Category Archives: Gene Medicine

Each week, The Dailys Science & Tech section produces a roundup of the most exciting and influential research happening on campus or otherwise related to Stanford. Heres our digest for the week of Feb. 2 Feb. 8.

CRISPR-based cancer therapy shows promise

A new FDA-approved cancer therapy uses the gene-editing technology Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) to enhance T-cells, a type of immune cell, in order to better fight cancer, a study published on Feb. 6 in Science found.

Lets say normally, theres a T-cell thats involved in an allergic reaction to pollen, genetics and dermatology professor Howard Chang told Stanford Medicines blog SCOPE. We can use CRISPR to alter the cell so that it doesnt react to pollen anymore, and instead, only fights cancer.

The researchers goal was to introduce three gene edits to T-cells, then reintroduce edited T-cells back into the patient. The gene edits would rid T-cells of their natural receptors and increase their immune activity to actively fight cancer. After three months, researchers took edited T-cell samples from patients to analyze their molecular characteristics.

If you think of all of these edited T-cells like theyre in a horse race, analyzing these cells is like being able to see which horse wins the race, but also that horses speed, gait and all of the critical details that make that horse the best, Chang told Stanford Medicines blog SCOPE.

The findings suggest the therapy is safe, and further studies will need to be performed in future human clinical trials.

Industry-backed studies show bias favoring indoor tanning

Studies on indoor tanning that are financially backed by the tanning industry are more likely to promote benefits and dismiss risks compared to studies without financial support, an investigation published on Feb. 4 in the British Medical Journal found.

The association is quite striking, dermatology professor Eleni Linos told Stanford Medicine News. We need scientific data to be independent of industry influence. I am concerned that funding sources may influence the conclusions of these papers.

The researchers analyzed 691 journal articles referencing indoor tanning and found that 50 had industry backing. 78% of articles with industry backing portrayed indoor tanning in a positive light, compared to 4% of articles without industry backing.

This is the first study to examine conflict of interest in indoor tanning literature, and it echoes whats been said about the influence of the tobacco and sugar industries on science, Linos told Stanford Medicine News. Researchers, public health experts and members of the general public should be aware of and account for industry funding when assessing the evidence related to the risks and benefits of indoor tanning.

Immigrants who obtain legal status might still fear deportation

Immigrants might continue to fear deportation even after receiving documentation, a study published on Jan. 29 in Law & Society Review found.

Documentation is hardly a shield from deportation fears, sociology assistant professor Asad Asad told Stanford News. Documentation affords some protection from deportation, but it can also heighten fears since the bureaucracies that document immigrants have a greater perceived ability to surveil and expel them.

Between 2013 to 2015, he conducted extensive interviews with 50 undocumented and documented immigrants living in the Dallas metropolitan area to learn about their everyday lives.

Some undocumented migrants may be chilled out of legalization opportunities in an attempt to maintain a sense of invisibility to a system they view as primarily punitive, Asad told Stanford News. If fears of deportation lead immigrants to pass up rare opportunities for legal status in their search for invisibility from a system they view as unforgiving, they and their U.S.-citizen children may face restricted opportunities for promoting their long-term well-being in this country.

Contact Derek Chen at derekc8 at stanford.edu.

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Research Roundup: CRISPR-based cancer therapy, tanning studies, immigration and deportation - The Stanford Daily

Feb. 12, 2020 13:00 UTC

BRISBANE, Calif.--(BUSINESS WIRE)-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced the appointment of Dr. John Markels, an accomplished pharmaceutical executive with three decades of general management, manufacturing and technology experience at Merck, to the Sangamo Board of Directors.

"We are very pleased to welcome John to our Board," said Sandy Macrae, Chief Executive Officer of Sangamo. Johns manufacturing expertise and global general management and therapeutic area leadership experience will greatly benefit Sangamo as we build out our own in-house gene and cell therapy manufacturing capabilities and advance our innovative pipeline of genomic medicine product candidates toward registration and eventual commercialization.

