Category Archives: Gene therapy

GREEN BAY, Wis. (WFRV) - (CBS News) The Food and Drug Administration approved a treatment that engineers a patient's own blood cells to fight cancer. It's the first type of gene therapy to hit the U-S market. It has already been used to treat children dying from leukemia, who are now cancer free.

The treatment reprograms a patient's own cells to destroy cancer, and then multiply to continue fighting disease for months or even years.

A new study looks at the risks associated with mononucleosis. A study in the journal Neurology finds that people who are infected with mono may have an increased risk for multiple sclerosis. White patients who had mono were twice as likely to develop MS. Black patients were four times more likely.

And American dads are getting older. A study at Stanford finds the average age of newborns' fathers has gone up by three and a half years since the 1970s. Men over the age of 40 now account for about 9-percent of U-S births. The potential downside is that older fatherhood is linked to higher rates of disorders including autism and schizophrenia.

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Healthwatch: Gene Therapy - WeAreGreenBay.com

MarketWatch rounded up 10 of its most interesting topics over the past week.

Campbell Soup Co. CPB, -1.73% had a rough quarter, but the company is facing a dire long-term problem.

Novartis AG NVS, -0.57% received FDA approval for the first cancer gene therapy available in the U.S. Emma Court explained how important this is for young people suffering from a type of acute lymphoblastic leukemia (ALL), and she interviewed Janney analyst Paul Knight, who made recommendations for investors on how to play a potential decade-long growth cycle for gene therapy.

Here are charts that will help you sift through a boatload of absurdity spouted every day by self-styled stock-market gurus.

Nissan Motor Co. 7201, +0.27% is about to launch a redesigned Leaf electric car. The company has a big advantage over Tesla Inc. TSLA, -0.14% because of its huge manufacturing scale, but one big question is the new Leafs battery range, as Claudia Assis reports.

The startling increase in value for bitcoin rivals that of other types of assets that have bubbled and burst. Andrew Left believes the Bitcoin Investment Trust GBTC, -20.40% is a very dangerous investment.

The damage from Hurricane Harvey to the Houston area has been devastating. The coming flurry of activity as the damage is repaired might cause a rise in U.S. GDP, but Caroline Baum calls claims of real economic benefits predictable nonsense.

Amazon.com Inc. AMZN, -0.24% was called the weakest major U.S. retailer this week by Moodys Investors Service. But T. Rowe Price Media and Telecommunications Fund PRMTX, -0.38% is a big believer. The fund, which had more than quadrupled the S&P 500s return over the past 15 years, had more than 10% of its assets in Amazons shares as of July 31.

If you are retired, you might think it will be very difficult to get a mortgage loan because of low income. But there are many financing options available for those without a steady monthly income, according to Darrow Kirkpatrick.

Jeff Reeves weighs the pros and cons of scooping up shares of Apple Inc. AAPL, +0.03% right now.

If you get excited by Labor Day sales, you might be missing out on bigger savings later.

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Weekend roundup: Campbell in the soup | New cancer gene therapy | Exposing bad investment advice - MarketWatch

HUMANKIND VIDEOS Mom gets big surprise after son is born via surrogate | 1:08

This new mom got the surprise of her life just 10 months after her son was born via surrogate. Humankind

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When a cross country runner collapsed, his opponent stopped to help him

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Dr. Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at C.S. Mott Children's Hospital in Ann Arbor, works with Maryam Rasheed of Macomb Township. Maryam was part of a clinical trial using gene therapy to successfully treat her leukemia.(Photo: Sophie Masson/Michigan Medicine)

The U.S. Food and Drug Administrationapproved on Wednesdaythe first-ever gene therapytotreat children and young adults withleukemia.

Called Kymriah, but better known as CAR T-cell treatment, the therapy is being hailed by doctors as revolutionary. Itinvolves genetically modifyinga patient's own T-cells, which thencantarget and kill a form of acute lymphoblastic leukemiacells.

This new treatment has the potential to change the face of cancer therapy for years to come, not just in childhood acute lymphoblastic leukemia but in other cancers in which a patients own T-cells can be collected, genetically modified and redirected to kill a patients tumor," said Dr.Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at the University of Michigan's C.S. Mott Children's Hospital. Mottwas one of a few hospitals nationally to take part inclinical trials of the treatment.

