Category Archives: Gene Therapy

Krystal Biotech has filed to raise about $35 million in a Nasdaq IPO. The money will equip Krystals ex-Intrexon executive team to file an IND for its rare skin disease gene therapy in the first quarter of next year. That gene therapy, KB103, is designed to treat dystrophic epidermolysis bullosa, a rare disease that makes the skin fragile and susceptible to blisters and tears. Krystal thinks it can improve outcomes in these patients, who currently only have palliative options, by using a HSV-1-based vector to fix defects in the COL7A1 gene. Pittsburgh, Pennsylvania-based Krystal has yet to back up the idea with clinical data. But having run preclinical tests of topical and intradermal formulations of KB103 and met with the FDA late last year, it plans to embark on a whistle-stop development program. The plan is to file an IND in the first quarter, have results by the middle of the year and use that readout to guide the design of a pivotal trial. Krystal will need more than the forecast IPO haul to execute that strategy, although it does expect to have some money left over from the Nasdaq listing to fund work on its follow-up program, KB104. That … Continue reading →

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apic Bio, Inc., a pre-clinical stage gene therapy company leveraging its proprietary platform to advance therapies to treat rare diseases with complex mechanisms, in particular Alpha-1 Antitrypsin Deficiency (Alpha 1), launched today with an initial investment led by the venture philanthropy arm of the Alpha-1 Foundation and a private investor with the disease. Its lead product, APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. APB-101 has achieved a pre-clinical proof of concept with efficacy demonstrated in vitro and in vivo. It is currently undergoing pre-clinical GLP toxicology studies in non-human primates. Patients living with Alpha 1 lack sufficient levels of circulating AAT protein to protect lung tissue against damage from proteases, and experience the accumulation of mutant AAT polymers in the liver. Clinically, the deficiency is manifested by progressive emphysema and the accumulation presents a significant risk of liver cirrhosis. John Reilly, Co-Founder & President said: We are grateful to TAP and A1AT Investors, LLC who have supported the successful start of Apic Bio by providing the first tranche of our seed financing round allowing us to secure key intellectual property rights and … Continue reading →

A darling British biotech that is working on the development of the worlds leading gene therapy for cancer has posted reduced losses and a leap in sales. Oxford BioMedica signed a deal worth up to $100m (77m) with Swiss drugs giant Novartis last month to supply cell material for its potential blockbuster treatment, known as CTL019, for a type of leukaemia. The living drug was recommended for approval by US regulators in June, with a final determination expected this autumn. It would be the first gene therapy for cancer and if approved is expected to generate a spike in revenues for Oxford BioMedica, with the potential for further tie-ups on other cancer treatments. Oxford BioMedica is also trialling its lentiviral vectors, which help manipulate genes, in potential treatments for Parkinsons Disease and an eye condition. In half-year results today, Oxford BioMedica pared back losses to 2.2m, compared to 6.9m in the red the previous year. Revenues jumped 26pc to 15.7m. More here: Gene therapy darling Oxford BioMedica pares losses - Telegraph.co.uk … Continue reading →

Investment Thesis With an undervalued stock, a critically-acclaimed drug leading in its space and a barrage of financial and expertise-based backing, Sangamo Therapeutics (NASDAQ:SGMO) is the ideal vehicle for exposure to a market with high growth potential and a hedge against the antiquated methods (CRISPR/Cas9) of treating hemophilia. The same method has also been found to work to a high degree of accuracy on other genetic disorders. Sangamo Therapeutics' accomplishments to date have helped to draw integrity to its operations and raise its profile against common competitors in the space (QURE, GSK, ONCE, BMRN, BLUE, BIVV (a pure-play hemophilia company). Principally, Sangamo has secured an exclusivity agreement (and subsequent vote of confidence) from Pfizer (NYSE:PFE) (also once a competitor) regarding the development and commercialization of gene therapy programs for hemophilia A (SB-525), which affects 85% of hemophilia sufferers (Nelson Pediatrics), bagging $70 million upfront and a potential $475 million in milestones and royalties (Reuters). (The Zinc Finger Protein Nuclease technology method adopted is targeted at diseases which are caused by genetic defects, as their removal is known to respond best to gene editing targeting, in particular, tauopathies, thalassemias, hemophilia, and HIV/AIDS). The company's strong balance sheet was further bolstered by … Continue reading →

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. The pre-clinical tests on animal models, clinical trials and the registration procedure may take up to 10 years, senior research fellow at the Epidemiology Central Research Institute of Rospotrebnadzor (the Federal Service on Surveillance for Customers Rights Protection and Human Well-Being) Dina Glazkova told TASS. "This is not about the next year, but rather in five years, at the earliest. It takes up to 10 years on the average," she said. Glazkova reiterated that the registration is made after the clinical trials. "Again, the clinical trials are costly, and the drug production is costly as well," the scientist added. Dinavir proved to be safe while tested on cells, in vitro. A Phase II pre-clinical trial will utilize animal models to test the efficiency and safety of treatment. A Phase I clinical trial will be carried out on humans to test safety of the therapy and will take up not less than a year. "Phase II takes up two to three years, and it is unclear how much will be required … Continue reading →

