An 11-year-old girl in Massachusetts is at the forefront of a disease so rare, that it is believed only 22 people worldwide have been diagnosed with it. Talia Duff, who was born with Down syndrome and later diagnosed with Charcot-Marie-Tooth Neuropathy Type 4J (CMT4J), is slated to be among the first to enroll in a clinical trial that is awaiting FDA approval after her parents refused to watch her fall victim to the degenerative genetic disease.

Its a horrible feeling to go to a doctor and be told that theres nothing that can be done that the best you can do is try to make your child comfortable and enjoy the time you have together, John Duff, Talias dad, told PEOPLE. I learned to cherish moments in life that I would otherwise take for granted.

PREGNANT MOM DELAYS CANCER TREATMENT TO PROTECT UNBORN TWINS

The Duff family, which includes mom Jocelyn and older sister Teaghan, had noticed Talia struggling to crawl at around age four, and a regression in a number of other motor skills that at the time was attributed to her Down syndrome, and later to Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). Subsequent failed therapies and a diagnoses of osteoporosis due to prescribed steroids caused her parents to push for another diagnosis at Boston Childrens Hospital, according to a post on the familys Cure CMT4J Foundation website.

We learned that Talia did not in fact have CIDP but instead had an extremely rare form of Charcot Marie Tooth Disease a degenerative, genetic disease called CMT4J, the post read.

MEREDITH VIEIRA SPEAKS OUT ON 'SILENT' BONE DISEASE

The family learned the disease would slowly take over Talias body like a form of amyotrophic lateral sclerosis (ALS), eventually causing paralysis and robbing her of her ability to breathe. In the two years since her diagnosis, Talia lost her ability walk or even raise her arms.

We were supposed to sit back and watch our child live her life in reverse, the post on Cure CMT4J Foundation read. I decided not to accept this. I stayed up late nights pouring over scientific papers and booked appointments with the top CMT doctors in the world. We traveled to the University of Iowa and then Vanderbilt University, where we met Dr. Jun Li.

CHRISTIAN ROCKER RAISING FUNDS FOR BANDMATE WHOSE WIFE DIED HOURS AFTER CHILDBIRTH

It was at the meeting with Li that the Duffs learned of a genetic therapy that could potentially cure Talias disease, but that it was eight-to-ten years away from production. Knowing that time was of the essence for Talia, Jocelyn began connecting with other parent advocates and the family started the Cure CMT4J Foundation with a goal of raising $1 million for research. She met with a team of eight researchers in Maryland, who concluded that the gene therapy would have a lasting effect on Talia, and they are now working to attain proof of concept approval from the FDA, PEOPLE reported.

With approval expected to come later this summer, Jocelyn is prepared to then push for approval of a human clinical trial, with Talia expected to be among the first to receive the gene therapy intravenously.

We feel hope now, Jocelyn told PEOPLE. People have said to me, This is a lot of work for you, and my response is, Hey, you would do this for your child, too. I simply cant stand by and do nothing.

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Massachusetts girl may be among first-ever to receive gene therapy for rare disease after parents push for cure - Fox News

June 29, 2017 A CRISPR protein targets specific sections of DNA and cuts them. Scientists have turned this natural defense mechanism in bacteria into a tool for gene editing. Credit: Jenna Luecke and David Steadman/Univ. of Texas at Austin

Scientists from The University of Texas at Austin took an important step toward safer gene-editing cures for life-threatening disorders, from cancer to HIV to Huntington's disease, by developing a technique that can spot editing mistakes a popular tool known as CRISPR makes to an individual's genome. The research appears today in the journal Cell.

Scientists already use the gene-editing tool called CRISPR to edit the genetic code of nearly any organism. CRISPR-based gene editing will have an enormous impact on human health. More than a dozen clinical trials employing CRISPR on human cells are reportedly already underway, but the approach is imperfect. In theory, gene-editing should work much like fixing a recurring typo in a document with an auto-correct feature, but CRISPR moleculesproteins that find and edit genessometimes target the wrong genes, acting more like an auto-correct feature that turns correctly spelled words into typos. Editing the wrong gene could create new problems, such as causing healthy cells to become cancerous.

The UT Austin team developed a way to rapidly test a CRISPR molecule across a person's entire genome to foresee other DNA segments it might interact with besides its target. This new method, they say, represents a significant step toward helping doctors tailor gene therapies to individual patients, ensuring safety and effectiveness.

"You and I differ in about 1 million spots in our genetic code," says Ilya Finkelstein, an assistant professor in the Department of Molecular Biosciences at UT Austin and the project's principal investigator. "Because of this genetic diversity, human gene editing will always be a custom-tailored therapy."

The researchers took a DIY approach to developing the equipment and software for their technique, using existing laboratory technology to develop CHAMP, or Chip Hybridized Affinity Mapping Platform. The heart of the test is a standard next generation genome sequencing chip already widely used in research and medicine. Two other key elementsdesigns for a 3-D printed mount that holds the chip under a microscope and software the team developed for analyzing the resultsare open source. As a result, other researchers can easily replicate the technique in experiments involving CRISPR.

"If we're going to use CRISPR to improve peoples' health, we need to make sure we minimize collateral damage, and this work shows a way to do that," says Stephen Jones, a postdoctoral researcher at UT Austin and one of three co-lead authors of the paper.

Andy Ellington, a professor in the Department of Molecular Biosciences and vice president for research of the Applied Research Laboratories at UT Austin, is a co-author of the paper. He says this method also illustrates the unpredictable side benefits of new technologies.

"Next generation genome sequencing was invented to read genomes, but here we've turned the technology on its head to allow us to characterize how CRISPR interacts with genomes," says Ellington. "Inventive folks like Ilya take new technologies and extend them into new realms."

This work can also help researchers predict which DNA segments a certain CRISPR molecule will interact with even before testing it on an actual genome. That's because they're uncovering the underlying rules that CRISPR molecules use to choose their targets. For example, they found that the CRISPR molecule they tested, called Cascade, pays less attention to every third letter in a DNA sequence than to the others.

"So if it were looking for the word 'shirt' and instead found the word 'short,' it might be fine with that," says Jones.

That sounds counterintuitive, but can be really useful. CRISPR originated from a natural defense in bacteria used to guard against invading viruses that evolve rapidly. A good defense sees through slight changes in the viral genetic code.

