Category Archives: Gene therapy

Sen. Johnny Isakson (R-GA) recently commended the Food and Drug Administrations (FDA) approval of the first cell-based gene therapy available in the United States.

Officials said Kymriah was approved for certain pediatric and young adult patients suffering from a form of acute lymphoblastic leukemia, serving as an innovative therapy that reprograms a patients own cells to attack a deadly cancer.

This type of therapy is exactly what we had in mind when I began working for the Advancing Hope Act, which was ultimately approved and extended in last years 21st Century Cures legislation, Isakson said. When I heard this wonderful news directly from the FDA, I thanked them and told them to get it on the market, because its time to start saving kids lives.

The FDA said Kymriah would be used to treat acute lymphoblastic leukemia, a cancer of the bone marrow and blood that progresses quickly and is the most common childhood cancer in the United States referencing the National Cancer Institute estimates 3,100 patients aged 20 and younger are diagnosed with acute lymphoblastic leukemia yearly.

Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb said. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.

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Sen. Isakson applauds FDA approval of Kymriah gene therapy - Life ... - Life Science Daily

For a few thousand people around the world, reaching the age of 20 is a landmark to dread, not to celebrate.

Coping since birth with Leber Congenital Amaurosis (LCA), anyone with this genetic eye disorder who hasnt already lost their sight can expect to be legally blind before they reach 21 years of age.

Characterized by deep-set eyes that are prone to involuntarily, jerky movements, LCA is caused by a fault in one or more of about 14 genes so far identified. There is no proven treatment, although that may soon change.

In late August, biotech company Spark Therapeutics Inc. ONCE, +1.70% was granted a priority review of a treatment for LCA that may make it the first gene therapy approved for use in the U.S. by the Food and Drug Administration (FDA).

Read: Novartis CAR-T therapy was the first to be approved in the U.S.

The Philadelphia-based company will by Jan. 12 discover whether the FDA will issue a biologics license for Luxturna, which can replace the faulty RPE65 gene that causes LCA with a properly functioning copy. Should it be approved, victims of this disease will soon be able to receive a single injection that may permanently restore functional eyesight.

Gene therapys payoffs

While traditional research is usually focused on unlocking a way to treat one condition, gene therapies such as Luxturna may be game changers because they are based on platforms that can be adapted and used to tackle multiple inherited disorders.

Using similar techniques, Spark is also working on a functional cure for hemophilia, a disease that afflicts about 20,000 people in the U.S. and around 400,000 globally for which the market is worth about $8.5 billion in the U.S. and European Union.

In-human trials of SPK-8011 recently showed that Sparks therapy has the potential to lift the Factor VIII protein necessary for normal blood clotting to functional and sustained levels. In short, as with the Luxturna, the therapy has the potential to offer a one-shot cure.

That would be seismic for hemophiliacs, whose main option today is regular infusions of Factor VIII protein. Unfortunately, within a few days almost none of the protein remains in the body and the hemophiliacs blood is again unable to clot normally. Spark is also developing a treatment for hemophilia B, a much smaller market.

A new dawn

Biotech companies have reached this point because research has advanced to the stage where weve figured out how to identify the genetic causes of disease and how to apply that knowledge to develop therapies that will replace defective genes to provide a lasting cure.

Voyager Therapeutics Inc. VYGR, +24.70% is focused on gene therapies for neurological disorders such as Parkinsons, Huntingtons, Lou Gehrigs disease or ALS, Friedreichs ataxia (which damages the nervous system), Alzheimers and chronic pain.

In addition to cancer immunotherapy and the more controversial gene editing, bluebird bio Inc. BLUE, +0.84% has eight gene therapy programs, including research into adrenoleukodystrophy, or ALD, a deadly brain disorder that mostly affects boys and men; beta thalassemia; and sickle cell, none of which have a cure.

Should Spark, or another company such as BioMarin Pharmaceutical Inc. BMRN, -0.72% or Sangamo Therapeutics Inc. SGMO, -4.43% which are also working on hemophilia, succeed with its gene therapy, it could adversely impact suppliers of traditional Factor VIII protein infusions, such as Shire PLC SHP, +0.89% which had revenue from hemophilia treatments of $870.9 million in the first quarter of 2017.

