Category Archives: Genetic Therapy

Doctors told a medical conference in Chicago Monday theyve had unprecedented success with a new cell and gene therapy for multiple myeloma, a blood cancer thats on the rise.

Though its still early and the study was small just 35 people every single patient responded. And all but two were in some level of remission within two months.

In a second study of nearly two dozen patients, everyone above a certain dose responded.

Experts at an American Society of Clinical Oncology conference at McCormick Place, where the results were announced Monday, say its a first for multiple myeloma and rare for any cancer treatment to have such success.

Dr. J. Leonard Lichtenfeld. | American Cancer Society

Chemotherapy typically helps 10 to 30 percent of patients. Immune system drugs often do somewhat better 35 to 40 percent, at best. And some gene-targeting drugs have had 70 to 80 percent success.

But you dont get to 100, said Dr. J. Leonard Lichtenfeld, deputy chief medical officer of the American Cancer Society.

These are impressive results but time will tell if they last,Lichtenfeld said.

Multiple myeloma affects plasma cells, which make antibodies to fight infection. More than 30,000 cases occur each year in the United States, more than 115,000 worldwide. Its the second-fastest growing cancer for men and third-fastest for women, rising 2 to 3 percent per year, according to the National Cancer Institute. About 60,000 to 70,000 Americans have it now.

Nine new drugs have been approved for it since 2000. But theyre not cures. Only about half of U.S. patients live five years after diagnosis.

With cell therapy, I cant say we may get a cure, but at least we bring hope of that possibility, said Dr. Frank Fan, chief scientific officer of Nanjing Legend Biotech, a Chinese company that tested the treatment with doctors at Xian Jiaotong University.

The treatment, called CAR-T therapy , involves filtering a patients blood to remove immune system soldiers called T cells. These are altered in a lab to contain a gene that targets cancer, then given back to the patient intravenously.

Doctors call it a living drug a one-time treatment to permanently alter cells that multiply in the body into an army to fight cancer. Its shown promise against some leukemias and lymphomas, but this is a new type being tried for multiple myeloma, in patients whose cancer worsened despite many other treatments.

In the Chinese study, 19 of 35 patients are long enough past treatment to judge whether they are in complete remission, and 14 are. The other five had at least a partial remission, with their cancer greatly diminished. Some are more than a year past treatment with no sign of disease.

Most patients had a group of side effects common with this treatment, including fever, low blood pressure and trouble breathing. Only two cases were severe, and all were treatable and temporary, doctors said.

The second study was done in the United States by Bluebird Bio and Celgene, using a cell treatment developed by the National Cancer Institute. It tested four different dose levels of cells in a total of 21 patients. Eighteen are long enough from treatment to judge effectiveness, and all 15 who got an adequate amount of cells had a response. Four have reached full remission so far, and some are more than a year past treatment.

Dr. Kenneth Anderson. | Dana-Farber Cancer Institute

The results are very remarkable not just for how many responded but also for how well, said Dr. Kenneth Anderson of the Dana-Farber Cancer Institute in Boston.

We need to be looking for how long these cells persist and keep the cancer under control, Anderson said.

Dr. Carl June, a University of Pennsylvania researcher who received the conferences top science award for his early work on CAR-T therapy, said its very rare to see everyone respond to a treatment. His lab also had this happen all 22 children testing a new version of CAR-T for leukemia responded, his colleagues reported at the conference.

The first patients we treated in 2010 havent relapsed, June said.

Dr. Michael Sabel of the University of Michigan called the treatment revolutionary.

This is really the epitome of personalized medicine, extending immune therapy to more types of patients, Sabel said.

Legend Biotech plans to continue the study in up to 100 people in China and plans a study in the United States early next year. The treatment is expected to cost $200,000 to $300,000, and whos going to pay for that is a big issue, Fan said.

The manufacturing process is very expensive, and you cant scale up, he said. Its individualized. You cannot make a batch as is done with a drug.

Nick Leschly, Bluebirds chief executive, said the next phase of his companys study will test what seems the ideal dose in 20 more people.