Knowing these rules will lead to better computer models for predicting which DNA segments a specific CRISPR molecule is likely to interact with. And that can save time and money in developing personalized gene therapies.

Explore further: Modifying fat content in soybean oil with the molecular scissors Cpf1

More information: Cell (2017). DOI: 10.1016/j.cell.2017.05.044 , http://www.cell.com/cell/fulltext/S0092-8674(17)30637-2

Journal reference: Cell

Provided by: University of Texas at Austin

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New technique enables safer gene-editing therapy using CRISPR - Phys.Org

Lonza will install four 2,000L single-use bioreactorsat its Singapore facilityto cater for growing demand for small to mid-volume biologics production.

At present the Tuas facility houses 86,500L of stainless steel bioreactor capacity including four 20,000L stirred fermenters.Last October Lonza announced it was making an undisclosed investment in single-usetechnology for mammalian manufacturing at the site.

And speaking in Tokyo this week, SVP of global sales Cindy Reiss-Clark revealed more details:

We are adding up to four by 2,000L single-use bioreactors, with the first two being on line in early 2018, she told delegates at the first BioPharma Expo, being held alongside Interphex Japan. This expansion is supporting the commercialisation strategies [of our customers] which require small to mid-scale [bioproduction].

She added one of the first customers to leverage the new technologies in Singapore would be Tracon Pharmaceuticals, a Californian-based oncology-focused firm which entered into a long-term agreement for commercial production of its lead candidate TRC105 in February this year.

Lonza will transfer the process to the 2,000L[line] in Singapore, Reiss-Clark said, while providing ongoing clinical support for the antibody which has orphan drug designation for the treatment of soft tissue sarcoma.

Cell and gene therapy space

She also spoke about Lonzas collaboration with Nikon CeLL innovation part of the Nikon Corporation. The firms are developing a cell and gene manufacturing services business in Japan.

The partnership was first announced in May 2015 and a GMP facility in Tokyois on track and will be ready in 2018, according to Reiss-Clark.

The Singapore site has cell and gene therapy capabilities but this latest project will strengthen Lonzas presence in Asia.

The firm recently acquired European cell and gene therapy maker PharmaCell in a deal that a spokesperson told this publication places Lonza as the leading contract development and manufacturing organisation offering an international cell and gene therapy manufacturing network, spanning the US, Europe and Asia.

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Lonza expands in Asia: 8000L single-use in Singapore, regenerative meds in Japan - BioPharma-Reporter.com

LogicBio Therapeutics has got off a $45 million series B funding round as it eyes the cash for disease-modifying gene therapies in rare childhood diseases.

London-based investment firm Arix Bioscience led the oversubscribed round in the semi-stealth biotech, with new investors OrbiMed, Edmond De Rothschild Investment Partners, Pontifax, and SBI, along with previous investors OrbiMed Israel Partners, also stumping up cash.

Arix Bioscience's investment manager, Daniel OConnell, M.D., Ph.D, will join Cambridge, Massachusetts-based LogicBios board as part of the raise. This brings its total raised to $50 million, much of which will be put toward finishing off preclinical work and moving them into human tests.

The biotech sets itself up as a breakthrough gene therapy company targeting lifelong cures for serious, early-onset rare diseases by combining the best of gene therapy and gene editing in a one-time treatment.

It was founded in 2014 with platform technologies discovered by Adi Barzel, Tel Aviv University, Dr Leszek Lisowski, Childrens Medical Research Institute, Australia, and Professor Mark Kay at Stanford University School of Medicine.

The first platform, GeneRide, is a technology that uses homologous recombination that is designed to allow site-specific transfer of therapeutic genetic material without the use of promoters or nucleases. The company says it also has access to a library of synthetic, non-pathogenic, recombinant adeno-associated viral (rAAV) vectors developed at Stanford that allows for better predictability of vector performance in clinical trials.

Joe Anderson, CEO of Arix Bioscience, said: Early intervention for rare genetic disorders in children is important and LogicBio is uniquely positioned at the forefront of this research area with its proprietary genetic therapy technology to deliver a durable cure for young patients with life-threatening genetic diseases and otherwise limited options. LogicBio has huge potential and, alongside its excellent team and investors, we look forward to supporting the company to achieve continued success in this area.

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Arix leads $45M series B for gene therapy biotech LogicBio - FierceBiotech

Dimension Therapeutics is winnowing out 25% of its staff as it concentrates on three gene therapy programs, including one partnered with Bayer that has the potential to generate some badly needed milestone cash.

The biotech says it has enough revenue in hand to operate for another year, adding that it can extend the runway out to the end of 2018, provided it bags about $15 million in cash in its deal with Bayer. Three years ago Dimension inked a $252 million pact with Bayer, with $20 million of that upfront.

Annalisa Jenkins

Counting milestone money in your business plan isnt likely to generate much confidence among investors, especially after some disappointing results and evidence of liver toxicity for its initial lead gene therapy for hemophilia B in January crushed the biotechs stock price. DTX101 which faced more advanced competitors with better data has now been shoved out of the spotlight.

The biotechs market cap has now shrunk to $38 million.

The lead program in the clinic now is DTX301 for rare cases of ornithine transcarbamylase (OTC) deficiency. The biotech is lining up two more programs for INDs, including DTX201 allied with Bayer.

Dimension was one of several gene therapy companies to get started with a technology licensing deal with ReGenX, a spinoff from the University of Pennsylvania which is working with AAV technology developed by scientific founder James Wilson.

Our key focus is to deliver initial data from our ongoing Phase I/II clinical trial for DTX301 in OTC deficiency, advance two proof-of-concept studies for glycogen storage disease type Ia (GSDIa) and hemophilia A, the latter in collaboration with Bayer, and advance our unique HeLa 2.0 manufacturing platform, says CEO Annalisa Jenkins. We believe we can deliver these important objectives in 2017-2018 with our current financial position.

News reports for those who discover, develop, and market drugs. Join 16,000+ biopharma pros who read Endpoints News articles by email every day. Free subscription.

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Hammered on a gene therapy setback, Dimension cuts staff and circles the wagons - Endpoints News

The go to gene-delivery vehicle, the adeno-associated virus (AAV), doesnt always go to where you would want it to go, particularly if the destinations you have in mind include the brain or the peripheral nervous system. Typically, viral vectors struggle to cross the bloodbrain barrier. Also, they tend to become dispersed across the body when they are tasked with delivering genetic cargo to far-flung neurons beyond the brain and spinal cord, such as those that sense pain and regulate heart rate, respiration, and digestion.

