Category Archives: Gene therapy

A University of Delaware professor is receiving grant funding to develop a new therapy for people with low blood platelet counts and other blood disorders.

When patients dont have enough blood platelets, they cant form clots and this can sometimes be a lethal condition. These patients rely on platelet donations as a treatment, but those are often in short supply since they cannot be frozen and are only good for a few days.

A few years ago, UDs professor of Chemical and Biomolecular Engineering Terry Papoutsakis discovered a way to get patients to produce more blood platelets in their own bloodstream by introducing microparticles made from cultured stem cells.

These microparticles can be frozen to be used on a large scale. And Papoutsakis says they could also be used to treat genetic blood disorders like primary immune deficiencies.

To be able to use those particles for applications in gene therapy for a variety of blood diseasesa lot of genetic diseases or acquired diseases for patients, said Papoutsakis. So thats a difficult problem to deal with and we think this has great promise.

Papoutsakis is being awarded $250,000 through a partnership between University City Science Center and CSL Behring meant to find new biotherapies. He says the award took a few years to secure as a result of contract negotiations around intellectual property rights.

It took a little while to do it, but that is to be understood that it would, because of the potential that this might develop into something quite bigger than we currently see, he said.

Papoutsakis says he has already had some success testing his discovery on mice. He says the award will fund a continuation of those tests, moving the therapy closer to clinical trials on human patients.

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UD professor working to use gene therapy to treat blood disorders - Delaware First Media

IRVINE, Calif., Feb. 14, 2020 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the Company), a leading platform neurosurgery company, today announced a strategic agreement with PTC Therapeutics, Inc. (PTC). The scope of the agreement includes hardware, software, clinical case and market development services for gene therapy cases in the United States and Europe to support PTCs potential commercialization in gene therapy globally upon regulatory approval. In addition to the announcement of this agreement, ClearPoint Neuro today announces the following 2020 Outlook for the companys performance:

We have put a tremendous amount of thought and effort into redefining our Company over the past two years, commented Joe Burnett, President and CEO of ClearPoint Neuro, Inc. This has included the vision, the team, the partnerships, and even the name of our company which officially changed this week to ClearPoint Neuro from MRI Interventions. We are thrilled by the evolution our Company has already undergone and believe we have only scratched the surface of the significant potential ahead.

We have evolved to become two companies in one, continued Burnett. On one side, we are a platform medical device company, consistently delivering double digit growth, and continuing to expand our installed base of neurosurgery centers in the U.S. Every year more surgeons and more patients gain access to the ClearPoint system and compatible disposable devices. On the other side of the business, we are a gene therapy and biologics enabling company, providing navigation, drug delivery, and case support to more than 20 companies in this exciting and growing space. Here we currently support pre-clinical and clinical efforts, but we believe that we are on the precipice of potentially explosive growth as these therapies progress through the regulatory process toward commercial launch. We feel that our company represents both scale and purpose through a unique combination of predictable device growth and a potential biologics opportunity, all supported under a common team dedicated to treating the most debilitating neurological disorders.

ClearPoint Neuros revenue outlook for 2020 includes an expectation that approximately 33% of total revenue will be derived from biologics and drug delivery products and services, up from approximately 20% in 2019, and reflecting the growth of this part of the business.

When planning for the development and potential commercialization of gene therapy globally, safety, consistency and predictability are crucial constructs that all must be included, commented Marcio Souza, Chief Operating Officer of PTC Therapeutics and ClearPoint Neuro Board Member. Our agreement with ClearPoint Neuro is designed to provide standardization across all of our centers of excellence as we can maintain consistency in navigation, delivery and clinical support. The more variables we can control, the more successful we believe the outcomes for patients will be.

As part of our Companys duality in devices and biologics, we are thrilled to deepen our partnership with PTC, commented Jacqueline Keller, Vice President and Program Manager at ClearPoint Neuro for the PTC Partnership. This new agreement with PTC highlights a turn-key solution for our gene therapy partners, whereby our commercial organization, with deep relationships in the neurosurgery community, can take on the burden of sales and clinical activities in the surgical suite, and allow our partners who are commonly small-molecule focused companies to continue to prioritize their efforts with neurologists, patients and reimbursement administrators. With the capacity to support thousands of cases each year, our team plans to provide this service across multiple companies and indications in neuro.