Cost problems

Cost has been a headwind for the two gene therapies so far approved. In April, Fierce Pharma reported that uniQure NV QURE, +4.42% would not ask the European Medicines Agency to renew its marketing authorization for Glybera, the worlds most expensive drug at $1 million, when it expires in October, because in the four years after it gained approval in 2012 it was used commercially and paid for once, according to the MIT Technology Review.

Europes other approved gene therapy has fared no better. GlaxoSmithKline Plc GSK, +0.28% said in July it is seeking a buyer for Strimvelis, a treatment for a rare inherited immune deficiency, which took a year after approval to gain its first patient.

Perhaps the solution is a new payments system for ultra-expensive and long-lasting gene therapies, based on annuities for each additional time period of a treatments effectiveness.

But how do you measure cost? In December, Biogen Inc. BIIB, +0.48% gained FDA approval for Spinraza, a treatment for spinal muscular atrophy, the leading genetic cause of infant death in the U.S. Spinraza is priced at $375,000 a year for life (after $750,000 in the first year of therapy), while a one-shot gene therapy being developed by AveXis Inc. AVXS, +1.89% for SMA may provide a cure to someone who could go on to live 80 or more years. What sort of a premium for AveXis approach is justified?

Pricing is not dissuading biotech companies. There are about 7,000 genetic diseases, and the whole pharmaceutical and biotech industry is now working to solve each of those problems.

Investors seeking to benefit from a potential medical moonshot should consider allocating capital on a long-term basis to well-managed gene therapy companies with transformative assets that give them a competitive advantage.

Ethan Lovell is co-portfolio manager of the Janus Henderson Investors Global Life Sciences strategy.

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Opinion: How investors should play gene-therapy stocks - MarketWatch

A

gene therapy from Voyager Therapeutics (VYGR) delivered surgically into the brains of patients with advanced Parkinsons disease reduced their need for standard Parkinsons medications and improved motor function, according to results from a small, early-stage clinical trial reported Wednesday.

Unlike most gene therapies for inherited disease in clinical development, the Voyager treatment does not aim to cure Parkinsons because nothing yet invented can fully prevent the loss of neurons in the brain. Instead, Voyagers gene therapy acts like a biological detour around dead neurons, so Parkinsons patients respond better and longer to levodopa, the standard drug used to keep motor function under control.

This is a STAT Plus article and is only available to STAT Plus subscribers.To read the full story, subscribe to STAT Plus or log in to your account.Good news: your first 30 days are on us.

Senior Writer, Biotech

Adam Feuerstein is STATs national biotech columnist, reporting on the intersection of biotech and Wall Street.

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Gene therapy from Voyager improves motor function in Parkinson's - STAT

A single gene therapy that silences the mutation responsible for oculopharyngeal muscular dystrophy (OPMD) and replaces the mutated gene with a normal one may advance into human studies in the second half of 2018.

Benitec Biopharma started its OPMD program in 2014 and now announced their clinical candidate BB-301 as a one-step gene therapy approach.

OPMD patients develop muscle weakness in the upper eyelids and throat in adulthood, typically after age 40. OMPD is a rare genetic disease caused by a mutation of the poly(A)-binding protein nuclear 1 (PABPN1) gene. Because it affects fewer than 200,000 people nationwide, OMPD is considered an orphan disease that benefits from encouraging programs for drugs targeting these rare diseases.

In collaboration with research groups in London and Paris, Benitec tested a genetic approach known as DNA-directed RNA interference (ddRNAi) to shut down and replace the mutant PABPN1 gene using two different viral vectors. In this pre-clinical study, researchers found that the two-vector system restored muscular function in A17 mouse model, which displays many OPMD clinical signs including fibrosis and loss of muscle strength.

Now, Benitec combined silence and replace gene functions into a single vector (a carrier system) ina new clinical candidate, BB-301. Using a single vector, they succeed in eliminating 88 percent of the mutant gene product while restoring the normal gene function up to 90 percent. As a single product, BB-301 simplifies the regulatory process and the clinical strategy for human studies.

This is an important development in our OPMD program. The single vector system shows the same excellent activity as the earlier generation dual vector system where the silence and replace constructs were delivered in separate vectors. Similar application of the single vector technology may allow development of novel therapeutics to treat other orphan diseases. OPMD is a significant commercial opportunity for Benitec and we are working with the regulators and key opinion leaders in this field to advance BB-301 into the clinic as quickly as possible, Greg West, CEO, explained in a press release.

Benitec is in discussions with a broad group of OPMD clinicians and experts about a clinical trial that will be proposed to regulatory agencies later this year. If approved by the U.S. Food and Drug Administration, human studies could start in 2018.