To help develop gene-delivery systems that can provide efficient transduction to neurons throughout the body, scientists based at the California Institute of Technology have introduced two AAV variants: one that efficiently ferries genetic cargo past the bloodbrain barrier and another that is efficiently picked up by peripheral neurons residing outside the brain and spinal cord.

Details appeared June 26 in the journal Nature Neuroscience, in an article entitled Engineered AAVs for Efficient Noninvasive Gene Delivery to the Central and Peripheral Nervous Systems. The vectors are able to reach their targets following a simple injection into the bloodstream. Also, the vectors are customizable and could potentially be used as part of a gene therapy to treat neurodegenerative disorders that affect the entire central nervous system, such as Huntington's disease, or to help map or modulate neuronal circuits and understand how they change during disease.

Here, we describe AAV-PHP.eB and AAV-PHP.S, capsids that efficiently transduce the central and peripheral nervous systems, respectively, wrote the articles authors. In the adult mouse, intravenous administration of 1 1011 vector genomes (vg) of AAV-PHP.eB transduced 69% of cortical and 55% of striatal neurons, while 1 1012 vg of AAV-PHP.S transduced 82% of dorsal root ganglion neurons, as well as cardiac and enteric neurons.

The work was led by Viviana Gradinaru, Ph.D., assistant professor of biology and biological engineering at Caltech. "We have now developed a new collection of viruses and tools to study the central and peripheral nervous systems," she said. "We are now able to get highly efficient brain-wide delivery with just a low-dose systemic injection, access neurons in difficult-to-reach regions, and precisely label cells with multiple fluorescent colors to study their shapes and connections."

The new vectors could help researchers study the activity and function of specific types of neurons within peripheral circuits using genetically encoded sensors and tools to modulate neuronal firing with light or designer drugs, respectively. The new vectors could also deliver genes that code for colorful fluorescent proteins, proteins that are useful in identifying and labeling cells.

The efficiency of these vectors facilitates robust cotransduction and stochastic, multicolor labeling for individual cell morphology studies, the articles authors noted. To support such efforts, we provide methods for labeling a tunable fraction of cells without compromising color diversity.

In the labeling process, multiple AAVseach carrying a distinct colorare mixed together and injected into the bloodstream. When they reach their target neurons, each neuron receives a unique combination of colors, thereby giving it a visually distinct hue that makes it easier for the researchers to distinguish its fine details from those of its neighbors. Furthermore, the team devised a technique to control the number of neurons labeledlabeling too many neurons makes it impossible to distinguish individual onesthat allows researchers to visualize individual neuron shapes and trace their connecting fibers through intact tissues using another technology that Dr. Gradinaru's laboratory has helped develop, known as tissue clearing.

"Usually, when researchers want a mouse or other animal model to express fluorescent proteins in certain cells, they need to develop genetically modified animals that can take months to years to make and characterize," said former graduate student and first author Ken Chan (Ph.D. '17). "Now with a single injection, we can label specific cells with a variety of colors within weeks after the injection."

"For our new systemic viral vectorsAAV PHP.S and AAV PHP.eBthere are many potential uses, from mapping circuits in the periphery and fast screening of gene regulatory elements to genome editing with powerful tools such as CRISPR/Cas9," asserted Dr. Gradinaru. "But perhaps the most exciting implication is that our tools, when paired with appropriate activity modulator genes, could enable noninvasive deep brain modulation for the treatment of neurological diseases such as Parkinson's disease."

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Gene Therapy Vectors Come to Grips with Nervous System, Root and Branch - Genetic Engineering & Biotechnology News

Many Helpers Make Light Work

Targeting cancer cells that have spread to several organs of the body is difficult. Targeted radiation therapy or chemotherapy tends to destroy not only the cancer cells but also normal cells. Turning to gene therapy to selectively deliver therapeutic genes into these cancer cells on a larger scale and eliminating them in one fell swoop is the ultimate goal of Tocagen.

Using two products, Toca 511 and Toca FC, the company plans on developing an effective combination therapy that could hit the cancer hard. Toca 511 is an injectable retroviral replicating vector (RRV) that provides the genetic material to encode a prodrug activator enzyme, cytosine deaminase (CD), which is derived from yeast and has no human counterpart. It is selectively delivered to only cancer cells, thus producing the CD protein in each cell.

Part two of this therapy involves a pill called Toca FC, which contains 5-fluorocytosine (5-FC) that converts to the anticancer agent 5-FU in the presence of CD protein. Toca FC kills not only the cancer cells, but also the myeloid-derived suppressor cells (MDSCs), which suppress the immune system, and tumor-associated macrophages (TAMs).

Harry Gruber, M.D., cofounder and former CEO of Tocagen, talks about the use of gamma-retroviruses: The advantage of using a gamma-retrovirus (as opposed to the lentivirus) is that it cannot enter the nucleus on its own. This makes it selective to dividing cells only, and since cancer cells are rapidly dividing, [gamma-retroviruses] help in spreading the virus and its genetic information. They live in defective cells that lack an innate immunity, and due to this selectivity, they are designed to be universally geared toward only cancer cells.

Dr. Gruber also mentioned that Toca 511/FC received the FDAs Breakthrough therapy designation, which expedites drug development.

The field of gene therapy has come a long way since its inception. Early failures and setbacks forced researchers back to the drawing board to figure out how viral vectors could be accepted by the human body, which ordinarily rejects foreign particles. Researchers also had to learn how such vectors could reach specific targets and deliver foreign DNA that could be integrated into the genome. This dance between therapy and the innate immune system is getting more complex, but is also showing its true beauty within the complexity.

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Gene Therapy: A New Twist on an Old Helix - Genetic Engineering & Biotechnology News

Bluebird Bio haspresented encouraging data from its gene therapy treatment for the rare condition, cerebral adrenoleukodystrophy (CALD).

The company is competing with other pioneering firms to develop transformative gene therapies for severe genetic diseases, as well as T cell-based immunotherapies for cancer.

It has had a run of good news recently, including encouraging early stage results from its multiple myeloma candidate bb2121 at ASCO.