Imagine only being able to see the things in front of you in soft focus, and just in black and white. For people with the genetic eye condition achromatopsia those are just some of the side effects. But, researchers are testing a new treatment designed to cure the condition by fixing the gene responsible. Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun. I need to have very dark, very tinted sunglasses to feel comfortable outside and to see really well. Cataldo said. Cataldo has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light. She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance. I cannot drive a car so I rely on public transportation and my bike to get around. Cataldo explained. "There are currently no approved and no effective treatments for achromatopsia, said University of Florida surgical ophthalmologist Christine Kay. Kay is working to change that. She is one of a handful of experts testing a gene therapy. For achromatopsia the cells we have to target are cone cells responsible for … Continue reading →

Human protein-coding genes number from 20,000 up to 25,000. If just one of these genes gets altered or a code gets missing, it can be fatal to an individual. In fact, approximately 30 per cent of infant mortality at birth in developed countries are caused by genetic disease. Almost 50 per cent of all miscarriages worldwide are due to chromosomally defective fetus. Furthermore, according to the World Health Organization, over 10,000 human diseases are linked to single gene mutation alone. Among these monogenic diseases are thalassaemia, sickle cell anemia, haemophilia, Fragile-X syndrome, cystic fibrosis, and Huntingtons disease. The other two major types of genetic disorders are chromosomal and complex disorder, where theres mutation in two or more genes. Genetic disease is not also simply inherited, our environment is another factor that can trigger mutation. Cancer, diabetes, and heart disease are classified as multifactorial inheritance genetic disorders. Considering all these, one would expect that the world will be welcoming the revolutionary gene therapy with wide-open arms. Yet, UniQures Glybera has been recently withdrawn from the European market in spite of its promising one-time cure for lipoprotein lipase deficiency (LPLD). LPLD is a rare genetic disorder characterized by the bodys lack of … Continue reading →

LONDON, Aug 8 (Reuters) - Gene therapy, which aims to patch faulty genes with working DNA, has been a long time in development. The following are major milestones: 1972 - Researchers first suggest gene therapy as a treatment for genetic diseases but oppose its use in humans "for the foreseeable future", pending greater understanding of the technology. 1990 - A four-year-old girl with severe immunodeficiency became the first patient to undergo gene therapy in the United States. 1999 - American patient Jesse Gelsinger dies following a gene therapy experiment, setting the field back several years as U.S. regulators put some experiments on hold. 2002-03 - Cases of leukaemia are diagnosed in French children undergoing gene therapy in a further blow to the field. 2003 - The world's first gene therapy is approved in China for the treatment of head and neck cancer. 2007 - Doctors carry out the world's first operation using gene therapy to treat a serious sight disorder caused by a genetic defect. 2012 - Europe approves Glybera, the first gene therapy in a Western market, for an ultra-rare blood disorder. 2016 - Europe approves Strimvelis for a very rare type of immunodeficiency. 2017 or 2018 - The … Continue reading →

Researchers from the University of Chicago have successfully completed a proof-of-concept studywhere they managed to treat obesity in mice using a new type of gene therapy that utilized skin transplants. Human skin transplantation is a well-established clinical approach that has been used for the treatment of burns. However, using it as a vehicle to deliver genetic treatments for non-skin diseases could be revolutionary. There are several reasons why skin stem cell therapy can be applicable to a broad type of diseases. The skin is the largest human organ, providing an easy access to cells needed for genetic treatments. The skin enables easy monitoring for potential off-target mutations resulting from the CRISPR intervention, as well as easy removal of such mutations, should they occur. Most importantly, proteins that are secreted by epidermal cells can reach the blood circulation and achieve desired therapeutic effects for the entire body. In the study published this month, titledEngineered Epidermal Progenitor Cells Can Correct Diet-Induced Obesity and Diabetes,the scientists genetically engineered skin cells to be able to deliver GLP1 (glucagon-like peptide 1) - a hormone which regulates blood glucose. Then they developed a surgical procedure which allowed them to successfully engraft the new skin onto a … Continue reading →

Pharmaceutical giant Pfizer is expanding its facilities in Sanford to accommodate the companys push into gene therapy. The state Department of Commerce announced earlier this week that Pfizer would invest $100 million in the site and create 40 jobs there within three years. The average annual salary for the new positions will be $97,500, much higher than Lee Countys average annual wage of $38,250. If it meets those goals, Wyeth Holdings, a wholly owned subsidiary of Pfizers, will receive a $250,000 grant from the One North Carolina Fund and a local incentive of up to $1,412,715 over five years. The announcement comes a week after Gov. Roy Cooper visited Pfizers facilities in Sanford and a year after Pfizer bought Bamboo Therapeutics, a Chapel Hill startup. Pfizer also bought Bamboos gene therapy manufacturing facility, which Bamboo had acquired from UNC-Chapel Hill in January, 2016. Gene therapy is an emerging technology that attacks the disorder by repairing mutated genes. Pfizer will use technology to introduce genetic material into a patients body so as to compensate for defective or missing genes. Link: Pfizer's push into gene therapy adds more jobs in Sanford - News & Observer … Continue reading →