"This allows us to turn patients own cells into a powerful weapon to fight the disease a weapon that does not rely on chemotherapy but takes a whole new approach to attacking childhood leukemia, Yanik said.

The CAR T-cell treatmentoffers new hope for children like Maryam Rasheed, 10, of Macomb Township.

Maryam was diagnosed with B-cell acute lymphoblastic leukemia at age 4, when her family was seeking refuge from religious persecution in Turkey, said Maryam's mother, Asmaa Rasheed.

Maryam Rasheed (right) with her brother, Rashid, and sister Samantha. Maryam, 10 of Macomb Township, survived acute lymphoblastic leukemia.(Photo: Rasheed family photo)

"My country is Iraq," Asmaa Rasheedsaid. "It wasnt safe. We are Christian. It was so hard over there in Baghdad. We run away to Turkey.

"We take her to hospital the first timebecause ... she stopped eating, stopped walking, stopped talking. We bring her to emergency. The doctor decided to take her bone marrow to do tests. Then the results came back, and she have leukemia."

Maryam underwent her firstchemotherapy treatment in Turkey.

"Over there, it was so hard," Rasheed said. "The doctors dont speak English over there. We know English a little bit. We speak Arabic."

Maryam Rasheed of Macomb Township undergoes treatment for acute lymphoblastic leukemia. She is now in remission.(Photo: Rasheed family photo)

Rasheed stayed with her daughter for two months in the Turkish hospital. A few months later,the Rasheed family was able to immigrate to the U.S. and settled in Michigan.

But Maryam's cancer returned. She was treated at Children's Hospital of Michigan with more chemotherapy and radiation. In 2013,her younger brother, Rashid, proved to be a match for a bone marrow transplant.

Still, the cancer wouldn't relent.

The Rasheed family learned of a clinical trial for CAR T-cell therapy under way atMott. It was the family's last chance,Rasheed said.

Maryam Rasheed, 10, of Macomb Township holds up her arms joyfully. She's surrounded by her sister Samantha (left), brother, Rashid, and baby sister Annabell.(Photo: Rasheed family photo)

"There was nothing to do," her mother said."In Detroit, there was chemo, radiation, bone marrow transplant. It returned back three times. She lose her hair three times. It was so hard for her and my family."

She remembers the date Maryam started the clinical trial at Mott: Dec. 17, 2014. Maryam spent Christmas and her seventh birthday in the hospital.

"I think we waited like 100 days,I dont remember exactly, and they did a bone marrow test, and the medicine, it work!" Rasheed said.

"It was like a dream, you know, like light coming from far away when youre in the dark. Theres nothing else we could do. But the CART-cell was like a shining light from far away."

Maryam has been in remission two years, andis starting fourth grade next week at Shawnee Elementary School in Macomb Township.

"Now, shes start her life, and doing everything a little kid is doing," said Rasheed, who says she hopes the treatment helps other children, too.

So does Yanik.

"Acute lymphoblastic leukemia is the most common form of cancer in children, accounting for approximately25% of all childhood cancers," Yanik said. "This particular therapy utilizes a childs own immune system to target their leukemia."

Theclinical trials focused on the 15% to 20% ofchildren whoseB-cell acute lymphoblastic leukemia had either relapsed or who had residual leukemia cells in their bone marrow after treatment.

"Historically, such patients would have an estimated cure rate of approximately 10%," Yanik said. "The two trials were groundbreaking. In the most recent trial, 52 of 63 patients with childhood leukemia successfully entered complete remission with this therapy."

Novartis Pharmaceuticals Corp. got the FDA approval for the gene cell therapy, whichinvolves drawing blood from childrenwith B-cell acute lymphoblastic leukemia. The T-cellsin the child's blood are thenshipped to a lab where they are genetically engineered so theywillseek outa particular protein in the leukemia cells and attack. Patients are then infused with the modified blood, and the T-cells go to work to find and kill the leukemia.

The New York Times reported Wednesday that the therapy will cost $475,000 for the initial treatment, with additional treatments administered at no cost.

Although 83% of the children in the clinical trials for CAR T-cell therapy went into remission, Yaniksaid it's too early to tell howcurative treatmentswill prove in the long run. And, its use will be limited to only a few medical centers in the U.S.

"The University of Michigan is the only site in the state and within this region that is licensed to administer these cells for childhood leukemia," he said.

Offering the treatment at a large medical center like U-Mis essential, said Dr. Rajen Mody,a pediatric oncologist at Mott, because of the severity ofpotential side effects.