But gene therapy Lenti-D is one of its two most advanced candidates, and has just produced encouraging topline interim data from its Phase 2/3 Starbeam Study (ALD-102).

Lenti-D is being studied in boys under 18 years old with CALD. The data shows that two years after receiving the gene therapy, 15 out of the 17 boys on the trial show are free of major functional disabilities (MFDs), the primary endpoint of the trial.

Also known as Lorenzos Oil disease, adrenoleukodystrophy (ALD) is estimated to affect one in every 21,000 male births worldwide. Cerebral adrenoleukodystrophy (CALD), is a potentially fatal form of ALD that affects the nervous system of boys, and involves a breakdown of the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control.

The current standard treatment for CALD is allogeneic hematopoietic stem cell transplant (HSCT), but complications including graft failure, graft versus host disease (GVHD) and opportunistic infections can be fatal.

The Lenti-D therapy involves genetically modifying a patients own stem cells to contain functional copies of the ABCD1 gene. This restores the production of the missing ALDP protein, which helps to break down the very long chain fatty acids (VLCFAs) which cause the neurodegeneration.

David Davidson

The hope that Lenti-D may benefit boys facing such a devastating disease inspires all of us at bluebird, said David Davidson, M.D., chief medical officer, Bluebird Bio.

Having this proportion of the initial cohort of patients meet the primary endpoint is truly gratifying, bringing us one step closer to our goal of making Lenti-D available for patients with CALD. The two patients who did not meet the primary endpoint underscore the devastating nature of CALD, the importance of early diagnosis through newborn screening, and the challenges of the current standard of care with allogeneic hematopoietic stem cell transplant (HSCT).

The company commented on the condition of the two patients who hadnt responded, including Patient 2016, whose disease progressed before he could receive the new treatment. He subsequently died from complications of the allogeneic transplantation, underlining the problems with current treatment.

Bluebird said the Starbeam Study showed the safety profile of Lenti-D was consistent with myeloablative conditioning, with no patients treated experiencing graft versus host disease (GvHD), graft rejection or clonal dominance.

The Starbeam study had been expanded to treat eight additional patients at sites in Europe and the US, and the study is currently enrolling the additional patients. It says this expansion is intended to enable the first manufacture of Lenti-D in Europe, expand treatment to Europe and to bolster its overall clinical data package ahead of filing.

The company has also just announced encouraging data from its other lead candidate, LentiGlobin.Its phase 3 Northstar-2 studyin transfusion-dependent -thalassemia (TDT) and non- genotypes shows that trial participants are free from transfusions, three and a half years after receiving treatment.

The firm, based in Cambridge, Mass, has also just announced announced a public offering of $350 million of common stock to help fund its R&D plans.

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More good news for Bluebird Bio with CALD gene therapy - pharmaphorum

Xconomy Boston

One of the years most closely watched clinical studies could lead to a landmark approval of a gene therapy and throw wide open the debate over how to pay for expensive drugs. The first drips of data have emerged.

Bluebird Bio (NASDAQ: BLUE) says the first three patientsof 15 total expectedhave had good results from a revised version of its LentiGlobin gene therapy to treat certain genetic variants of the rare blood disease beta-thalassemia, which causes severe anemia and requires frequent transfusions.

Bluebird has changed the way it manufactures the product, which requires extracting a patients bone marrow cells, altering their DNA outside the body, then reintroducing the cells to the patient. This study, called NORTHSTAR-2, is the first test of the improved process, which regulators said last year would not require rewinding its clinical program back to the beginninga sigh of relief at the time for the company and its shareholders.

Caveats abound. The results are not only a small sample size, they are also early. Typically data from three patients in a study would not be worth singling out. But Bluebird, of Cambridge, MA, is trying to produce a type of medicine never approved before in the U.S. (Two have been approved in Europe, but one never took hold.)

And the FDA has already shown willingness to consider approval of medicines for rare diseases based on tiny sample sizeswith considerable controversy, in the case of a drug approved last year to treat Duchenne muscular dystrophy.

In one NORTHSTAR-2 patient, the healthy version of the blood protein hemoglobin has reached normal levels six months after a single dose of treatment. The second patients healthy hemoglobin levels are rising but lower than the first patient after three months. The third patient is only two months out from treatment.

For patients with good results, the treatments staying power will be crucial. Bluebird wants it to be a one-time cure, as of course will patients. Insurers will undoubtedly want the samebut what to do if something that costs hundreds of thousands or more than a million dollars, stops working after a few years?

Bluebird officials say they have already begun talking to payers about pay for performance arrangements. Our hope is to tie outcomes of the patient to the value generated, says chief financial and strategic officer Jeff Walsh. It can come in many different forms. (Xconomy reported on several creative drug-pricing ideas in this article.)

Bluebird hopes to make a case for approval for beta-thalassemia before U.S. and European regulators, perhaps in 2019, using data from the NORTHSTAR-2 trial and from previous trials that used the older LentiGlobin version. The main goal of NORTHSTAR-2 is for patients to produce enough of their own healthy hemoglobin to eliminate the need for regular blood transfusions. The first patient has reached that goal, says chief medical officer David Davidson.

The new version of LentiGlobin product, among other things, squeezes more copies of the correct gene into each targeted cellmore shots on goal to change each malfunctioning cell for the better, in other words.

The NORTHSTAR-2 patient with six months of results to report has fared better than similar beta-thalassemia patients six months after they received the previous version of LentiGlobin in a study called HGB-204. The NORTHSTAR-2 patient is producing 13.3 g/DL of hemoglobin, within the normal range for a woman; the median production among 10 HGB-204 patients after six months was 9.7 g/DL.

A doctor working on the study is presenting the data, along with updates from its LentiGlobin treatment for sickle cell disease, at the European Hematology Association meeting this weekend.

Alex Lash is Xconomy's National Biotech Editor. He is based in San Francisco.

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Bluebird Reports Early Results From Upgraded Gene Therapy - Xconomy

As a pioneer in the new wave of gene therapy treatments that have been steadily winding their way toward regulators with the promise of a once-and-done genetic fix for a wide range of ailments, bluebird bio $BLUE also got the first taste of the kind of backlash that can occur when a new technology fails to live up to the hope and hype that spurs billions of dollars of investments.