About the Company

The Companys mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep-brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and installed in 60 active clinical sites in the United States. The Companys SmartFlow cannula is being used in partnership or evaluation with more than 20 individual biologics and drug delivery companies in various stages from preclinical research to late stage regulatory trials. To date, more than 3,500 cases have been performed and supported by the Companys field-based clinical specialist team which offers support and services for our partners. For more information, please visit http://www.clearpointneuro.com.

Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 regarding the planned offering. Additionally, all statements relating to any closing(s) of, and the amount or use of any proceeds from, the transactions described in this press release are considered to be forward-looking statements. Other forward-looking statements may be identified by the words guidance, plan, anticipate, believe, estimate, expect, intend, may, target, potential, will, would, could, should, continue, and similar expressions. Forward-looking statements are subject to risks and uncertainties, and the Companys actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of such risks and uncertainties, which include, without limitation, risks and uncertainties associated with market conditions and the satisfaction of closing conditions related to the transactions described in this press release. There can be no assurance that the parties will be able to complete the transactions described in this press release on the terms described herein or in a timely manner, if at all. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section of the Companys Annual Report on Form 10-K for the year ended December 31, 2018, and the Companys Quarterly Reports on Form 10-Q for the periods ended March 31, 2019, June 30, 2019 and September 30, 2019, all of which have been filed with the SEC, as well as the Companys Annual Report on Form 10-K for the year ended December 31, 2019, which the Company intends to file with the Securities and Exchange Commission on or before March 30, 2020. You are urged to carefully consider all such factors. Copies of these and other documents are available from the Company. The forward-looking statements contained herein represent the Companys views only as of the date of this press release the Company does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

For More Information

ClearPoint Neuro, Inc.Matt KrepsDarrow Associates Investor Relations(214) 597-8200mkreps@darrowir.com

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ClearPoint Neuro, Inc. Announces 2020 Revenue Outlook Ahead of Nasdaq Investor Presentation TodayNew US and European Partnership Agreement with PTC...

The lottery that began [recently] was not about money, or about choosing a school, or about obtaining a visa. It was about a childs life. In this case, the children selected would receive a drug that otherwise was not available.

The treatment, a gene therapy calledZolgensma, is designed for children like Wynter who have a neuromuscular disease called spinal muscular atrophy, or SMA.

The lotterywas devised by the drugs manufacturer, Novartis, to give families in those places a chance to get it through a novel form of compassionate use a way to get medications that have not been approved while they wait. Fifty doses are slotted to be given away for free in the first half of the year, with up to 100 total.

Ethicists and advocates have debated the merits and the design of the unusual arrangement. Parents said that it was uncomfortable to cast their childs fate into what felt like a sweepstakes a kind of bizarre Willy Wonka contest in which, as Maura Blair, a Canadian mother of a child with SMA put it, were talking about lives.

Zolgensma costs $2.1 million in the United States the worlds most expensive drug.

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Unconventional drug 'lottery' will hand out 50 doses of Zolgensma, world's most expensive gene therapy - Genetic Literacy Project

Dianomi Therapeutics is expanding the use of its patented drug delivery system to include gene therapies.

This comes after licensing a second set of intellectual property from WARF.

The Madison-based startup is developing a technology known as mineral-coated microparticles, which can stabilize and control the release of therapeutic drugs and improve their effect. A release shows the MCM technology shares a capacity for biological molecule storage with human bones and teeth.

The MCM technology was developed by UW-Madisons William Murphy, a professor of biomedical engineering and co-founder of Dianomi. He created the technology in hopes of improving upon artificial polymer-based drug delivery systems, which have more limited function.

Dianomi has demonstrated success in developing and optimizing MCM delivery for biologics and other small molecules, Murphy said in a release. I look forward to Dianomis expansion into the area of nucleic acid therapy, building upon the early results of our nucleic acid delivery in regenerative medicine applications.