Dr. Markels has over 30 years of leadership experience in the pharmaceutical industry. He currently serves as President of Global Vaccines at Merck, a role in which he leads an integrated team dedicated to discovery and development, supply and access, and global marketing and long-term strategy for the vaccines portfolio. Earlier roles at Merck included President, Latin America, as well as a long career in senior leadership positions in major regions worldwide in manufacturing technology, operations and strategy, business development, alliance management, and supply chain. Dr. Markels received his Ph.D. in chemical engineering from the University of California, Berkeley and his B.S. in chemical engineering from the University of Delaware.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and genome regulation. For more information about Sangamo, visit http://www.sangamo.com.

Forward Looking Statements

This press release contains or refers to forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding the potential benefits of cell therapy, the Company's ability to develop and commercialize product candidates to address genetic diseases with the Company's proprietary technologies and the timing of commencement or next stages of such programs and the anticipated benefits therefrom. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 1, 2019 and Sangamo's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 that it filed on November 6, 2019. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200212005263/en/

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Sangamo Therapeutics Appoints John Markels to Its Board of Directors - BioSpace

Predictive Technology Group Inc (OTCMKTS:PRED) just announced that it and Atrin Pharmaceuticals LLC are entering into a collaboration agreement to develop molecular diagnostic tools to facilitate improved selection of cancer patients who would most benefit from treatment with DNA Damage and Response (DDR) inhibitors, including Atrins and other small molecule ATR inhibitors.

According to the release, Atrinand Predictive will jointly utilize Predictive Laboratories state-of-the-artsequencing capabilities and genomics expertise to identify cancer patients withspecific molecular markers that predict the level of clinical response toAtrins, and other, targeted therapies. This is intended to improve patientoutcomes as well as improve Atrins ability to successfully progress itsproduct pipeline, and upon commercialization, improve on the treatments forwomen with cancer.

Predictive Technology Group Inc (OTCMKTS:PRED) promulgates itself as a company that, together with its subsidiaries, develops and commercializes discoveries and technologies involved in novel molecular diagnostic and pharmaceutical therapeutic/human cells, tissues, and human cellular and tissue-based products (HCT/Ps).

The company was formerly knownas Global Enterprises Group, Inc. and changed its name to Predictive TechnologyGroup, Inc. in July 2015. Predictive Technology Group, Inc. was founded in 2005and is headquartered in Salt Lake City, Utah.

The company operates through two segments, Regenerative Medicine Products and HCT/Ps, and Diagnostics and Therapeutics. It offers ARTguide, a genetic diagnostic and prognostic test for women experiencing infertility as a result of endometriosis and other health concerns; and regenerative medicine products, including AmnioCyteT, AmnioCyte PlusT, PolyCyteT, and CoreCyteT.

According to companymaterials, Predictive Technology Group aims to revolutionize patient carethrough predictive data analytics, novel gene-based diagnostics and companiontherapeutics through its subsidiaries Predictive Therapeutics, PredictiveBiotech, and Predictive Laboratories. These subsidiaries are focused onendometriosis, scoliosis, degenerative disc disease and human cell and tissueproducts. The subsidiaries use genetic and other information as cornerstones inthe development of new diagnostics that assess a persons risk of illness andtherapeutic products designed to identify, prevent and treat diseases moreeffectively.

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As noted above, PRED just announced that it and Atrin Pharmaceuticals LLC are entering into a collaboration agreement to develop molecular diagnostic tools to facilitate improved selection of cancer patients who would most benefit from treatment with DNA Damage and Response (DDR) inhibitors, including Atrins and other small molecule ATR inhibitors.

Even in light of this news, PRED has had a rough past week of trading action, with shares sinking something like -2% in that time. That said, chart support is nearby and we may be in the process of constructing a nice setup for some movement back the other way. Moreover, the stock has seen interest climb, with an increase in recent trading volume of 12% beyond what we have been seeing over the larger time frame.