David Davidson

For bluebird, that moment of truth came a little more than two years ago, when the company reported that a handful of patients had inadequate responses to its gene therapy for sickle cell disease and beta-thalassemia. Company investigators went back to the drawing board, changed the manufacturing process on LentiGlobin with a new approach they had been working on that they felt would deliver a better gene therapy. And today they unwrapped a snapshot of the impact theyve had.

Bottom line: The first glimpse of human data looks promising.

The first three patients in bluebirds Phase III study for transfusion-dependent beta-thalassemia patients offered some of the human proof-of-concept data they were looking for, multiplying the number of cells they were delivering with corrective genes and producing a healthy flow of hemoglobin needed to correct the disease in the first patient that was evaluable at 6 months. Two other patients offered indications of a similar success though one of the patients also registered a positive but lower number of vector positive cells than the other two leaving bluebird execs hoping that they have the right potency in place for a durable cure.

That uneven response took a quick bite out of bluebirds shares Friday morning, sliding 6% in early trading.

This is an exciting validation of the changes in the manufacturing process, says bluebird CMO David Davidson. He explained: We added two small molecule enhancers transduction enhancers that we have been researching for many years, to find ways of increasing the efficiency of the viral vector to enter and integrate into stem cells.

Heres the slide on the first patients response from bluebirds presentation at the European Hematology Association meeting in Madrid, which the biotech shared with me in a preview.

That 13.3 grams per deciliter column on the right at 6 months includes a substantial amount of hemoglobin produced specifically by the therapy after transfusions were stopped.

It is, to be certain, a tiny number of patients and there are no guarantees in this business. But then bluebirds fortunes in gene therapy have always been decided by the responses of a handful of patients. Its pivotal Phase III data from which will be delivered to the FDA along with another late-stage trialinitially will recruit 15 adult and adolescent patients, then bring in 8 more children in an extension study. Theyll all be tracked for a considerable amount of time as investigators look for any variation in efficacy and safety.

Bluebird still has a long ways to go and isnt free of concerns on the safety front. Davidson also highlighted two patients who were hospitalized after experiencing acute gastroenteritis and acute chest syndrome in their earlier -205 study, though the company also believes that investigators are increasingly confident that they can avoid repeats in the future.

So bluebird takes another step down the late-stage pathway, with plans to seek an early, conditional approval in Europe based on the small early studies it completed.

European regulators have been a little more creative and more willing to advance these therapies forward than the FDA, Davidson adds. But they are advancing on both fronts today.

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Bluebird bio steps up with a promising snapshot of its gene therapy ... - Endpoints News

GenSight Biologics has raised(PDF) 22.5 million ($25.2 million) to prepare to bring gene therapy GS010 to market in the U.S. and Europe. The financing gives the Novartis-backed biotech enough cash to deliver data from two phase 3 trials next year and gear up for anticipated approvals on both sides of the Atlantic. Paris-based GenSight raised the cash from a mix of new and existing institutional investors, most of which are based in the U.S. Strong interest from these backers saw GenSight ease past its initial target of 20 million to pull in 22.5 million in the private placement. When added to the 48.8 million GenSight had in the bank at the end of March, management thinks the money moves its runway out to the first quarter of 2019. That runwaycovers a critical period for GenSight. Topline 48-week data from two phase 3 trials of GS010 in patients with Leberhereditary optic neuropathy (LHON) are due in the second and third quarters of next year. GenSight is looking to the trials for evidence GS010 improves the clarity of the vision of patients with LHON, a hereditary form of vision loss caused by mitochondrial defects. GS010 is injected into the eye to deliver the human wild-type ND4 gene via an adeno-associated virus to deliver. This gene encodes for a protein typically produced by mitochondria. One trial is assessing GS010 in patients who started losing their vision in the six months prior to enrolling in the study. The other is recruiting patients whose vision started deteriorating between seven and 12 months ago. Both trials are injecting GS010 into one eye of each participant and pretending to inject it into the other eye. Data from an earlier phase 1/2 trial suggest the gene therapy is most effective in patients whose vision started deteriorating less than two years ago. A recent 96-week update found the treated eyes of such patients had a mean gain of 29 ETDRS letters, as compared to an increase of 15 letters in untreated eyes. ETDRS is the test showing progressively smaller letters opticians use to gauge vision. The performance of GS010 to date has enabled GenSight to secure the support of some big-name backers. Following the latest financing, its biggest shareholders are Novartis, Versant, Abingworth and Fidelity. GenSight tried to turn this transatlantic support into a Nasdaq listing in 2015, but it was forced to downsize and ultimately scrap the plan in the face of an unreceptive market. GenSight was part of a clutch of European biotechs that arrived on Wall Street as the IPO window was closing. And like others from that group, including Basilea Pharmaceutica and Bavarian Nordic, it has so far held off on trying again in favor of raising cash from other sources.

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Novartis-backed GenSight gets cash for gene therapy launch - FierceBiotech

Sarepta Therapeutics is expanding its arsenal of drug development initiatives aimed at finding a treatment for Duchenne muscular dystrophy (DMD).

The biopharmaceutical company will collaborate with Genethon, a non-profit R&D organization to jointly create gene therapies for DMD.

Genethon specializes in a micro-dystrophin gene therapy approach that can target the majority of patients with DMD, according to the announcement. The organization has demonstrated proof-of-concept of their program through a robust gene expression in a large animal model of DMD.

Heres how this deal will work.

Genethon will handle the early development work. Sarepta will then have the option of co-developing and gaining exclusive licensing rights to products that emerge from Genethons micro-dystrophin program.

Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industrys most comprehensive franchise in DMD, said Sareptas Chief Executive Officer Edward Kaye, in a statement. This partnership brings together our collective experience in Duchenne drug development and Genethons particular expertise in gene therapy for rare diseases. We look forward to working with Genethon given their knowledge, large infrastructure and state-of-the-art manufacturing capabilities to advance next generation therapies for DMD.

This pact marks the second partnership pertaining to DMD that Sarepta formed this year.

The company announced in January that it signed a licensing agreement with Nationwide Childrens Hospital regarding their Galgt2 gene therapy program.

This initiative is aimed at exploring a surrogate gene therapy approach to Duchenne muscular dystrophy, which makes induced genes produce proteins that can basically do the same job as dystrophin, the protein people diagnosed with DMD are unable to make to generate normal muscle growth.