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Gene therapy to fight blood cancer finds unprecedented success - Chicago Sun-Times

VBL Therapeutics won an ILS8.75 (approximately $2.5 million) grant from the Israel Innovation Authority to support continued clinical development of the firms lead Phase III-stage gene therapy ofranergene obadenovec (VB-111). The antiangiogenic candidate is being evaluated in a pivotal Phase III GLOBE study in patients with recurrent glioblastoma (rGBM) under an FDA Special Protocol Assessment. Phase II studies with VB-111 have previously been carried out in patients with platinum-resistant ovarian cancer and differentiated thyroid cancer indications.The gene therapy is administered as an IV infusion once every two months.

VBL says the grant will fund clinical trials and development activities for the 2017 calendar year. The continuous financial support for the VB-111 program is an important contribution to our ability to execute on our plans into 2019, as we prepare for the commercialization of VB-111 and establish our new manufacturing site, commented Dror Harats, M.D., CEO at VBL Therapeutics. We believe this nondilutive grant for the VB-111 program underscores the confidence that the Innovation Authority has in our technology and its potential for commercialization.

Vascular Biogenics Ltd., operating as VBL Therapeutics, is developing a portfolio of anticancer and anti-inflammatory programs based on its proprietary Vascular Targeting System (VTS) and lecinoxoidplatforms. VB-111 is an antiangiogenic, adenovirus 5 vector-based gene therapy developed using the VTS technology.

VBL is, in addition, developing a series of orally available, small-molecule lecinoxoid compounds that exhibit immune modulating anti-inflammatory properties for treating chronic immune-related conditions. An exploratory Phase II study with lead lecinoxoid candidate VB-201 indicated that the compound reduces vascular inflammation in atherosclerosis. DuringApril, VBL separately presented data from a retrospective analysis of Phase II studies indicating that oral administration of VB-201 reduces levels of liver enzymes. The firm said the data support the potential use of lecinoxoids for liver-related indications, including nonalcoholic steatohepatitis (NASH).

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VBL Wins $2.5M Grant to Progress Pivotal-Stage Anticancer Gene Therapy - Genetic Engineering & Biotechnology News

Multiple myeloma (also known as plasma cell myeloma) is a cancer of plasma cells, specifically, the kind of white blood cell that produces antibodies to help fight infection. Based on statistics from the National Cancer Institute, its currently one of the fastest growing cancer types the second for men and the third for women. In the U.S. alone, it is estimated that 30,280 new cases will be diagnosed this year, and 12,590 cases will turn out to be fatal.

Since the year 2000, a total of nine drugs have been approved as treatments for multiple myeloma. But the drugs cant be considered as cures because even with the treatment, only around 50% of patients live up to 5 years after being diagnosed with the disease. Thankfully, that might soon change.

A few days ago, at the American Society of Clinical Oncology conference held in Chicago, doctors reported about the success of a new treatment for multiple myeloma called CAR-T (short for Chimeric Antigen Receptor-T) therapy an individualized treatment that makes use of blood filtering and genetic reprogramming.

The process works by removing immune system cells called T cells from a patients blood, after which, the filtered blood is modified to contain a cancer-targeting gene, then re-injected into the patient to seek and destroy cancer cells. Its being called a living drug a one-time treatment designed to permanently alter cells that multiply in the body, turning those cells into an army of cancer-fighting cells.

In the clinical trial led by Dr. Wanhong Zhao associate director of hematology at The Second Affiliated Hospital of Xian Jiaotong University in Xian, China 33 out of 35 patients responded positively within two months of the treatment, with some patients responding as early as 10 days after the first injection (three separate injections were given in a span of more than a week).

So far, 19 of the patients are currently well beyond the timeframe needed for full efficacy assessment by the International Myeloma Working Group (IMWG) consensus. In other words, it is now reasonable to conclude if complete remission has been achieved in a patient or not. And the results are: 14 are now in complete remission, 4 have achieved very good partial remission, and 1 is in partial remission.

In terms of side effects, most experienced typical ones such as fever, low blood pressure and difficulty in breathing. Only 2 patients experienced severe side effects, though such were temporary and easily manageable.