"It can cause serious side effects, especially within the first 21 days," said Mody, who is Mott's director of pediatric oncology. "Patients can have high fevers, bleeding complications, trouble breathing, infections. ... Thats why a hospital like the University of Michigan is the ideal place. ... Patients who undergo this treatment are usually so sick after an infusion of the CAR-T cells, that they can't be safely treated at smaller hospitals."

Dr. Rajen Mody, a pediatric oncologist at the University of Michigan's C.S. Mott Children's Hospital.(Photo: University of Michigan)

Yanik is hopeful that successful treatment with CAR T-cell therapy in children with leukemia will open the door for similar therapies targeting other cancers.

"Aseparate CAR T-cell trial targeting diffuse large-cell lymphoma was recently completed with the results in that clinical trial now under review at the FDA," he said. That trial alsoincluded adult patientsat the University of Michigan.

Mody called the gene therapy revolutionary.

"This is clearly a life-saving and potentially curative therapy," he said."Its being tested in other types of leukemia and solid tumors. Its too early to say whether its going to work as well for other cancers.... We are not there yet."

Still, he said, it's made all the difference for Maryam and her family.

"She was one of the lucky ones coming from Iraq, and with all the things she has survived. And then coming here and surviving this,... she clearly has some goodluck.

"I think she should do very well. Patients who actually survive the first six months and still have CAR T-cells detected in their systems tend todo very, very well."

Contact Kristen Jordan Shamus: 313-222-5997 or kshamus@freepress.com. Follow her on Twitter @kristenshamus.

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First gene therapy to treat cancer gets FDA approval; UM only Michigan hospital to use it - Detroit Free Press

MINNEAPOLIS (WCCO) Colin Cooley of Burnsville beat lymphoma four years ago, but the lymphoma came back in a different spot two years later.

Chemo wasnt cutting it, Cooley said. It was keeping it in check, but it wasnt getting rid of it.

He decided to undergo a clinical trial at the University of Minnesota. He received a gene therapy called CAR-T and is now cancer-free.

The FDA approved CAR-T Wednesday as the first type of gene therapy in the United States.

The treatment has been called a breakthrough in the fight against cancer. It is only approved right now to treat children with acute lymphoblastic leukemia, but doctors are excited about its potential for other cancers and diseases.

Doctors at the Masonic Cancer Center, University of Minnesotas Goldy vs. Cancer Day at the Minnesota State Fair called the therapy a major leap.

(credit: CBS)

Were able to take a patients own cells and turn them into something that can actually attack their specific cancer, said Dr. Edward Greeno, medical director of the University of Minnesotas Masonic Cancer Clinic. Many people have referred to this as living cancer because were taking live cells and turning them into your treatment.

First, a patients blood is drawn and their T-cells, or immune cells, are separated out. Those T-cells are then sent to a laboratory to be genetically modified and reprogrammed to zero in on the cancer.

Those modified cells are then multiplied in the lab before being returned to the patient via blood. They are essentially revved-up cells that are missiles for the cancer.

In one significant study, 83 percent of the patients who received CAR-T went into remission.

This treatment is expected to be offered for lymphoma patients next year. Dr. Greeno says it could be decades, though, before its offered to patients with other types of cancer.

Right now, its expensive almost $500,000 and used mostly on patients when other methods of treatment, like chemotherapy, have failed.

Before I didnt know if Id be here in three or four or five years, I didnt know, Cooley said. Now I feel like I have a new lease, some minor issues, but a new lease on life, and thats pretty exciting.

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How Does Gene Therapy Work? - CBS Minnesota / WCCO

Personalized cancer treatments known as CAR-T cells (chimeric antigen receptor T cells) have dominated the headlines lately, thanks to Novartis tisagenlecleucel, which won an early approval from the FDA for the treatment of leukemia on Aug. 30. But CAR-T treatments are labor-intensive and expensive to make, and they can attack healthy tissues in the body, leading to dangerous side effects.

Scientists at the Fred Hutchinson Cancer Research Center have developed a tool that they believe could address both those shortcomings of CAR-T and other forms of cell engineering. They have invented nanoparticles that deliver proteins to cells, which in turn edit those cells genes temporarily. Lead author and bioengineer Matthias Stephan describes it as hit-and-run gene therapy, and he believes the technique will streamline the manufacturing of cell-based therapies.