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Gene Therapy for OPMD Nears Human Studies, Benitec Announces - Muscular Dystrophy News

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to Research and Markets' offering.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026.

The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 76 tables and 22 figures.

Profiles of 189 companies involved in developing gene therapy are presented along with 240 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.

These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

Key Topics Covered:

Part I: Technologies & Markets

Executive Summary

1. Introduction

2. Gene Therapy Technologies

3. Clinical Applications of Gene Therapy

4. Gene Therapy of Genetic Disorders

5. Gene Therapy of Cancer

6. Gene Therapy of Neurological Disorders

7. Gene Therapy of Cardiovascular Disorders

8. Gene therapy of viral infections

9. Research, Development and Future of Gene Therapy

10. Regulatory, Safety, Ethical Patent Issues of Gene Therapy

11. Markets for Gene Therapy

12. References

Part II: Companies

13. Companies involved in Gene Therapy

For more information about this report visit https://www.researchandmarkets.com/research/q99xbz/gene_therapy

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Global Updated Gene Therapy Technologies, Markets and Companies Report 2017-2026 - Research and Markets - Business Wire (press release)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), announced today that following a recent end-of-phase-II meeting with the U.S. Food and Drug Administration (FDA) covering the clinical, non-clinical and manufacturing data available to date, Agilis will begin preparing a biologics licensing application (BLA) for its gene therapy program for the treatment of Aromatic L-amino acid decarboxylase (AADC) deficiency, AGIL-AADC, targeted for submission in 2018.

We are pleased that the FDA agreed with our assessment of the potential clinical benefit of AGIL-AADC as observed in treated patients to date, and with the plan to submit a BLA for review, said Mark Pykett, President and CEO of Agilis.

Twenty-three patients have received AGIL-AADC to date, likely representing the largest cohort of patients in any CNS gene therapy program. Clinical data have been analyzed and presented on patients with severe AADC deficiency from two clinical studies in which each patient received a single administration of the AGIL-AADC gene therapy, an adeno-associated virus (AAV) vector containing the human gene for the AADC enzyme. Data on the first cohort of patients demonstrating sustained improvement in motor function over a 5-year period post-treatment were also presented. The Company has recently completed enrollment of a Phase IIb clinical study for AGIL-AADC which may be extended to permit the treatment of additional patients in need of therapeutic intervention.

Over the period of observation, we have seen important gains in motor function and a good safety and tolerability profile to date in treated children. Some of the children can maintain head position, sit unassisted, and stand with assistance, milestones these children would never have achieved without treatment, said Paul Hwu, M.D., Ph.D., Professor of Pediatrics at National Taiwan University Hospital, and study principal investigator.

AGIL-AADC has previously received Orphan Drug Designation and Rare Pediatric Disease (RPD) designation in the U.S., as well as Orphan Medicinal Product status in Europe. The U.S. Orphan designation allows Agilis to leverage the FDAs Priority Review pathway, hastening patient access to AGIL-AADC by shortening the review of the marketing application by as much as four months. The complementary RPD designation gives Agilis the ability to qualify for a Priority Review Voucher (PRV) upon approval of AGIL-AADC. PRVs can be redeemed or transferred to a third party to receive Priority Review on a subsequent marketing application for a different product. This voucher will be requested at the time of AGIL-AADCs marketing application and awarded upon approval of AGIL-AADC.

Agilis gene therapy program has been developed in collaboration with National Taiwan University and the U.S. National Institutes of Healths National Center for Advancing Translational Sciences (NCATS) through a Cooperative Research and Development Agreement (CRADA). NCATS is supporting this work through its Therapeutics for Rare and Neglected Diseases program (TRND). The University of Florida Powell Gene Therapy Center contributed manufacturing and toxicology work on the initial product.

An important mission in the TRND program is to help accelerate development of promising treatments for those diseases that are often overlooked, underfunded and continue to have significant unmet medical needs, said Nora Yang, Ph.D., Director of TRND Portfolio Management and Program Operations.

AADC Deficiency is a rare disease with a devastating clinical course. Symptoms include absence of motor milestone development, seizure-like events (termed oculogyric crisis) and hypotonia described by many as floppiness. Because of the nature of symptoms, the disease can be confused with other, better known disorders such as cerebral palsy, making diagnosis difficult and often leading to a protracted diagnostic odyssey in correctly identifying the disease. Unfortunately, many of our families wait several years for a diagnosis, said Lisa Flint, AADC Research Trust Founder and Managing Director. We are thankful that Agilis has worked closely with the AADC community to bring forward a potential major treatment for our kids.