As Dr. Zhao said: Although recent advances in chemotherapy have prolonged life expectancy in multiple myeloma, this cancer remains incurable. It appears that with this novel immunotherapy there may be a chance for cure in multiple myeloma, but we will need to follow patients much longer to confirm that.

Following the success of this small clinical trial, the researchers are now planning a bigger trial involving 100 patients. Early next year, they are also planning a similar run in the U.S.

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Breakthrough Gene Therapy Approach Raises Hopes For Patients With Blood Cancer - Wall Street Pit

May 30, 2017 08:05 ET | Source: Abeona Therapeutics Inc

NEW YORK and CLEVELAND, May 30, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today that the FDA has granted Rare Pediatric Disease Designation for Abeonas EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa (DEB), including recessive dystrophic epidermolysis bullosa (RDEB), which are life-threatening genetic skin disorders characterized by skin blisters and erosions that cover the body.

These designations are granted to drugs with high promise that may address areas of unmet medical need for children with rare diseases. RDEB is a debilitating and life threatening inherited disorder with no approved treatment options available for patients today," stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. Building upon the already granted FDA and EMA Orphan Drug Disease Designations for the EB-101 gene therapy program, receiving the Rare Pediatric Disease Designation is another important validation of the science and clinical approach to developing a novel gene therapy for RDEB patients.

Typically, wounds on patients with RDEB, also known as "butterfly skin" syndrome, can remain unhealed for months to years due to the inability of the skin to stay attached to the underlying dermis and can cover a large percentage of the body. In the ongoing Phase 1/2 clinical trial, EB-101 was administered to non-healing chronic wounds on each subject and assessed for wound healing at predefined time points over years. The primary endpoints of the clinical trial assess safety and evaluate wound healing after EB-101 administration compared to control untreated wounds. Secondary endpoints include expression of collagen C7 and restoration of anchoring fibrils at three and six months post-administration.

About Rare Pediatric Disease Designation: The rare pediatric disease designation indicates that the FDA may give the company a pediatric priority review voucher if the drug is approved for the pediatric indication. That voucher could then be used by the company for another drugany drugto be given a priority review. A priority review mandates that the FDA will review a BLA drug submission within six months instead of the standard 10 months. Normally, a priority review designation would only be given to a drug that is for a serious condition and has demonstrated the potential to be a significant improvement in safety and effectiveness. The priority review voucher may be used by the sponsor, sold or transferred.

EB-101 Gene Therapy Program Highlights:

About EB-101: EB-101 is an autologous, ex-vivo gene therapy in which COL7A1 is transduced into autologous keratinocytes for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). RDEB is a subtype of an inherited genetic skin disorder characterized by chronic skin blistering, open and painful wounds, joint contractures, esophageal strictures, pseudosyndactyly, corneal abrasions and a shortened life span. Patients with RDEB lack functional type VII collagen owing to mutations in the gene COL7A1 that encodes for C7 and is the main component of anchoring fibrils, which stabilize the dermal-epidermal basement membrane. Patients are being enrolled in the ongoing Phase 2 portion of the Phase 1/2 clinical trial (NCT01263379). The EB-101 program has also been granted orphan drug designation by the FDA and European Medicines Agency (EMA).

About Epidermolysis Bullosa (EB): EB is a group of devastating, life-threatening genetic skin disorders that is characterized by skin blisters and erosions all over the body. The most severe form, recessive dystrophic epidermolysis bullosa (RDEB), is characterized by chronic skin blistering, open and painful wounds, joint contractures, esophageal strictures, pseudosyndactyly, corneal abrasions and a shortened life span. Patients with RDEB lack functional type VII collagen (C7) owing to mutations in the gene COL7A1 that encodes for C7 and is the main component of anchoring fibrils that attach the dermis to the epidermis. EB patients suffer through intense pain throughout their lives, with no effective treatments available to reduce the severity of their symptoms. Along with the life-threatening infectious complications associated with this disorder, many individuals often develop an aggressive form of squamous cell carcinoma (SCC).