Heres how it works: The nanoparticles home in on specific cells, such as the T cells in the immune system. They then deposit messenger RNA (mRNA) to those cells, which triggers short-term changes in the proteins the genes produce. The technology does not permanently change the DNA, but it makes enough of an impact on it to produce a therapeutic outcome.

RELATED: Can CAR-T cancer treatments be fine-tuned to avoid toxic side effects?

Whats more, the nanoparticles can be freeze-dried and then activated with a small amount of water. They really let you fulfill all your wishes as a genetic engineer because you can pack in all your different [gene-therapy] components and further improve the therapeutic potential of your cell product without additional manufacturing steps, Stephan said in an article posted on Fred Hutchs website.

Stephans team proved out their concept by testing the nanoparticles in three different cell-engineering applications, one of which was CAR-T. Currently, CAR-T treatments are made by giving T cellsgenes that teach them to destroy cancer cells. The Fred Hutch scientists used their nanoparticles to remove a different gene from T cellsone that normally prompts them to attack healthy tissue.

Then they tried enhancing the CAR-T cells in a different manner. They temporarily gave them genes that have the potential to make central memory T cells, which are able to survive over the long term, remembering their cancerous targets and attacking them should they ever resurface.

The scientists tested their engineered CAR-T cells in mouse models of leukemia and found that the animals that received them lived twice as long as mice that got conventional CAR-T cells. They also tested the nanoparticles in two other cancer-related applications of gene therapy.

Despite all the excitement over CAR-T, concerns about side effects continue to dog the field. A dangerous immune reaction known as a cytokine storm has been seen in trials of both Novartis treatment and Axi-Cel, a CAR-T from Kite Pharma, which is being acquired by Gilead. The third player in the CAR-T field, Juno Therapeutics, saw its late-stage trials delayed when some patients died of neurological side effects.

Fred Hutch scientists have been working on other techniques for improving CAR-T. In December, a set of researchers there who receive funding from Juno announced positive results from a trial of a fine-tuned CAR-T treatment in patients with chronic lymphocytic leukemia (CLL). Instead of using just one type of CAR-T, the team combined two specially selected cell subtypes into one treatment. They also announced they had identified biomarkers that they believe can be used to predict which patients are likely to have severe reactions to the treatment.

Stephans team is now collaborating with several companies to fine-tune CAR-T treatments for cancer, according to Fred Hutch. And they believe their freeze-dried nanoparticles may prove useful in developing treatments for a range of other diseases, too, including HIV and blood disorders caused by defective hemoglobin.

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'Hit-and-run' gene therapy nanoparticles could enhance CAR-T ... - FierceBiotech

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KANSAS CITY, Mo. -- Lucas Novick, 27, has been in a battle with leukemia since his freshman year of college.

"I was having headaches that were so bad that they were causing vomiting pretty regularly and I couldn`t see straight well enough that I felt safe driving myself to school," Novick said.

Since 2009, Novick has endured a number of treatments including chemotherapy and a bone marrow transplant. The treatments have taken a physical and mental toll on Novick's body.

"The transplant that was supposed to save my life also nearly took it from me," Novick said. "The damage chemotherapy did to my body when I was first treated in 2009 and 2010 was such that I was walking with a cane after my 21st birthday. It did so much damage to my hip joints that they were replaced in 2011."

But after Novick's leukemia returned for a second time, he went to Children's Mercy Hospital where doctors were performing an experimental treatment.

"The approval of the CTL019 product for pediatric patients with relapsed refractory acute lymphoblastic leukemia is really exciting for us," Doctor Doug Myers, of Children's Mercy Hospital, said. "We`ve spent a lot of time working on ways to get the immune system into the fight against cancer because we think it can decrease toxicity, decrease the amount of chemotherapy and radiation that we use for these cancers."

Dr. Myers said the treatment helped Novick, a musician, back onto the stage and has held his leukemia awayfor two years.

"Those are really special rewards for us in this field that have seen so many failures of this type of therapy in the past. To see this go forward, move forward, do well enough for a pharmaceutical company will pick this up and take it the rest of the way, that`s a really special time for us," Dr. Myers said.

While doctors believe it's too early to call the new treatment a cure, many agree this is the first step to a new generation of cancer treatment.

"I know at the end of the day that this is the future of medicine," Novick said.