Researchers have demonstrated favorable results with the MCM technology combined with nucleic acids, the release shows. Early animal studies have shown improvement in the delivery of these genetic materials, promising a potentially potent and sustained therapeutic effect.

The new gene therapy application builds on the companys first product, which delivers therapeutic drugs over time to patients with osteoarthritis. According to the release, company leaders plan to continue developing other applications for the companys drug delivery method. Targets include heart health, cancer treatment, vaccines, regenerative medicine, neuromuscular and spinal degeneration, and more.

This new suite of intellectual property expands the capability of Dianomis core technology into new indications and markets having significant commercial and clinical interest, said Martin Ostrowski, chief operations officer and general counsel of Dianomi.

The newly licensed IP from the Wisconsin Alumni Research Foundation includes issued U.S. patents and pending U.S. and international patent applications.

Dianomi Therapeutics is based in FORWARD Biolabs, part of UW-Madisons Forward BIO Institute. Murphy, Dianomis chief science officer, is the institutes director.

See an earlier story on the company: http://www.wisbusiness.com/2018/forward-bio-institute-awarded-5-million-nih-grant/

By Alex Moe

WisBusiness.com

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Dianomi Therapeutics expanding drug delivery system - Wisbusiness.com

If you want a stock market bet that's 74% likely to lose money, try investing in high-flying stock that sells for 100 times revenue or more. I call such investments "near insanity," since in the vast majority of cases these stocks crash back to earth.

How dizzying is 100 times revenue? Well, consider Tesla (NASDAQ:TSLA), a stock often criticized as overpriced. I happen to agree that it is, but Tesla sells for about six times revenue. That's a far cry from 100.

The price-sales ratio for the Standard & Poor's 500 now is 2.43, which is a record high. Over the years, a normal ratio has been around 1.5. The low, in March 2009, was 0.8.

Can a sky-high price-revenue multiple ever be justified? Sure, if the company has developed a blockbuster drug or breakthrough product, and hasn't started marketing it yet. But these are rare birds, much rarer than most people think.

Biotech hopes

This year, the ranks of stocks selling for 100 times earnings are dominated by early-stage biotech companies.

It's easy for people to imagine superb futures for young biotech stocks. They are using brand-new technology, and their products, if successful, will alleviate pain and suffering - or even save lives.

But consider a few of the exciting technologies of the past: auto stocks in the 1920s, radio stocks in the 1930s, television stocks in the 1950s. A plethora of companies entered these promising fields, and many of them bit the dust.

To make it to market, a drug has to successfully negotiate three levels of clinical trials. Most make it through Phase 1 (a basic safety check), but only 33% pass Phase 2 tests (efficacy), and only about 10% survive Phase 3 (larger-scale randomized double-blind studies).

It's probably beneficial to society that upstart biotech companies can fund important research by issuing stock to the public. But if you're an investor, realize that the odds don't favor you.

Adverum

One of the most extreme valuations belongs to Adverum Biotechnologies Inc. (NASDAQ:ADVM) of Redwood City, California, which is working on several forms of gene therapy for ocular and rare diseases. It sells for more than 3,000 times revenue.

According to its website, Adverum has one drug in Phase 1 clinical trials and two drugs in preclinical trials (animal testing).

For the moment, annual revenue is about $320,000 while the stock's market value is about $1 billion.

Homology Medicines

Selling for 1,345 times revenue is Homology Medicines Inc. (NASDAQ:FIXX), based in Bedford, Massachusetts, which seeks cures for nervous-system disorders.

In the past 12 months, Homology had revenue of about $660,000 and a loss of about $100 million. Market value? About $1 billion.

Julian Robertson (Trades, Portfolio), for many years the manager of The Tiger Fund, an extremely successful hedge fund, bought shares of Homology in the fourth quarter of 2018. Possibly influenced by Robertson's tacit endorsement, investors pushed Homology shares up strongly in the first quarter of 2019.