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Sarepta Signs Gene Therapy R&D Deal for DMD - Drug Discovery & Development

NobleGnthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease.

Dedicated to rare diseases since 1990 and more recently to gene therapy, Gnthon is one of veryfew not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is well established as a nonprofit, credit for which is due toits creator, the French Muscular Dystrophy Association, AFM Tlthon. The company has nowteamed up with the (in)famous American company, Sarepta, to work on a gene therapy for Duchenne Muscular Dystrophy (DMD).

DMD first affects the shoulder and upper arm muscles and the muscles of the hips and thighs. (Source: mda.org)

When I last spoke to Gnthons CEO,Frdric Revah, he told me that while the majority of the companys financial support comes from Tlthon, an increasing amount comes fromsuch partnerships.We get more and more support from industrial partnerships as we outlicense more of our drugs, explains Revah. However, Tlthon will always remain the main source of our funding; the funding from out-licensing is a complement.

But is Sarepta the best partner? While the American biotech can boast about its FDA-approved drug for DMD, Exondys 51, (which is just sold to Gilead for $125M,) this achievement isdubious:not only was the key clinical trial tiny, the FDAadmittedthat patients did not receive aclear benefit fromthe drug in the study. These circumstances prompted arenowned journalist to suefor thedocuments pertaining to the decision, an attack thatSareptas stock into a downward spiral in May.

Nevertheless, under the terms of the partnership agreement,Gnthon will trust Sarepta as a potential co-developer ofits DMD candidates, all of which are preclinical. The French biotechhas been developing amicro-dystrophin gene therapythat has proven itself applicable to the disease. As it countswith Europes largest cGMP vector manufactory, YposKesi, andone of the worlds largest research and clinical groups developing rare disease therapies, Gnthon is an attractive partner for any companies prospecting in the field.

Sareptas pricing practices may also offendthe sensibilities of a nonprofit, since theyraised the hackles of the drug pricing patrol with a plan to charge $300k per year for Exondys 51. According to STAT News, the companysCEO, Edward Kaye, saidthis figure isin the middle of the range for rare disease drugs,and given the sensitivity to pricing, we tried to be reasonable when looking at all the costs.

Though financial details of the agreement have not been disclosed, Gnthon may have enough influence to sway Sareptaaway from gouging.Our main goal is to ensure that patients have access to drugs and that they are affordable. Price should not be an obstacle,Revah told me.

Whatever we do here, we hope to apply the same tech to diseases that affect more people, like sickle cell anemia and cancer,he continued. With a crowded arena of companies like CRISPR Therapeutics, AMO Pharma, andDebiopharmall battling to bring the next DMD drug to market, having a back up plan via a platform seemssensible.

Images via Alila Medical Media, Anatomy Insider / shutterstock.com

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French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy - Labiotech.eu (blog)

Sarepta Therapeutics has penned its second DMD gene therapy pact this year as it announces a tie-up with Frances Genethon, a nonprofit R&D org.

The research collaborationwill see the Franco-American pair jointly develop treatments for Duchenne muscular dystrophy and comes after Sareptas first FDA approval for DMD with its controversial med Exondys 51 (eteplirsen).

RELATED: FDA expert lashes out at 'worrisome' Sarepta approval in JAMA

Sarepta is looking to tap into Genethons preclinical microdystrophin gene therapy approach, which can target the majority of patients with DMD. Its current med can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.

It is hoping that with new tie-ups, it could produce a gene therapy that could treat many more, if not all, patients with the disease, although this is still some years off. DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease primarily affects young boysand occurs in about one out of every 3,600 male infants worldwide.

This builds on the pacts announced at the start of the year at the JPM conference, which saw it sign a deal with the Nationwide Childrens Hospital, which also focuses on the microdystrophin gene therapy program, as well as another form of gene therapy.

An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Childrens. It also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program, originally developed by researcher Paul Martin. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin. The goal will be to produce a muscle cell that can function normally even when dystrophin is absent, Sarepta said at the time.

Under the terms of its latest collaboration, Genethon will be responsible for the early development work. Sarepta has the option to co-develop Genethons microdystrophin program, which includes exclusive U.S. commercial rights. Financial terms, as is becoming more common with these pacts, have not been disclosed.

RELATED: With Exondys 51 approved, Sarepta chief Ed Kaye to bow out

Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industrys most comprehensive franchise in DMD, said Ed Kaye, Sareptas outgoing chief. This partnership brings together our collective experience in Duchenne drug development and Genethons particular expertise in gene therapy for rare diseases. We look forward to working with Genethon given their knowledge, large infrastructure and state-of the-art manufacturing capabilities to advance next generation therapies for DMD.

Frederic Revah, CEO of Genethon, added: Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. In order to accelerate the development of a treatment, we are very pleased to partner with Sarepta Therapeutics, which has demonstrated commitment and success for innovative therapies for Duchenne muscular dystrophy. This partnership brings together the highly complementary and synergistic expertises of Sarepta and Genethon, to the benefit of the patients.

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Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment - FierceBiotech

San Francisco-based Vineti, a cell and gene therapy software and analytics company, has closed on Series A funding round that pulled together nearly $14 million.

Backing came from General Electric Ventures, Mayo Clinic and new investor Draper Fisher Jurvetson.

The company will use the funds to continue growing its team and to deliver cloud-based software to improve patient access. It also plans to speed its work on life-saving treatment delivery and to promote safety and FDA compliance for individualized cell therapies.

The Vineti platform integrates logistics, manufacturing and clinical data.

Physicians, medical researchers and pharmaceutical companies are working together to develop successful therapies, transitioning from a one-size-fits-all model to individualized treatments for each patient, Vineti CEO Amy DuRoss said in a statement. But, the process for administering these treatments is broken and outdated, restricting access to terminal patients and creating unnecessary risk.

DuRoss added that Vineti developed the platform to ensure treatments reach the patients who need them the most. She added that many patients who are excellent candidates dont have access to the most innovative therapies and discovery timelines are more challenging than necessary.

GE Ventures formed Vineti based on customer requests to bridge the technology gap between individualized cell therapies and production.

Modern technology solutions to address complex production and delivery processes are lacking. GE Ventures, Mayo Clinic and DFJ have invested in Vineti to rectify these problems.

Vineti is led by DuRoss, Chief Strategy Officer Heidi Hagen and CTO Razmik Abnous.