About AADC Deficiency

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic condition resulting in lack of functioning AADC enzyme responsible for the final step in the synthesis of key neurotransmitters dopamine (a precursor of norepinephrine and epinephrine) and serotonin (a precursor of melatonin). AADC deficiency results in developmental failure, global muscular hypotonia, severe, seizure-like episodes known as oculogyric crises, autonomic abnormalities, and the need for life-long care. Given this neurologically devastating illness, patients with severe AADC deficiency have a high risk for death during childhood. Treatment options for patients with AADC deficiency are limited and there are currently no approved therapies.

About Agilis Biotherapeutics, Inc.

Agilis is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Agilis gene therapies are engineered to impart sustainable clinical benefits by inducing persistent expression of a therapeutic gene through precise targeting and restoration of lost gene function to achieve long-term efficacy. Agilis rare disease programs are focused on gene therapy for AADC deficiency, Friedreichs ataxia, and Angelman syndrome, all rare genetic diseases that include neurological deficits and result in physically debilitating conditions.

We invite you to visit our website at http://www.agilisbio.com.

Safe Harbor Statement

Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

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Agilis Biotherapeutics Updates Progress of its CNS Gene Therapy ... - Business Wire (press release)

This article originally appeared on Kaiser Health News

The countrys first approved gene therapy approved Wednesday to fight leukemia that resists standard therapies will cost $475,000 for a one-time treatment, its manufacturer announced.

Switzerland-based Novartis, which makes the innovative therapy, announced that the drug will cost nothing if patients fail to benefit in the first month.

TheFood and Drug Administrationapproved the therapy, called Kymriah, in children and young adults with acute lymphoblastic leukemia whose disease has come back in spite of previous treatments. These patients typically have a poor prognosis, surviving three to nine months, according to Novartis.et

In the study that led to Kymriahs approval, 83 percent of patients went into remission within three months, according to the FDA. Novartis estimates about 600 patients a year would be eligible for the treatment, which belongs to a class of drugs known as CAR T-cell therapies.

Kymriah treats cancer in an entirely new way.The individualized approachinvolves harvesting cancer patients immune cells, genetically engineering them, then returning them to patients bodies. The genetic engineering process aims torev up patients immune systemsto better fight cancer.

Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, said Dr. Scott Gottlieb, the FDA commissioner. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.

Novartis officials said it is working with 20 hospitals to provide Kymriah within a month. Eventually, the therapy will be offered at 32 sites, the company said. The first patients could be treated within days. The company is carefully training hospitals and staff to provide the treatment, which can cause a life-threatening immune reaction, as well as long-term complications.

Novartis said that it priced its drug based on several considerations. British health authorities have said a price of$649,000for a one-time treatment would be cost-effective given Kymriahs significant benefits. Novartis also considered the cost of bone-marrow transplants, which are currently given to many leukemia patients whose cancer relapses. Those transplants can cost up to $800,000, Novartis said.

Dr. Stephan Grupp, a researcher at Childrens Hospital of Philadelphia who helped test Kymriah in early studies, said he hopes the therapy could eventually replace bone-marrow transplants for these young patients. It would spare them serious and long-term side effects of a transplant, such as problems caused when the immune system attacks transplanted cells, he said. Some children whove received Kymriah have already received a transplant. For others, Kymriah serves as a bridge to transplant, keeping them alive longer enough to undergo this therapy.

An advocacy group called Patients for Affordable Drugs recently met with officials at Novartis to ask it to set a fair price for the drug, whose early development was supported by $200 million in federal research grants.

David Mitchell, a multiple myeloma patient and president of the advocacy group, described the drugs price tag as excessive.

Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more, he said. The drug pricing system in America is completely broken. Until policy in this country changes, the vicious cycle of patients struggling under high drug prices will continue.

The FDA also is considering a CAR T-cell therapy from California-based Kite Pharma. Gilead Sciences which has been criticized for the $84,000 price tag of its hepatitis C drug announced Monday that it will buy Kite for $11.9 billion.

Novartis officials said that they will offer a patient assistance program to help people with out-of-pocket costs.

Experts have noted that hidden costs could further add to patients financial burdens.