About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF (Salt Diafiltration) ethanol-free process. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Christine Silverstein Vice President, Investor Relations Abeona Therapeutics Inc. +1 (212)-786-6212 csilverstein@abeonatherapeutics.com

Media Contact: Andrea Lucca Vice President, Communications & Operations Abeona Therapeutics Inc. +1 (212)-786-6208 alucca@abeonatherapeutics.com

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, the expected receipt of a Priority Review Voucher and that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the ability to secure licenses for any technology that may be necessary to commercialize our products; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

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Abeona Therapeutics Receives Rare Pediatric Disease Designation ... - GlobeNewswire (press release)

Xconomy Texas

San Antonio Most diabetes treatments work by giving the body the insulin it needs to break down sugar. But that approach deals with the symptoms of diabetes. In recent years, scientists and companies have taken aim at the root cause of the condition by attempting to stimulate or replace the cells in the pancreas responsible for producing insulin in the first place. One of them is a San Antonio researcher hoping to use gene therapya potentially one-time, long lasting treatmentto do the trick.

When cells in the pancreas, known as beta cells, either get destroyed by the immune system or stop producing enough insulin, the result is type 1 or type 2 diabetes. Companies large and small-from European diabetes drug giant Novo Nordisk to privately held startups ViaCyte, of San Diego, and Semma Therapeutics, of Cambridge, MAwant to engineer stem cells that develop into pancreatic beta cells to help a patient produce insulin.

Other researchers, such as Bruno Doiron, a scientist and assistant professor at the University of Texas Health Science Center at San Antonio, have different ideas. Doiron has developed an injectible treatment consisting of three molecules glucokinase, a second that targets a protein known as PTP1B, and a third that targets a protein called Pdx-1, a so-called transcription factor that regulates genesthat, when infused into the body, are meant to help stimulate the formation of new beta cells. Doiron has tried the method on mice, and based on some encouraging early results, intends to move the work forward through a startup company.

You have to prove you can translate that to a large animal model, he says.

The San Antonio company, Syner-III, got its name because of the synergistic use of three molecules to generate the beta cells, he says. Those molecules are administered via a gene therapy procedure: theyre stuffed into a modified virus and injected directly into the pancreas in a one-time treatment, where they are meant to stimulate beta cell production. The work was published in the peer-reviewed journal Current Pharmaceutical Biotechnology in 2016.

Doiron hopes to raise as much as $10 million to complete preclinical testing.

Others, including Novartis, are considering different ways of boosting beta cell production. Researchers from the Swiss company published findings in Nature Communications that showed a group of compounds called aminopyrazines could be packed into a pill and similarly lead to more beta cells, and more insulin, in mice. Such attempts are fraught with failure, however. In an article on its own website, Novartis notes that researchers have succeeded in producing beta cells in mice many times, but havent been able to reproduce those results in humans.

The potential payoff, however, is huge. Some 29.1 million Americans have diabetes, and 1.25 million of them have type 1 diabetes, according to the American Diabetes Association. Doiron believes the therapy may be able to help both types. While stem cell research has had its share of failures and competition continues to increase in insulin therapysuch as pumps that automatically deliver the treatmentDoiron says a gene therapy, if successful, could result in a longer-lasting, more effective treatment.

When I use your own body to produce medicine, that drastically changes the field, he says.

David Holley is Xconomy's national correspondent based in Austin, TX. You can reach him at dholley@xconomy.com

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With Gene Therapy for Diabetes, San Antonio Researcher Eyes Funding - Xconomy

After deals and acquisitions with Spark Therapeutics and Bamboo, Pfizer is once again looking to bolster its rare and gene therapy pipeline as it outlines a new drug pact with Sangamo.

The collaborationlicense agreement focuses on the development and eventual sale of the biotechs gene therapy programs for hemophilia A, including SB-525, one of Sangamos four lead product candidates.

This early candidate is slated to enter the clinic this quarter, centering on testing safety as well as blood levels of Factor VIII protein, and other efficacy endpoints.

Sangamo gets $70 million upfront from the Big Pharma, and could gain $475 million in biobucks and sales royalties on any medications from the collaborationthat gain approval.