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Man describes new FDA-approved gene therapy for leukemia that ... - fox4kc.com

Gene therapy is the use of DNA as a potential therapy to treat a disease.In many disorders, particularly genetic diseases caused by a single genetic defect, gene therapy aims to treat a disease by delivering the correct copy of DNA into a patients cells.The healthy, functional copy of the therapeutic gene then helps the cell function correctly.

In gene therapy, DNA that encodes a therapeutic protein is packaged within a vector, often a naked virus, which is used to transfer the DNA to the inside of cells within the body. Gene therapy can be delivered by a direct injection, either intravenously (IV) or directly into a specific tissue in the body, where it is taken up by individual cells. Once inside cells, the correct DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patients disease and can provide long-term benefit.

Abeona is developing next generation adeno-associated virus (AAV) gene therapies. Viruses such as AAV are utilized because they have evolved a way of encapsulating and delivering one or more genes of the size needed for clinical application, and can be purified in large quantities at high concentration. Unlike AAV vectors found in nature, the AAV vectors used by Abeona have been genetically-modified such that they do not replicate. Although the preclinical studies in animal models of disease demonstrate the promising impact of AAV-mediated gene expression to affected tissues such as the heart, liver and muscle, our programs use a specific virus that is capable of delivering therapeutic DNA across the blood brain barrier and into the central nervous system (CNS), making them attractive for addressing lysosomal storage diseases which have severe CNS manifestations of the disease.

Lysosomal storage diseases (LSD) are a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. These diseases are characterized by progressive accumulation of storage material within the lysosomes of affected cells, ultimately leading to cellular dysfunction. Multiple tissues ranging from musculoskeletal and visceral to tissues of the central nervous system are typically involved in disease pathology.

Since the advent of enzyme replacement therapy (ERT) to manage some LSDs, general clinical outcomes have significantly improved; however, treatment with infused protein is lifelong and continued disease progression is still evident in patients. Thus, viral gene therapy may provide a viable alternative or adjunctive therapy to current management strategies for LSDs.

Our initial programs are focused on LSDs such as Mucopolysaccharidosis (MPS) IIIA and IIIB, also known as Sanfilippo syndromes type A and type B. MPS III is a progressive neuromuscular disease with profound CNS involvement. Our lead product candidates, ABO-101 and ABO-102, have been developed to replace the damaged, malfunctioning enzymes within target cells with the normal, functioning version.

Delivered via a single injection, the drug is only given once.

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Gene Therapy - Abeona Therapeutics

CSL Behring has paid out $91 million upfront for biotech Calimmune and gains a preclinical asset for sickle cell disease and -thalassemia, adding to its blood disease pipeline.

The biotech is working onexvivo hematopoietic stem cell (HSC) gene therapy, with R&D facilities in Pasadena, California, and Sydney, New South Wales, Australia, that will now transfer over to CSL.

For its $91 million, and $300-plus in biobucks, CSL also gets two platform technologies, Select+ and Cytegrity, which are designed to address some of the major challenges currently associated with the commercialization of stem cell therapy, according to the company.

This includes the ability to manufacture consistent, high-quality productsand to improve engraftment, efficacy and tolerability, it said in a statement. Both technologies have broad applications in ex vivo stem cell gene therapy.

Calimmune shares in our promise and focus to improve the lives of patients with rare and serious medical conditions, said CSL CEO and managing director Paul Perreault. The acquisition represents another important step in the execution of our strategy for sustainable growth. Calimmunes scientific accomplishments are impressive.

The team has built a robust technology platform, and designed a promising HSC gene therapy candidate, CAL-H, which strongly aligns with our longer-term strategic goals, and complements our core competencies and areas of therapeutic focus, Perreault added. While Calimmune is still in the early stages, we believe that our combined strengths have tremendous potential to change treatment paradigms, and most importantly, significantly improve the lives of our patients.

RELATED: CSL heart attack drug hits on safety, misses efficacy in PhIIb

Calimmunes chief, Louis Breton, added: We are excited to become part of CSL Behring. They are an established global industry leader in protein-replacement therapies and have a proven track record of driving innovations through the development pipeline and delivering differentiated products to the global marketplace. Together, we are well-positioned to take our achievements to the next level.

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CSL Behring buys Calimmune for early stem cell, gene therapy boost - FierceBiotech

Regenxbio has penned a deal to acquire Dimension Therapeutics. The takeover will see Regenxbio pay about $86 million to add two of early-stage gene therapies to its pipeline.