Beyond the cost of the procedure, patients would need to pay for traditional chemotherapy, which is given before CAR T-cell therapy to improve its odds of success. They would also have to foot the bill fortravel and lodging to one of the hospitals equipped to provide the high-tech treatment.

Because patients can develop life-threatening side effects weeks after the procedure, doctors will ask patients to stay within two hours of the hospital for up to a month. In New York, even budget hotels cost more than $200 a night an expense not typically covered by insurance. Patients who develop a dangerous complication, in which the immune system overreacts and attacks vital organs, might need coverage for emergency room care, as well as lengthy stays in the intensive care unit.

Doctors dont yet know what the full range of long-term side effects will be. CAR T-cell therapies can damage healthy immune cells, including the cells that produce the antibodies that fight disease. Some patients will need long-term treatments with a product called intravenous immunoglobulin, which provides the antibodies that patients need to prevent infection.

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Pioneering cancer gene therapy gets green light and $475,000 ... - Salon

FDA has approved the first gene therapy in the United States, tisagenlecleucel (KymriahNovartis), ushering in a new approach to treatment of cancer and other serious and life-threatening diseases, said the agency in a recent news release.

Tisagenlecleucel is the first autologous T-cell immunotherapy with genetically modified immune cells that target B-cell precursor acute lymphoblastic leukemia (ALL), the most common pediatric malignancy in children and young adults up to age 25 years. The product is indicated for patients who have treatment-resistant, relapsed, or refractory disease.

We consider this a major milestone in the development of cancer immunotherapies, said William Greene, PharmD, chief pharmaceutical officer at St. Jude Childrens Research Hospital, in a statement to pharmacist.com. It has the potential not only to improve outcomes, but also to reduce treatment-related complications for all cancer patients.

Approval of the product was based on a multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL, whose overall remission rate within 3 months of treatment was 83%.

Each dose of tisagenlecleucel is a customized treatment created using an individual patients own T-cells. The cells are collected and sent to a manufacturing center, where they are genetically modified to include a new gene that contains a chimeric antigen receptor (CAR). The CAR directs the T-cells to target and kill leukemia cells that have a CD19 antigen on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

The CD19-CAR was originally developed more than a decade ago at St. Jude by Dario Campana, MD, Greene said.

Adverse effects of tisagenlecleucel include serious infections, low blood pressure, acute kidney injury, fever, and decreased oxygen. The symptoms can appear within 1 to 22 days following infusion. The product was approved with a risk evaluation and mitigation strategy.

Tisagenlecleucel carries a boxed warning for severe, possibly life-threatening adverse effects such as neurological events and cytokine release syndrome (CRS), a systemic response to the activation and proliferation of CAR T-cells that causes high fever and flulike symptoms. FDA expanded the approval of tocilizumab (ActemraGenentech) to treat CRS and is requiring staff involved in prescribing, dispensing, or administering tisagenlecleucel to be trained to recognize and manage CRS and neurological events. Practitioners must have protocols in place to ensure that tisagenlecleucel is given to patients only after verifying that tocilizumab is available for immediate administration.

Optimal management [will require] careful planning for the provision of appropriate supportive care, Greene said. He also noted that approval of this therapy as a pharmaceutical presents interesting challenges to institutions in regard to handling, cost (budget implications), and reimbursement.

FDA is requiring Novartis to conduct a postmarketing observational study of patients treated with tisagenlecleucel.

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FDA approves first gene therapy in the United States - Pharmacy Today, American Pharmacists Association, pharmacist.com

In BriefResearchers healed bone fractures by attracting stem cells to the area and injecting a mix of microbubbles and DNA encoding a bone protein at the break. This method could replace bone grafting for nonhealing fractures.

Fixing broken limb bones after serious injuries can challenge even the most skilled orthopedic surgeons. Too much bone loss makes regrowth impossible, and even smaller fractures make bone growth problematic if the patient is in poor health or at an advanced age.

When physicians encounter these kinds of nonhealing fractures, autologous bone grafts are the gold standard for treatment. These bone grafts involve harvesting a segment of healthy bone, typically from the pelvis of the patient, which is then used to bridge the portion of the break that isnt growing new bone adequately. However, bone grafts are not always possible, depending on the patients health and the extent of the damage from the break.

Some doctors in recent years have started to try something new: incorporating bone morphogenetic proteins (BMPs) into bone implants to enhance healing. This isnt a sure thing, though. Through their traditional administration, BMPs come with significant side effects including bone formation in soft tissues and bone resorption.