We are very pleased to workwith Atrin Pharmaceuticals, a recognized leader in the development ofanti-cancer therapeutics targeting DDR, said Bradley Robinson, president andchief executive officer of Predictive Technology Group. We see an opportunityto develop a precision medicine approach to address unmet medical needs bycombining our state-of-the-art sequencing capabilities, genomics expertise andcompanion diagnostics with Atrins targeted therapeutics. This collaboration isconsistent with our vision of building a leading womens health platform, andwe look forward to working together on this important initiative.

Earning a current market capvalue of $252M, PRED has a stash ($842K) ofcash on the books, which compares with about $8.6M in total currentliabilities. PRED is pulling in trailing 12-month revenues of $43.7M. Inaddition, the company is seeing major top-line growth, with y/y quarterlyrevenues growing at 2.4%. You can bet we will update this one again as newinformation comes into view. Sign-up forcontinuing coverage on shares of $PRED stock, as well as other hot stock picks,get our free newsletter today and get our next breakout pick!

Disclosure: we hold no position in $PRED, either long orshort, and we have not been compensated for this article.

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Targeting gene linked to Alzheimers may reduce dementia risk in Parkinsons

Clumps of the Parkinsons protein alpha-synuclein (red) are visible inside neurons (green) in the brain of a mouse. Researchers at Washington University School of Medicine in St. Louis have discovered that the genetic variant APOE4 long linked to dementia spurs the spread of harmful clumps of Parkinsons proteins through the brain. The findings suggest that therapies that target APOE might reduce the risk of dementia for people with Parkinsons disease.

Dementia is one of the most debilitating consequences of Parkinsons disease, a progressive neurological condition characterized by tremors, stiffness, slow movement and impaired balance. Eighty percent of people with Parkinsons develop dementia within 20 years of the diagnosis, and patients who carry a particular variant of the gene APOEare at especially high risk.

In new research, scientists at Washington University School of Medicine in St. Louis have found a clue to the link between Parkinsons, APOEand dementia. They discovered that harmful Parkinsons proteins spread more rapidly through the brains of mice that have the high-risk variant of APOE, and that memory and thinking skills deteriorate faster in people with Parkinsons who carry the variant. The findings, published Feb. 5 in Science Translational Medicine, could lead to therapies targeting APOEto slow or prevent cognitive decline in people with Parkinsons.

Dementia takes a huge toll on people with Parkinsons and their caregivers, said Albert (Gus) Davis, MD, PhD, an assistant professor of neurology and the studys lead author. The development of dementia is often what determines whether someone with Parkinsons is able to remain in their home or has to go into a nursing home.

An estimated 930,000 people in the U.S. live with Parkinsons. The disease is thought to be caused by toxic clumps of a protein called alpha-synuclein that build up in a part of the brain devoted to movement. The clumps damage and can kill brain cells.

Cognitive problems tend to arise many years after the motor symptoms. The protein clusters implicated in movement problems also are linked with dementia, but how this happens is not clear. Davis and his colleagues including senior author David Holtzman, MD, the Andrew B. and Gretchen P. Jones Professor and head of theDepartment of Neurology saw a clue in the risky nature of APOE.

A variant of APOE known as APOE4 raises the risk of Alzheimers disease threefold to fivefold. Like Parkinsons, Alzheimers is a neurodegenerative condition caused by the spread of toxic protein clusters throughout the brain, although some of the proteins involved are different. APOE4 increases the chance of Alzheimers dementia partly because it spurs Alzheimers proteins to collect into clumps that injure the brain. The researchers suspected that APOE4 similarly triggers the growth of toxic clusters of Parkinsons proteins.

Studying mice with a form of alpha-synuclein prone to clumping, Davis, Holtzman and colleagues genetically modified the mice to carry human variants of APOE APOE2, APOE3 or APOE4 or no APOE at all.

The researchers found that APOE4 mice had more alpha-synuclein clusters than APOE3 orAPOE2mice. Further experiments showed that the clumps spread more widely in APOE4 mice as well. Together, the findings showed that APOE4 was directly involved in exacerbating signs of disease in the mices brains.

