• Qpex Biopharma launched with a $33 million Series A financing from New Enterprise Associates and joined by Adams Street Partners, LYZZ Capital, Hatteras Venture Partners and Stanford University Draper Fund. Notable: Congratulations to this group! With the science advancing, an expanding pipeline and the FDA and EMA on board with the technology, we see a backdrop that is favorable to an accelerating number of deals. Intellia Therapeutics (NTLA) Intellia Therapeutics (NASDAQ:NTLA) is also working on a sickle-cell … concept for their platform of cell therapy. 1 CRISPR: Clustered regularly interspaced short palindromic repeats; ZFN: Zinc finger nuclease; TALEN: transcription activator-life effector-based nucleases. The company's stock is down by 30% since the beginning of the year. And even American Gene Technologies,2 a private company, which is expected to go into the clinic in the first half of 2020, may be attractive to gene therapy buyers. Published as “Yes, Science Can Save Us.” in the May 2020 issue of Philadelphia magazine. Points: 25 Its STP705 therapy for non-melanoma skin cancer is currently in Phase II trials. Gene therapy relies on viruses as vectors to help transport therapeutic genes to a patient’s cells. The company said that it witnessed a "near-complete elimination" of VOCs in SCD patients during the trial. In short, it is worth investing in this biotech stock today. • Twentyeight-Seven’s lead program focuses on regulating levels of let-7, an important micro RNA that suppresses the translation of major oncogenes.

Sharpening the efficacy of gene therapy for nervous-system diseases. The drug was discontinued just five years after it was first approved in 2012. As few as five percent of people with glioblastoma survive five years beyond a diagnosis; this is the cancer that killed U.S. • The company has two compounds in Phase II clinical trials, one for Parkinson’s and another for epilepsy, and a compound in Phase I for psychosis. The company is working … This can vary dramatically from the date the company was founded, which is typically the date incorporation papers were filed. Following these unfortunate events, interest in the area of gene therapy evaporated. Launched: 2019 From Kite Pharma’s announcement of its new state-of-the-art cell manufacturing facility in Frederick County, Maryland, and the opening of the 32,000 square foot JLABS @ Washington, D.C. to Viela Bio’s $150M IPO and Paragon Bioservices $1.2B acquisition by Catalent, 2019 was an exciting year for so many companies and people in the region. While many with HIV avoid taking the medicines in the early stage of the disease due to cost and side effects, the possibility of a cure would drive substantial demand. During the first quarter, CRISPR Therapeutics recorded a net loss of $69.7 million, but the company also had a cash and cash equivalents balance of $889.7 million as of March 31. emerge in the new year? With a deep and Bristol Myers Squibb, for example holds over $32 billion in cash and maintains an interest in gene therapy while others such as Biogen (NASDAQ: BIIB), Gilead (NASDAQ: GILD) and Sanofi (NASDAQ: SNY) all have reasons to find attractive partners and acquisitions in genomic medicine. Founded: 2016Funding: $59 millionTeam: 30Up next: Carisma plans to initiate a clinical trial for certain types of breast, lung and gastric cancers. Launched: 2018 Combining our unique skill sets opens a new chapter of promise and possibility for gene therapy.” — Jeff Marrazzo, CEO, Targeting solid and liquid cancer tumors with next-generation cell therapies. Launched: 2018 tumors as well as autoimmune indications. And even American Gene Technologies, 2 a private company, which is expected to go into the clinic in the first half of 2020, may be attractive to gene therapy buyers. of the immune system’s tumor-targeting receptors to address both newly Scientists can use the cells in this form to develop any kind of cell needed for therapeutic purposes. This product is being developed as a potential treatment for two blood disorders called sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The environment for genomic medicine M&A appears to be favorable with advances in science, regulatory receptiveness to the class, plenty of cash on big pharma balance sheets and a need to add growth opportunities to their portfolio. © 1985 - 2020 BioSpace.com. variegated pipeline and strong backing from both U.S. and Chinese investors, Location: Boston, MA Founded: 2015Funding: $231 millionTeam: 60Up next: Tmunity expects to continue enrolling patients in clinical trials to further test its latest approach: targeting a sugar that’s uniquely expressed in tumor cells to develop more effective cancer therapies. In my view, Bluebird's stock will rebound in the coming months as the company's revenue increases. The company announced that the first patient treated with TDT was still transfusion-independent 15 months after receiving the treatment, and the SCD patient who was first treated with CTX001 remained free of VOCs nine months after the treatment. two of its potential therapies for liver cancer and liver fibrosis have Orphan Drug Status. If you're a human and see this, please ignore it. Gene therapy players like Sarepta Therapeutics , UniQure and Novartis are also competing in this arena. Notable: Points: 15


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