Twitter: @Bernie_HITN Email the writer: bernie.monegain@himssmedia.com

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Vineti to fast-track cell and gene therapy tech with $14 million first ... - Healthcare IT News

SAN FRANCISCO, Calif. Mayo Clinic Ventures has partnered with a California-based company to make cancer-fighting gene therapies available to the public.

Vineti, a pioneering cell and gene therapy software and analytics company, announced Tuesday that it had completed its initial round of funding raising $13.75 million aimed at delivering "the first cloud-based software solution to improve patient access, accelerate life-saving treatment delivery, and promote safety and regulatory compliance for individualized cell therapies."

The funding was provided by Mayo Clinic Ventures, GE Ventures, DFJ and LifeForce Capital. It's just the 15th company that Mayo Clinic Ventures has backed since it was formed, according to Andy Danielson, vice chairman of Mayo Clinic Ventures.

"One thing with Vineti that we liked is that we have a commitment to cell and gene therapies at Mayo," Danielson told TechCrunch.com. "Vineti will make the gene and cell therapy production process more efficient and as a result, less costly. It's all part of the equation of making these therapies more affordable and opening them up to a greater number of people."

The targeted cancer therapy under development by Vineti is part of a thriving field that conducted more than 800 clinical trials in 2016 while investing nearly $6 billion. It's all aimed at positively impacting the oncology field, the largest market in medicine that's expected to grow to $165 billion by 2021.

The first two cell therapies are expected to hit the market later this year.

Vineti touts its plans as one that "integrates logistics, manufacturing and clinical data to improve product performance overall and enable faster, broader access for patients."

"Physicians, medical researchers and pharmaceutical companies are working together to develop successful therapies, transitioning from a one-size-fits-all model to individualized treatments for each patient," said Amy DuRoss, CEO at Vineti. "Now, the process for creating and delivering these treatments can be as innovative as the therapies themselves. We are developing the Vineti platform to help these treatments reach the patients who need them the most, and are confident the partnership between our advances technologies and leading medical research will deliver better outcomes across the globe."

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Mayo Clinic Ventures funds new cancer-fighting cell, gene therapy ... - Post-Bulletin

British drugmaker GlaxoSmithKline made headlines last year when it won approval for its gene therapy Strimvelis in Europe. But, due to a small patient population and high price tag, the drug has only been used once. So far, despite higher levels of safety and efficacy than previous iterations, the new wave of gene therapies still face commercial hurdles.

Spark Therapeutics looks set to be the next company to take on this challenge in the U.S. The biotech is currently awaiting approval of its treatment for a rare genetic form of blindness a potential one-time cure. Yet pricing will be the most closely watched aspect of this therapy, likely serving as an early barometer of what might be sustainable for a pipeline of treatments still in development.

While gene therapy offers the promise of cures and new ways of revolutionizing treatment of genetic diseases, society remains a long way from fully realizing those advances.

After decades of setbacks, a slew of next-gen gene therapies are ready to hit the U.S. market, prompting questions about manufacturing and pricing. Read More >>

A pricing conundrum and ethical decisions are clouding an already hazy path to market for many gene therapy drugs and providers. Read More >>

With an approval of Spark Therapeutics' gene therapy for a rare eye disease rapidly approaching, new questions about pricing are being raised. Read More >>

In a field shaped by small patient populations and eye-popping cost considerations, understanding gene therapy's promise and challenges comes down, in part, to the numbers. Read More >>

While many are optimistic about gene editing's ability to cure disease, it seems not enough realize the more dangerous aspects of treatment. Read More >>

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Gene therapy: What you need to know - BioPharma Dive

Behind the incredible process of developing targeted gene therapies to fight diseases like cancer lies an incredibly mundane problem that prevents these treatments from getting to patients paperwork and procedures.

While $5.7 billion was invested in companies developing cellular and genetic therapies, and with 800 clinical trials initiated worldwide and the first two CAR-T cell therapies expected to launch into market later this year, businesses still saythe ability to get these treatments to patients is limited by paperwork, supply chain management, and last mile delivery.

So GE (through its GE Ventures arm), the Mayo Clinic (through Mayo Clinic Ventures) and the venture investment firm DFJ have invested $13.75 million to back Vineti a software platform the companies are billing as a solution to gene therapys supply chain problem.

Its only the sixth company to have actually been built by GEs internal business team and spun out by the conglomerates venture arm.

According to company co-founder and former GE Ventures managing director Amy DuRoss, the process for developing and managing gene therapies is critical to the success of the treatment.

Amy DuRoss, chief executive at Vineti

To that end, Vinetis software tracks logistics, manufacturing and clinical data to improve treatments and drive down the cost of these therapies (which are mainly only accessible to those people with the very best health plans).

The startups technology was actually born out of necessity (always the mother of invention) and came from conversations that GE was having with a large, undisclosed customer.

A pharma company that is a regular client of GE Healthcare said we are solving late stage cancer and we want to take this commercial but we have not got the technology that can ensure that we can scale out these technologies in the commercial phase, DuRoss told me.

GEs healthcare business then took the problem to the companys venture investment and new business arm and began the development process of building a business.

In addition to DuRoss, who has been a luminary in the life sciences field since she helped with the push to get stem cell research approved in California; Vineti has a murderers row of leading healthcare talent.

Chief strategy officer Heidi Hagen, was the former SVP of Operations for cell immunotherapy pioneer Dendreon; chief technology officer Razmik Abnous was the chief technology officer at the healthcare data management juggernaut Documentum; and Malek Faham, the companys chief science officer, literally worked on some of the foundational science for gene therapies.

While the companys technology could have applications for a number of different treatments, and be used for several kinds of therapies, the focus, for now, is on cancer.

Cancer is a bullseye, says DuRoss. It is arguably the biggest cause of human suffering [and] there are treatments already in phase three, that if brought to market effectively could mark a turning point in medicines battle against the deadly disease, she said.

We see an opportunity as data accrues to the system over time for a use case in predicting therapy based on outcome data but were not making these claims today, said DuRoss.

Mayo Ventures had been working with GE for two years from the initial concept to the close of this new round of financing for Vineti. Its one of only 15 companies that the Clinic has backed since the formation of Mayo Clinic Ventures, and according to Andy Danielsen, the vice chair of Mayo Clinic Ventures.