What really stood out is how much less affected the APOE2 mice were than the others, Davis said. It actually may have a protective effect, and we are investigating this now. If we do find that APOE2 is protective, we might be able to use that information to design therapies to reduce the risk of dementia.

To study the effect of APOEvariants on dementia in people with Parkinsons, the researchers analyzed publicly available data from three separate sets of people with Parkinsons. Two of the cohorts one from the Parkinsons Progression Markers Initiative, with 251 patients, and the other from the Washington University Movement Disorders Center, with 170 patients had been followed for several years. In both cohorts, cognitive skills declined faster in people with APOE4 than in those with APOE3. People with two copies of APOE2 are very rare, but none of the three patients in the group with two copies of APOE2showed any cognitive decline over the period of the study.

The third cohort, fromthe NeuroGenetics Research Consortium, was made up of 1,030 people with Parkinsons whose cognitive skills had been evaluated just once. The researchers found that people with APOE4 in the cohorthad developed cognitive problems at a younger age and had more severe cognitive deficits at the time they were evaluated than people with APOE3 or APOE2.

Parkinsons is the most common, but there are other, rarer diseases that also are caused by alpha-synuclein aggregation and also have very limited treatment options, Davis said. Targeting APOE with therapeutics might be a way to change the course of such diseases.

APOE doesnt affect the overall risk of developing Parkinsons or how quickly movement symptoms worsen, so an APOE-targeted therapy might stave off dementia without doing anything for the other symptoms. Even so, it could be beneficial, Davis said.

Once people with Parkinsons develop dementia, the financial and emotional costs to them and their families are just enormous, Davis said. If we can reduce their risk of dementia, we could dramatically improve their quality of life.

Davis AA, Inman CE, Wargel ZM, Dube U, Freeberg BM, Galluppi A, Haines JN, Dhavale DD, Miller R, Choudhury FA, Sullivan PM, Cruchaga C, Perlmutter JS, Ulrich JD, Benitez BA, Kotzbauer PT, Holtzman DM. APOE Genotype Regulates Pathology and Disease Progression in Synucleinopathy. Science Translational Medicine. Feb. 5, 2020. DOI: 10.1126/scitranslmed.aay3069

This work was supported by an American Academy of Neurology/American Brain Foundation (Clinical Research Training Fellowship); the BrightFocus Foundation; the Mary E. Groff Charitable Trust; the Dobbins Family Fund; the Foundation for Barnes-Jewish Hospital (Elliot Stein Family Fund); the Riney Foundation; the American Parkinson Disease Association; the Greater St. Louis Chapter of the American Parkinson Disease Association; The JPB Foundation; and the National Institutes of Health (NIH), grant numbers K08NS101118, R01AG044546, RF1AG053303, RF1AG058501, U01AG052411, U01AG058922, NS075321, NS097799, R01NS090934 and R01AG047644.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Gene ID'd as potential therapeutic target for dementia in Parkinson's - Washington University School of Medicine in St. Louis

DetailsCategory: DNA RNA and CellsPublished on Sunday, 09 February 2020 11:42Hits: 258

-- Robust efficacy with evidence of a dose response ---- 6/6 patients rescue-injection-free in cohort 1, with 3 patients at 52 weeks ---- 4/6 patients rescue-injection-free in cohort 2 (lower dose) at 24 weeks --

REDWOOD CITY, CA, USA I February 08, 2020 IAdverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced new interim clinical data from the OPTIC Phase 1 dose-ranging clinical trial of ADVM-022 intravitreal injection gene therapy. OPTIC includes treatment-experienced patients with wet age-related macular degeneration (AMD). The data are being presented today by David S. Boyer, M.D., senior partner, Retina-Vitreous Associates Medical Group and adjunct clinical professor of ophthalmology with the University of Southern California/Keck School of Medicine in Los Angeles, at the Angiogenesis, Exudation, and Degeneration 2020 Annual Meeting in Miami.

A copy of the presentation is available on the Adverum corporate website under Events and Presentations in the Investors section.