But Robertson bailed out in the first quarter, and since then the stock has descended from about $30 to about $19.

Forty-Seven

Forty-Seven Inc. (NASDAQ:FTSV) shares have risen 189% in the 12 months through Feb. 14. The Menlo Park, California-based company is working on ways to make people's natural immune systems function better in fighting cancers.

In layman's terms (as explained on the company's web site), many cancer cells give off "don't eat me" signals that stop the body's white cells (macrophages) from destroying them. Forty-Seven is working on drugs that disable these destructive signals. It has two drugs in Phase 2 trials.

The stock sells for 114 times revenue.

The record

Over the past 20 years, I've written 15 columns about stocks selling for 100 times revenue. The average 12-month return on the high flyers I warned about was a loss of 29.8%%.

That compares with an average gain of 9.8% for the Standard & Poor's 500.

Of the 70 stocks I've written about, 52 have declined. Hence, the 74% likelihood of losing money that I mentioned at the outset.

Bear in mind that performance figures for my column are theoretical and don't reflect actual trades. Their results shouldn't be confused with the performance of portfolios I manage for clients. And past performance doesn't predict future results.

Story continues

The stocks I maligned a year ago managed to post a 5.8% gain. Then again, the S&P 500 was up 21.8%.

Immunomedics Inc. (NASDAQ:IMMU) notched a 29% return, and Denali Therapeutics Inc. (NASDAQ:DNLI) gained 21%. However, Tellurian Inc. (NASDAQ:TELL) dropped 33%.

Disclosure: I have no positions, long or short, for myself of clients, in the stocks discussed in today's column.

John Dorfman is chairman of Dorfman Value Investments LLC in Newton Upper Falls, Massachusetts, and a syndicated columnist. His firm or clients may own or trade securities discussed in this column. He can be reached at jdorfman@dorfmanvalue.com.

This article first appeared on GuruFocus.

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3 Stocks That Are Way More Expensive Than Tesla - Yahoo Finance

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Research report explores the PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Size 2019 to 2025 - Instant Tech News

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (NASDAQ: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced new initial data from the first patient dosed in the investigational gene therapy program for cystinosis, showing improvements in early measures at three months compared to baseline. The company also unveiled new clinical data showcasing a sustained biomarker response in patients for up to 32 months after receiving the companys investigational gene therapy for Fabry disease across metrics including vector copy number (VCN), substrate levels and enzyme activity. Additionally, the company reported on the clinical debut of its platoTM gene therapy platform. These data showed improved enzyme activity, transduction efficiency and VCN in drug product manufactured using plato compared with drug product produced using the academic platform, as well as higher in vivo enzyme activity at one month in the first patient treated with plato, as compared to other patients treated using the academic platform. All these data will be presented today, during the 16th Annual WORLDSymposiumTM in Orlando, Fla.

We have now dosed 10 patients across three trials for two lysosomal disorders and were delighted with the data were seeing. We have followed six patients in our Fabry trial for more than a year and one for nearly three years, and they are consistently producing the functional enzyme that was missing as a consequence of their genetic disease, suggesting a potentially durable effect from a single dose, said Geoff MacKay, AVROBIOs president and CEO. Furthermore, we believe that early data from the first clinical application of plato support our decision to invest heavily from AVROBIO's earliest days in this state-of-the-art gene therapy platform. We believe these data collectively indicate that were making exciting progress toward our goal of freeing patients and families from the life-limiting symptoms and relentless progression of lysosomal disorders.

Three-month data from first patient in investigational AVR-RD-04 trial in cystinosisAVROBIO reported initial data from the first patient dosed in the investigator-sponsored Phase 1/2 trial of the companys AVR-RD-04 investigational gene therapy for cystinosis, a progressive disease marked by the accumulation of cystine crystals in cellular organelles known as lysosomes. Patients with cystinosis accumulate the amino acid cystine, which can lead to crystal formation in the lysosomes of cells, causing debilitating symptoms including corneal damage, difficulty breathing and kidney failure, often leading to a shortened lifespan. The current standard of care for cystinosis, a burdensome treatment regimen that can amount to dozens of pills a day, may not prevent overall progression of the disease.