These side effects might haveoccurred because BMPs wereadministered in large doses, so researchers came up with a new strategy: use gene therapy to deliver not the protein itself, but the underlying gene instead. This way the cells will get BMP at physiological levels solely at the site of the injury.

However, gettinggene therapiesinto the right cells isnt always easy. The genes are typically delivered using viral vectors, and these come with their own safety concerns. The researchers in this case used a relatively new delivery mechanism instead: sonoporation.

In sonoporation, an ultrasound is used to cause gas-filled microbubbles with lipid shells to oscillate and create tiny, easily repaired holes in cells. These tiny holes allow DNA for gene therapy to enter into the right place without affecting other areas. The next step was ensuring that the gene therapy targeted the correctcells. The team targeteda special form of stem cells that can become bone cells and produce BMPs proficiently.

The researchers trialled their new strategy in broken pig shinbones and found that the technique healed fractures after a single dose. They first inserted collagen scaffolds, because they attract the stem cells, and then waited for two weeks to allow the scaffolds to recruit sufficient numbers of stem cells.

Next, they injected a mix of microbubbles and BMP-encoding DNA at the fracture site, and applied an ultrasound pulse. The team then waited for eight weeks after the single instance of the gene therapy. The experimental fractures were healed, while the control animals fractures were not.

This innovative therapy could improve the recovery of millions of people around the world. While human trials must be conducted before we know whether hospitals should adopt the procedure,many of its components have shown enough promise for scientists to utilize them insimilar bone-healing experiments: One fracture-fixing strategy incorporates a specific form of BPM, and another therapy uses stem cells to revitalize bone growth.

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Soon, Broken Bones Could be Fixed Using Gene Therapy and Microbubbles - Futurism

Nightstar Therapeutics has filed to raise up to $86 million in a Nasdaq IPO. The money will equip Nightstar to complete a phase 3 trial of its choroideremia gene therapy and advance two other eye disease candidates through early-stage clinical studies.

London-based Nightstar, also known as NightstaRx, is set to move the choroideremia asset into phase 3 in the first half of next year. The therapy, NSR-REP1, is advancing into the 140-patient trial on the strength of data on 32 subjects treated in investigator-sponsored studies. Those trials found 90% of patients either maintained or improved their visual acuity in the year after receiving the gene therapy.

Given choroideremia causes currently-untreatable progressive vision loss, Nightstar sees the data as supporting further development. The asset is moving forward with a fairly clean safety profile in the 50 people treated to date. Investigators have seen one adverse eventtransient intraocular inflammationthat may have stemmed from treatment with NSR-REP1.

Challenges await Nightstar as it scales up for the phase 3 trial and potentially commercial sales, though. The biotech acknowledges the administration of NSR-REP1 requires significant skill and training, potentially creating a bottleneck to use of the gene therapy. And as a small, unpartnered player in a new field, any number of events could knock it off course.

What Nightstar does have is a head start. Spark Therapeutics has the most advanced challenger to NSR-REP1 but its program is yet to move past phase 1/2. The field is similarly clear for Nightstars follow-up candidate NSR-RPGR, which moved into the clinic just ahead of MeiraGTxs rival X-linked retinitis pigmentosa gene therapy. AGTCs Biogen-partnered candidate is close behind.

Nightstar has reached this point using money from a succession of private rounds, starting with the 12 million Syncona invested when the biotech spun out of the University of Oxford in 2014. The biotech pulled in a further 5 million when it named former Johnson & Johnson VP David Fellows as CEO early in 2015. A $35 million series B round followed late in 2015. And Nightstar broadened its investor base and raised a further $45 million in a series C round a few months ago.

Along the way, Nightstar has built out a team in preparation for the broadening of its clinical trial program and life on public markets. Last month, Ex-Pfizer clinical lead Tuyen Ong, M.D., left PTC Therapeutics to serve as chief development officer. And in April, Nightstar hinted at its IPO plans by recruiting the man who led Intercept Pharmaceuticals repeated public raises, Senthil Sundaram.

The question now is how receptive public investors are to gene therapy biotechs. The companies in the sector to go public to date have delivered mixed returns, with the successes of bluebird bio and Spark offset by the steady decline of uniQure and implosion of Dimension Therapeutics.

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Nightstar files for $86M IPO to fund gene therapy trials - FierceBiotech