For the first time, data are being presented from patients in cohort 2 (n=6) at 24 weeks following treatment with a single intravitreal injection of a three-fold lower dose of ADVM-022 (2 x 10^11 vg/eye) compared to the cohort 1 dose (6 x 10^11 vg/eye).New data as detailed in the table below include:

OPTIC Phase 1 Clinical Trial Data:

I am very encouraged that this difficult-to-treat patient population enrolled in OPTIC is maintaining vision and anatomical improvements for an extended period of time, said David S. Boyer, M.D., senior partner, Retina-Vitreous Associates Medical Group and adjunct clinical professor of ophthalmology with the University of Southern California/Keck School of Medicine in Los Angeles, California. Additionally, ADVM-022continues to be safe and well tolerated, with ocular inflammation that is manageable with steroid eye drops. Patients with wet AMD and their caregivers carry a significant treatment burden from the current standard-of-care anti-VEGF injections, and real-world vision outcomes are suboptimal due to undertreatment. ADVM-022 as a one-time intravitreal injection therapy could transform the treatment paradigm for patients and their caregivers.

Aaron Osborne, MBBS, chief medical officer of Adverum, added, ADVM-022 has demonstrated a robust efficacy signal and evidence of a dose response in the OPTIC Phase 1 trial with data from 12 patients and two doses now available. Patients in cohort 2 received a three-fold lower dose of ADVM-022 than in cohort 1, and 4 of 6 of these patients are rescue injection-free through 24 weeks, whilst all 6 patients in cohort 1 remain rescue free with a median follow up of 50 weeks. OPTIC is progressing well, with the key objectives for cohorts 3 and 4 being to further evaluate dose response and to assess a 6-week prophylactic course of steroid eye drops instead of the 13-day oral steroid prophylaxis used in cohorts 1 and 2. We look forward to presenting clinical data from all four cohorts of OPTIC during this important year in the clinic for our novel gene therapy, ADVM-022.

KOL Discussion Tomorrow:In addition, Adverum will host an event with expert retinal specialists to discuss the OPTIC data presented at Angiogenesis and the potential opportunity for ADVM-022. The discussion will be held on Sunday, February 9, 2020 beginning at 10:00 am EST. The event will be webcast live from Adverums website at http://www.adverum.com in the Investors section under the Events and Presentations page. A replay of the webcast will be archived and available for replay following the event. A copy of the slide presentation will also available on the Adverum corporate website under Events and Presentations in the Investors section.

About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMDThe multi-center, open-label, Phase 1, dose-ranging trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. In cohort 1, patients (n=6) received ADVM-022 at a higher dose of 6 x 10^11 vg/eye and in cohort 2, patients (n=6) received ADVM-022 at a lower dose of 2 x 10^11 vg/eye. In cohort 3, patients (n=9) also are receiving a dose of 2 x 10^11 vg/eye and in cohort 4, patients (n=9) will receive a dose of 6 x 10^11 vg/eye. Patients in cohorts 3 and 4 will receive prophylactic steroid eye drops instead of oral steroids which were used in cohorts 1 and 2. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as mean number of anti-VEGF rescue injections and percentage of patients needing anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.

Eight leading retinal centers acrossthe United States(U.S.) are participating in the OPTIC Phase 1 trial for ADVM-022. For more information on the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD, please visithttps://clinicaltrials.gov/ct2/show/NCT03748784.

About ADVM-022 Gene TherapyADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for wet AMD and diabetic retinopathy patients.

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for ADVM-022 for the treatment of this disease.

Adverum is currently evaluating ADVM-022 in the OPTIC study, a Phase 1 clinical trial in patients 50 years and older with wet AMD. Additionally, Adverum plans to submit an Investigational New Drug Application for ADVM-022 for the treatment of diabetic retinopathy to the U.S. Food and Drug Administration in the first half of 2020.

About Wet Age-related Macular Degeneration (AMD)Age-related macular degeneration (AMD) is a progressive disease affecting the macula, the region of the retina at the back of the eye responsible for central vision. In patients with wet AMD, an aggressive form of AMD, abnormal blood vessels grow underneath and into the retina. These abnormal blood vessels leak fluid and blood into and beneath the retina, causing vision loss.