As of the safety data cut-off date of Jan. 27, 2020, which was approximately three months following administration of the investigational gene therapy to the first patient in the AVR-RD-04 program, there have been no reports of safety events attributed to the investigational drug product. In addition, no serious adverse events (SAEs) have been reported as of the safety data cut-off date. Adverse events did not suggest any unexpected safety signals or trends.

Three months following administration of AVR-RD-04, the first patient had a VCN of 2.0. VCN measures the average number of copies of the lentiviral-vector inserted transgene integrated into the genome of a cell and can be used to help assess the durability of a gene therapy. Initial data on another biomarker show that the patients average granulocyte cystine level -- one of the trials primary endpoints -- decreased from 7.8 nmol half cystine/mg protein two weeks after cysteamine discontinuation, to 1.5 at three months post-gene therapy.

The ongoing open-label, single-arm Phase 1/2 clinical trial evaluating the safety and efficacy of AVR-RD-04 is sponsored by AVROBIOs academic collaborators at the University of California San Diego (UCSD), led by Stephanie Cherqui, Ph.D. The trial is actively enrolling up to six participants at UCSD.

Interim data continue to support potential first line use of AVR-RD-01 in Fabry diseaseFour patients have been dosed in the Phase 2 trial (FAB-201), and five patients in the Phase 1 investigator-led trial of AVR-RD-01 in Fabry disease.

VCN data continue to be stable at 32 months following AVR-RD-01 treatment for the first patient in the Phase 1 trial, suggesting successful engraftment, which is critical to the long-term success of investigational ex vivo lentiviral gene therapies. The VCN data trend was generally consistent across the seven other Phase 1 and Phase 2 trial participants out six to 24 months.

The first three AVR-RD-01 Phase 2 patients entered the study with minimal endogenous enzyme activity. At nine, 12 and 18 months after dosing, data from these three patients indicate sustained increased leukocyte and plasma enzyme activity, suggesting that they are now producing an endogenous supply of functional alpha-galactosidase (AGA) enzyme. This enzyme is essential for breaking down globotriaosylceramide (Gb3) in cells; without it, a toxic metabolite, lyso-Gb3, may accumulate, potentially causing cardiac and kidney damage and other symptoms.

For two Phase 2 patients, data indicate that their decreased plasma lyso-Gb3 levels, a key biomarker for monitoring Fabry disease, have been sustained below their baseline at six and 18 months after dosing. The third Phase 2 patient, a cardiac variant who does not have classic Fabry disease, did not show a decrease in plasma lyso-Gb3 levels, as expected. Cardiac and kidney function measures in the Phase 2 trial remained within normal range for patients who had available 12-month data.

As previously reported, a kidney biopsy taken at 12 months post-treatment for the first patient in the Phase 2 trial showed an 87-percent reduction in Gb3 inclusions per peritubular capillary. The company believes this data point, the primary efficacy endpoint for the Phase 2 trial, supports the potential of AVR-RD-01 to reduce Gb3 levels in tissue, including in the kidney.

In the Phase 1 trial of AVR-RD-01, four of the five patients had their plasma lyso-Gb3 levels reduced between 26 and 47 percent compared to their pre-treatment baseline levels. Data from the other patient in the trial, who remains off enzyme replacement therapy (ERT), through month six showed an initial decline and at month 12 showed a 23-percent increase in lyso-Gb3 levels, as compared to pre-treatment levels. This patients lyso-Gb3 levels remain within the range for the Fabry disease patients on ERT observed in this study.

Overall, three of the five Phase 1 patients have discontinued ERT and all three remain off ERT for six, 14 and 15 months.