Rockville, Maryland-based Regenxbio swooped on Dimension to gain the rights to DTX301 and DTX401. DTX301 is the more advanced of the candidates, having already moved into a phase 1/2 trial in patients with late-onset ornithine transcarbamylase deficiency.

Regenxbio expects glycogen storage disease type Ia candidate DTX401 to reach the IND stage early next year, boosting the depth of its pipeline.

We believe that DTX301 and DTX401 are product candidates that address diseases with high unmet need and will become an important part of a strong internal pipeline at Regenxbio that has the potential to achieve multiple milestones through the end of 2018, Regenxbio CEO Kenneth Mills said in a statement. The deal also gives Regenxbio a stake in Bayer-partnered hemophilia A gene therapy DTX201.

The notable absence from the list of programs Regenxbio is excited aboutand the reason it is set to buy Dimension for a knockdown priceis DTX101. That was once Dimensions top prospect. But the release of data from a phase 1/2 trial of the hemophilia B candidate over the first half of the year marked it out as unlikely to succeed, particularly in light of clinical data generated by Spark Therapeutics rival gene therapy.

That setback means the takeout by Regenxbio is a small, low-key exit for the once hard-charging biotech.

Dimension grew out of Fidelity Biosciences belief gene therapy was ready for the big time. And, having been spawned through an alliance with ReGenX, Dimension quickly pulled in $30 million from Fidelity and OrbiMed, was heralded as having the best AAV technology in the industry, landed a $250 million deal with Bayer, signed up Merck KGaA chief Annalisa Jenkins as CEO and capped off a busy first year with a Fierce 15 award. An IPO followed late in 2015.

That was about as good as it got for Dimension.

This year, the biotechs A-list credentials butted up against negative data. The negative data won. Dimensions market cap went on to slip to $30 million, attracting the interest of Regenxbio. A bid worth about three times Dimensions pre-news market cap proved enough to get the fallen gene therapy star to sign on the dotted line.

Shares in Dimension rose about 160% in premarket trading.

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Regenxbio to buy battered Dimension for gene therapies - FierceBiotech

CSL Behring will launch itself into the stem cell gene therapy through the acquisition of Calimmune, adding a preclinical candidate and two manufacturing efficiency technologies.

CSL Behring is set to pay $91m (76m) upfront and up to $325m in milestone payments over the next eight years for Calimmune.

According to spokeswoman Natalie de Vane, the acquisition is the first step into the cell and gene therapy space for CSL with the addition of an ex vivo hematopoietic stem cell (HSC) gene therapy candidate and R&D facilities in Pasadena, California, and Sydney, Australia a natural complement to the firms protein-replacement business and its rare diseases focus.

We have been interested in the field for some time, and believe that Calimmune is an appropriate point of entry into this area for us, and a good strategic fit for our business and our longer-term strategic goals, she told Biopharma-Reporter.

Calimmunes preclinical hematopoietic stem cell (HSC) gene therapy, CAL-H, for the treatment of sickle cell disease and -thalassemia, aligns nicely with our deep expertise and leadership in haematology.

Commercial manufacturing platforms

The deal also brings CSL the Select+ and Cytegrity platform technologies which de Vane told us have broad applications in other ex vivo stem cell gene therapies.

[The platforms] have the potential to address some of the major challenges currently associated with the commercialisation of stem cell therapies, such as the ability to manufacture consistent, high-quality product, and to improve engraftment, patient to patient variability, efficacy and tolerability.

The Select+ platform, for example, looks to reduce the required intensity of the conditioning chemotherapy currently used, which could make HSC therapy an outpatient procedure, while the Cytegrity tech looks to provide a scalable manufacturing platform with high batch-to-batch consistency.

[This] would be a tremendous advantage as today lentiviral vectors are generally manufactured in small batches through a convoluted process, she said.

The deal is expected to close in the next two weeks at which point CSL will hope to integrate the business with minimal disruption.

Immediately after close, Louis Breton, Calimmunes CEO, will report directly to our Chief Scientific Officer and R&D Director, Andrew Cuthbertson. Additionally, we will form a joint integration team focused on growing and building the Calimmune assets and technologies.

Excerpt from:
CSL adding scalable stem cell gene therapy tech in $91m Calimmune buy - BioPharma-Reporter.com