Wet AMD is a leading cause of vision loss in patients over 60 years of age, with a prevalence of approximately 1.2 million individuals in the U.S. and 3 million worldwide. The incidence of new cases of wet AMD in the U.S. is approximately 150,000 to 200,000 annually, and this number is expected to grow significantly as the countrys population ages.

The current standard-of-care therapy for wet AMD is anti-VEGF intravitreal injections. These are effective but typically require eye injections every 4-12 weeks in order to maintain vision. Compliance with this regimen can be difficult for patients, caregivers, and healthcare systems, leading to undertreatment and resulting in loss of vision.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration. For more information, please visit http://www.adverum.com.

SOURCE: Adverum Biotechnologies

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Adverum Biotechnologies Reports New Interim Data from Cohorts 1 and 2 of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD at...

Spurred by promising clinical results in an important trial, each of the three major CRISPR stocks had a great performance in the second half of 2019. Unfortunately, they didn't keep the momentum going in the first month of 2020.

Shares of Intellia Therapeutics (NASDAQ:NTLA) fell 18.8% in January, according to data provided by S&P Global Market Intelligence. That was followed by a 14.7% loss for shares of CRISPR Therapeutics (NASDAQ:CRSP) and a 10.7% tumble for shares of Editas Medicine (NASDAQ:EDIT).

While each has recovered some ground in the first week of February, this trio of pharma stocks is no stranger to volatility. Investors should probably expect that to continue as clinical programs advance in 2020.

Image source: Getty Images.

In November, CRISPR Therapeutics reported data for the first two individuals in the trial, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), treated with its lead drug candidate CTX001. Both enjoyed significant benefits in their standard of living, which investors interpreted as a sign that CRISPR gene editing might actually live up to the hype.

That fueled annual gains of 113% for CRISPR Therapeutics last year. While Editas Medicine and Intellia Therapeutics gained only 30% and 7%, respectively, each had been sitting at a year-to-date loss in October.

What relevance does that have for the tumbles taken in January? First, it's not unusual for stocks to regress to the mean. Stocks that are red hot eventually cool off, while those that tumble without good reason eventually recover some ground.

Second, and the more important consideration for investors, is that the early stage results for CTX001 mean relatively little for the industry's pipeline of CRISPR-based gene editing drug candidates.

Consider that CTX001 is an ex vivo tool. Researchers harvest bone marrow from patients, extract specific types of stem cells, and engineer those with CTX001. The engineered stem cells are then grown in the lab before being reinjected into the patient.

Many other CRISPR-based drug candidates are designed as in vivo tools. That means the gene editing payloads are designed to engineer a patient's DNA while inside the body. An in vivo approach is inherently more complex and will be more difficult to control compared to an ex vivo approach.

Put another way, investors cannot take the promising, early stage results from CTX001 and extrapolate it broadly across all first-generation CRISPR tools. Wall Street certainly isn't, if the correlation between technical approach and stock performance is any guide.

Consider that the two most advanced drug candidates from CRISPR Therapeutics rely on ex vivo engineering. By contrast, the lead drug candidate from Editas Medicine relies on in vivo methods.

The lead pipeline asset from Intellia Therapeutics is also an in vivo tool, though unlike the lead assets from its peers, it has yet to advance to clinical trials.

Investors should expect 2020 to be a busy year for these CRISPR stocks. CRISPR Therapeutics will have more clinical data from CTX001 and the first set of data for its lead oncology asset CTX110.

Similarly, Editas Medicine should have results for EDIT101 and progress additional assets, while Intellia Therapeutics is preparing to finally enter the clinic with NTLA-2001 in the second half of the year.

Investors cannot know if the next batch of results will be as rosy as the initial data for CTX001, but they can probably expect another year of volatile stock movements.

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Here's Why CRISPR Stocks Fell in January - The Motley Fool