As of the safety data cut-off date of Nov. 26, 2019, there have been no safety events attributed to AVR-RD-01 drug product in either the Phase 1 or Phase 2 trial. Through the safety data cut-off date, four SAEs have been reported in the FAB-201 trial and two SAEs in the Phase 1 trial. The fourth Phase 2 patient, who was dosed after the safety data cut-off date, has reported an SAE, which was not attributed to AVR-RD-01 and which subsequently resolved. Across both studies, each of the SAEs has been consistent with the conditioning regimen, stem cell mobilization, underlying disease or pre-existing conditions. Pre-existing low anti-AGA antibody titers have been detected in four patients in the Phase 1 trial and a transient low titer was observed but not detectable in subsequent measures in one patient in the Phase 2 trial.

The Phase 1 trial is fully enrolled. AVROBIO continues to actively enroll the Phase 2 trial in Australia, Canada and the U.S. The FAB-201 trial is an ongoing open-label, single-arm Phase 2 clinical trial evaluating the efficacy and safety of AVR-RD-01 in eight to 12 treatment-nave patients with Fabry disease.

Successful clinical debut of platoTM gene therapy platformAVROBIO also shared preliminary results from the first two patients to receive busulfan conditioning. Conditioning is an essential step in ex vivo lentiviral gene therapy designed to clear space in the bone marrow for the cells carrying the therapeutic transgene to engraft. The conditioning regimen developed as part of AVROBIOs plato platform includes therapeutic dose monitoring to assess how rapidly the individual patient metabolizes busulfan so physicians can adjust the dose as needed, with a goal of minimizing side effects while maximizing the potential of durable engraftment.

AVROBIO is implementing its precision dosing conditioning regimen across its company-sponsored clinical trials as part of the plato platform. The fourth patient in AVROBIOs Phase 2 Fabry trial received a precision dosing conditioning regimen with busulfan as part of the plato platform, while the first patient in the investigator-led cystinosis trial received busulfan but not as part of the plato platform.

These two patients both had rapid neutrophil and platelet count recovery, with a trajectory that was similar to the patients who enrolled earlier in the Fabry trials and who received a melphalan conditioning regimen. Side effects, which included nausea, mucositis, fever, rash and hair loss, developed eight to 10 days after dosing with busulfan and then resolved quickly.

The company also reported preliminary data from the first drug product produced using the plato gene therapy platform, which was used to dose the fourth patient in the Phase 2 Fabry trial (FAB-201). Early data indicate that enzyme activity and transduction efficiency for the drug product used to dose the fourth patient were 2.2 times higher than the mean of the drug product used to dose the first three patients in FAB-201. VCN for the drug product used to dose the fourth patient was 1.8 times higher than the mean of the drug product for the first three patients dosed in FAB-201. The drug product for the first three patients in FAB-201 was manufactured using a manual process first developed by AVROBIOs academic collaborators. The automated manufacturing embedded in plato leverages optimized processes developed at AVROBIO.

At one month following administration of the plato-produced investigational gene therapy for the fourth patient in the Phase 2 Fabry trial, initial data show the patients plasma enzyme activity level to be 4.0 times higher than the mean activity level of the first three patients in the Phase 2 Fabry trial at the same timepoint.

The investigational drug product used to dose the first patient in the AVR-RD-04 program for cystinosis, which included a four-plasmid vector but not platos automated manufacturing process, also showed increased performance in line with the increased performance recorded for the drug product in the Fabry trial. The investigational drug product and VCN assay are different for each trial.

We believe these data are an early, but exciting, validation of our decision to invest in technological innovation rather than build expensive bricks-and-mortar manufacturing facilities, said MacKay. The plato platform gives us control over the production and scaling of our investigational gene therapies through an efficient, automated manufacturing system that is designed to be deployed in standard contracted sites around the world. The four-plasmid vector, conditioning regimen with precision dosing and other elements of plato are designed to optimize the safety, potency and durability of our investigational lentiviral gene therapies.

About AVROBIOs ex vivo approach to gene therapyOur investigational ex vivo gene therapies start with the patients own stem cells. In the manufacturing facility, a lentiviral vector is used to insert a therapeutic gene designed to enable the patient to produce a functional supply of the protein they lack. These cells are then infused back into the patient, where they are expected to engraft in the bone marrow and produce generations of daughter cells, each containing the therapeutic gene. This approach is designed to drive durable production of the functional protein throughout the patients body, including hard-to-reach tissues such as the brain, muscle and bone. It is a distinguishing feature of this type of gene therapy that the corrected cells are expected to cross the blood-brain barrier and thereby potentially address symptoms originating in the central nervous system.