One thing with Vineti that we liked is that we have a commitment to cell and gene therapies at Mayo, said Danielsen, so the interests were aligned. Vineti will make the gene and cell therapy production process more efficient and as a result, less costly. Its all part of the equation of making these therapies more affordable and opening them up to a greater number of people.

Therapy supply chain

External ordering pages

Product tracking

Therapy scheduling

Identity verification

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GE and the Mayo Clinic back software to bring cancer-fighting gene ... - TechCrunch

"We are thrilled to have Chris join Brammer at this key time of our growth. Chris brings a unique blend of commercial and clinical manufacturing experience with deep gene therapy knowledge and an outstanding leadership ethos that will strengthen Brammer's presence as the best in class gene therapy CDMO," said Mark Bamforth, Brammer's President and CEO.

Chris started his career at American Cyanamid (now Pfizer) in Pearl River, NY, where he held positions in quality control, viral vaccine development, and bulk manufacturing.He later joined BioReliance in Rockville, MD, with responsibility for US contract manufacturing operations, focused primarily on viral vector manufacturing and cell banking in support of gene therapy pre-clinical and clinical studies.

Regarding joining Brammer, Chris shared, "I am passionate about the gene therapy industry and excited to be joining the leading gene therapy CDMO.Gene therapy will improve lives and transform healthcare in the coming years.Brammer is uniquely positioned with exceptional scientific know-how and top class manufacturing and quality operations. I am delighted to join a team that has honed its expertise through the delivery of over 150 clinical batches and is now preparing to supply phase III and commercial gene therapy products for clients."

"Chris's experience manufacturing viral vectors and his understanding of their quality attributes together with his commercial manufacturing track record is unique. He is a great addition to our team," noted Richard Snyder, Ph.D., Brammer's CSO.

Chris holds a Bachelor's degree in Biological Sciences from Rutgers University Cook College and a Master's degree in Biochemistry from New York Medical College. Chris is active in the community including serving as Vice Chairman, Board of Directors for Massachusetts Biotechnology Education Foundation and on the Board of Directors for the West End House Boys and Girls Club of Boston.

About BrammerBrammer Bio is a leading CDMO providing clinical and commercial supply of vectors for in vivo gene therapy and ex vivo modified-cell based therapy, along with process and analytical development, and regulatory support, enabling large pharma and biotech clients to accelerate the delivery of novel medicines to improve patients' health. Brammer is owned by Ampersand Capital Partners, the only institutional investor in the company, and its founders. For more information, please visit http://www.brammerbio.com

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Brammer Bio Appoints Leading Commercial Manufacturing And Gene Therapy Expert As Chief Manufacturing Officer - PR Newswire (press release)

LONDON--(BUSINESS WIRE)--Cell Medica today announced the acquisition of Catapult Therapy TCR Limited, a subsidiary of Cell and Gene Therapy Catapult (CGT Catapult), and the initiation of a collaboration to establish cell therapy manufacturing for Cell Medica at CGT Catapults GMP manufacturing facility in Stevenage, UK. Financial terms were not disclosed.

Catapult Therapy TCR Ltd is a special purpose company set up by CGT Catapult, UCL Business and Imperial Innovations, and managed by CGT Catapult, for the development of the WT1 T cell receptor (TCR) cell therapy discovered through research at University College London (UCL) and Imperial College London. The WT1-TCR cell therapy enhances the immune system to fight cancer by genetically engineering the patients T cells to target WT1, a tumour-associated antigen which is expressed in both solid tumours and blood cancers.

CGT Catapult has been developing the WT1-TCR cell therapy for the treatment of acute myeloid leukaemia and myelodysplastic syndrome. Early development work, including initiation of a Phase I trial, was conducted at UCL and Imperial College London with funding from the UK charity Bloodwise. CGT Catapult advanced the product to a larger Phase I/II clinical trial and developed an improved manufacturing process. Having completed the treatment of eight patients with promising results, CGT Catapult will now transfer the WT1-TCR cell therapy rights to Cell Medica for continued development towards regulatory approval.

The WT1-TCR cell therapy will be integrated with the Dominant TCR platform technology which Cell Medica licensed from UCL Business in 2016. Applying the Dominant TCR technology to the WT1-TCR cell therapy is expected to result in a more efficacious product with the potential to treat patients with solid tumours such as mesothelioma and ovarian cancer, which have proven very difficult to treat with conventional therapies. Cell Medica is planning to initiate a Phase I/II clinical trial with a Dominant WT1-TCR version in late 2018.

Cell Medica and CGT Catapult have also initiated a collaboration to establish cell therapy manufacturing operations for Cell Medica at the GMP production facility recently built by CGT Catapult in Stevenage. The collaboration will include transferring the current WT1-TCR cell therapy manufacturing process to Stevenage over the next twelve months while Cell Medica and CGT Catapult work to develop a commercial scale production process using advanced manufacturing techniques. Cell Medica will also evaluate the feasibility of manufacturing additional cell therapy products at the site.

The acquisition of the WT1-TCR cell therapy leverages the investment we made in 2016 for exclusive rights to the Dominant TCR technology, said Gregg Sando, CEO of Cell Medica. Our objective is to show how we can enhance any existing TCR cell therapy with the Dominant TCR technology to create a more effective treatment for patients with solid tumours who otherwise have a very poor prognosis. We are also looking forward to an important collaboration with CGT Catapult to initiate manufacturing at the Stevenage GMP facility where we will work together on scale-up strategies for commercial production.

About Cell Medica

Cell Medica is committed to transforming patients lives through developing the significant therapeutic potential of cellular immunotherapy for the treatment of cancer. In collaboration with our strategic partners, Cell Medica is developing a range of products using three proprietary technology platforms including activated T cells, chimeric antigen receptors (CARs) and engineered T cell receptors (TCRs). Our lead product is CMD-003 is being tested in an international Phase II trial for the treatment of cancers associated with the oncogenic Epstein Barr virus. We are working with the Baylor College of Medicine and the University of North Carolina to develop next generation CAR-modified NKT cells including an off-the-shelf product. In the field of engineered TCRs, we are collaborating with University College London to develop the Dominant TCR technology platform. Cell Medica is headquartered in London with subsidiaries in Zurich and Houston.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 120 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

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Cell Medica Acquires WT1 Cancer Immunotherapy from Cell and ... - Business Wire (press release)