Under the deal, Sangamo will take the lead on the SB-525 phase 1/2 test as well as unspecified manufacturing activities.

Pfizer, meanwhile, will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for the therapy, as well as any additional products, if any.

Sangamo will also work with Pfizer on manufacturing and technical ops using viral delivery vectors.

SB-525 works as a AAV vector carrying a Factor VIII gene construct driven by a synthetic, liver-specific promoter. The FDA has already cleared the start of human trials for SB-525, and given it an orphan drug tag.

The deal has proved powerful for Sangamo, with its shares jumping 44% after hours on the news last night.

This marks another step into the new world of gene therapies for Pfizer, coming less than a year after its $700 million buy of Bamboo Therapeutics, adding advanced recombinant adeno-associated virus (rAAV)-based gene therapies to its pipeline.

It also has a long-standing deal with Spark Therapeutics, in hemophilia, penned in 2014. Back in January, Pfizer in fact paid a $15 million milestone bonus to Spark for hitting its marks in the ongoing hemophilia B phase 1/2 trial FDA breakthrough-tagged SPK-9001.

Pfizer also has a series of preclinical gene therapies, including a neuromuscular candidate for Duchenne muscular dystrophy (DMD), as well as preclinical candidates to treat Friedreichs ataxia and Canavan disease, and a phase I candidate for giant axonal neuropathy.

Pfizer also gained an operating gene therapy manufacturing facility that Bamboo bought from the University of North Carolina last year.

The pharma also has several academic research agreements, including one with Kings College London to develop a series of rAAV gene therapy vectors and another with the University of Iowa Research Foundation to develop a potential gene therapy for cystic fibrosis.

And its partnered with Emeryville, CA-based Molecular Therapeutics (4DMT) to discover and develop targeted next-generation rAAV vectors for cardiac disease; it made an investment in the company a few years back.

Once seen as the next big thing in research, gene therapies have however come under pressure in recent months about just how viable they are on the market. After struggling for years to make a commercial success out of Glybera, the worlds first approved gene therapy, uniQure recently called it quits on the treatment.

The drugmaker said it wouldnt bother asking European authorities to renew the $1-million-plus gene therapys marketing authorization when it expires in October, and comes after it abandoned plans to gain an approval in the U.S. Reports from MIT Technology Review suggest only one patient ever used the med.

GlaxoSmithKline has also been struggling in Europe with its bubble boy syndrome gene therapy Strimvelis. Mindful of Glyberas cost, GSK put its price tag at half that of Glybera, at $665,000, and also offered a money-back guarantee.

Its been approved in Europe for nearly a year, but it only treated its first patient this month, according to Business Insider.

Treatment is tough as the drug is not so much manufactured as it is created for each individual patient, with a site in Italy currently the only approved site in the world for this type of manufacture, and thus the only place where patients can be treated. Only around 15 patients in Europe are believed to have the condition.

Other biotechs are however working on the manufacturing side in order to try and make these therapies more available for patients, and thus open up their viability.

There are already a number of medications on the market for hemophilia, such as from Biogen spin-off Bioverativ and Sobi, with gene therapy predicted by some also working in the space, including uniQure and BioMarin, to be the next class for treating the blood disorder.

But speaking to FierceBiotech at the start of the year, Bioverativs new chief and former Biogen exec John Cox told me that while they are to working on gene therapy approaches to hemophilia, there are reasons to be cautious: There are of course question marks over gene therapy: The obvious one is safety, because of the history here, and this is a risk-averse population, for good reason, and the other question is naturally over efficacy, and how long does it last, as well as manufacturing, scale and so on.

Were all hoping for a cure, and of course were doing work on gene therapy now, but I dont think people are looking at these now as a permanent cure; the questions are over durability, rather than cure.

He said that investors and even doctors talk a lot about gene therapy in the hemophilia space, but that if you talk to hemophilia A patients about what they really want, being able to dose, once a week [which is the target with its candidate, or even just less frequently, is what they want.

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Pfizer doubles down on gene therapy pipeline with $70M Sangamo buy-in - FierceBiotech