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the transgenes presence as the patients cells divide, which may improve the expected durability of the therapy and potentially enable dosing of pediatric patients, whose cells divide rapidly as they grow. Because the transgene is integrated ex vivo into patients stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the viral vector.

Analyst and investor event and webcast informationAVROBIO will host an analyst and investor event today, Monday, Feb. 10, 2020, in conjunction with the WORLDSymposiumTM, an annual scientific meeting dedicated to lysosomal disorders, in Orlando, FL. The presentation at the event will be webcast beginning at 7:00 p.m. ET. The webcast and accompanying slides will be available under Events and Presentations in the Investors & Media section of the companys website at http://www.avrobio.com. An archived webcast recording of the event will be available on the website for approximately 30 days.

About AVROBIOOur mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, and anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Presents Positive Initial Data for its Investigational Cystinosis Program and Plato TM Platform, as well as Positive Data Out to 32 Months for...

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Medically reviewed by Drugs.com. Last updated on Dec 19, 2019.

Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren't the only vectors that can be used to carry altered genes into your body's cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

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Gene therapy - Drugs.com

For Immediate Release: January 28, 2020

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patients cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.

In that spirit, today, the FDA is announcing the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, said FDA Commissioner Stephen M. Hahn, M.D. We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

One of the most important steps the FDA can take to support safe innovation in this field is to create policies that provide product developers with meaningful guidance to answer critical questions as they research and design their gene therapy products.

The six final guidances issued today provide the agencys recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas. The six guidance documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure for the development and manufacture of gene therapies. The agency is issuing this suite of documents to help advance the field of gene therapy while providing recommendations to help ensure that these innovative products meet the FDAs standards for safety and effectiveness.

The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality. In addition, the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs, such as questions about the durability of response, and these questions often cant be fully answered in pre-market trials of reasonable size and duration. For some gene therapy products, therefore, although they have met the FDAs standards for approval, we may need to accept some level of uncertainty around questions of the duration of the response at the time of marketing authorization. Effective tools for reliable post-market follow up, such as post-market clinical trials, are going to be key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

The draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations provides the FDAs proposed current thinking on an interpretation of sameness between gene therapy products for the purposes of obtaining orphan-drug designation and eligibility for orphan-drug exclusivity. The draft guidance focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease. As laid out in the draft guidance and our regulations, the agencys determination will consider the principal molecular structural features of the gene therapy products, which includes transgenes (the transferred gene) and vectors (the vehicle for delivering the transgene to a cell).

With the large volume of products currently being studied, gene therapy product developers have asked the agency important questions about orphan-drug designation incentives to develop products for rare diseases with very small patient populations. The draft guidance has potential positive implications both for product developers and patients by providing insight into the agencys most current thinking on the sameness of products, and thus, not discourage the development of multiple gene therapy products to treat the same disease or condition. For patients, this policy could help lead to the development and approval of multiple treatments, creating a more competitive market with choices. We encourage stakeholders to provide their comments.

In sum, these policy documents are representative of efforts to help advance product development in the field of gene therapy. We will continue to work with product innovators, sponsors, researchers, patients, and other stakeholders to help make the development and review of these products more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We also encourage developers of new gene therapy products to make full use of our expedited programs available for products intended to address unmet medical needs in the treatment of serious or life-threatening conditions. These programs include breakthrough therapy designation, regenerative medicine advanced therapy designation, and fast track designation, as well as priority review and accelerated approval. Developers should pursue these programs whenever possible to help bring the benefits of important advances to patients as soon as possible. We believe our work will help advance innovations in a way that assures their safety and effectiveness, provides new therapeutic choices to patients and providers and continues to build confidence in this novel and emerging area of medicine.

The FDA is an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov