, 2022
- The gene therapy that could transform the lives of millions - ABC News - September 8th, 2022
- As Philly becomes a hub for life sciences, a new program will train workers for jobs in the field - The Philadelphia Inquirer - September 8th, 2022
- Mayflower BioVentures to Announce New Cell & Gene Therapy Companies within Months - BioSpace - September 8th, 2022
- Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market - Business Wire - September 8th, 2022