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Biotech News

Biotech Announces Encouraging HIV Vaccine Results
May 23, 2018 ( Newswire) Ram Selvaraju, an analyst with H.C. Wainwright & Co., explained the recently released data from a study of one of this immunotherapy firm's therapeutics.

Analyst: 'New CEO, Larger Sales Team and Greater SUI Focus Should Help Medical Device Growth'
May 23, 2018 ( Newswire) Cowen analyst Dr. Joshua Jennings highlighted this medical device firm’s Q1/18 developments and financial results.

Cancer in the Crosshairs: IMV Looks to Deliver Immunotherapy a New Way
May 16, 2018 ( Newswire) In this interview with The Life Sciences Report, IMV Inc.'s CEO Fred Ors and Pierre Labb, chief financial officer, describe the innovative immunotherapy platform technology that has attracted partnerships with big pharma, prompted reorganization of the company, and holds long-term promise for cancer patients.

Biotech's Launch of Pain Product Shows Encouraging Early Metrics
May 14, 2018 ( Newswire) Francois Brisebois, an analyst with Laidlaw & Co., reviewed this firm's progress since commercially debuting its primary asset.

Cell Therapy Firm Finalizing $5.1M Strategic Investment and Partnership with Chinese Company
May 9, 2018 ( Newswire) The terms of a proposed agreement between this cell therapy company and YOFOTO, a Chinese health and consumer products company, have been revised and will result in a $5.1 million investment.

ChroMedX (CSE: CHX) (OTCQB: CHXIF) (FSE: EIY2) Signs Deal to Acquire UX Data Sciences Corp.
Toronto, Ontario - May 9, 2018 (Newsfile Corp.) ( Newswire) ChroMedX Corp. (CSE: CHX) (OTCQB: CHXIF) (FSE: EIY2) (the "Company"), developer of the HemoPalm Handheld Blood Analyzer Platform, is pleased to announce that it has entered into a binding Letter-of-Intent ("LOI") to acquire Ottawa, Ontario based medtech data science company, UX Data Sciences Corp. ("UXD").

CVR (TSXV: CVM) (FSE: B3BN) (OTCQB: CRRVF) & U.S. FDA: A Meeting of the Minds
Vancouver, British Columbia - May 9, 2018 (Newsfile Corp.) ( Newswire) CVR Medical Corp. (TSXV: CVM) (FSE: B3BN) (OTCQB: CRRVF) ("CVR Medical") announces the receipt of official meeting minutes for their Pre-Submission meeting on March 23, 2018 with the U.S. Food and Drug Administration (FDA) regarding the Carotid Stenotic Scan (CSS) device and it's upcoming FDA submission for market release.

ChroMedX (CSE: CHX) (OTCQB: CHXIF) Reports on HemoPalm Techno-Commercial Analysis and Productization Plan
Toronto, Ontario - May 8, 2018 (Newsfile Corp.) ( Newswire) ChroMedX Corp. (CSE: CHX) (OTCQB: CHXIF) (FSE: EIY2) (the "Company") developer of the HemoPalm Handheld Blood Analyzer Platform, is pleased to report on HemoPalm activities.

Tom J. Harris Former CFO Time at Warner Cable And Associated Press Appointed Chief Financial Officer Of CVR Medical (TSXV: CVM) (FSE: B3BN) (OTCQB: CRRVF)
VANCOUVER, British Columbia - May 3, 2018 (Newsfile Corp.) ( Newswire) CVR Medical Corp. (TSXV: CVM) (FSE: B3BN) (OTCQB: CRRVF) ("CVR Medical") has appointed Mr. Tom J. Harris to the position of Chief Financial Officer effective May 1, 2018.

Novoheart (TSXV:NVH) (FWB:3NH) to Debut New, State-of-the-art 'Human Heart-in-a-Jar 2.0' Technology at the 2018 International Society for Stem Cell Research (ISSCR) Annual Meeting
VANCOUVER, British Columbia - May 2, 2018 ( Newswire) Novoheart ("Novoheart" or the "Company") (TSXV:NVH) (FWB:3NH) today announced that the next generation of its 'Human heart-in-a-jar' technology - which makes up a key component of its comprehensive MyHeartTM Platform

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Biotech Stock ETF's

First Trust NYSE Arca Biotech ETF ( NYSEArca: FBT ) The investment seeks investment results that correspond generally to the price and yield (before the fund's fees and expenses) of an equity index called the NYSE Arca Biotechnology Index(SM). The fund will normally invest at least 90% of its net assets plus the amount of any borrowings for investment purposes in common stocks that comprise the index. The index is an equal-dollar weighted index designed to measure the performance of a cross section of companies in the biotechnology industry that are primarily involved in the use of biological processes to develop products or provide services. The fund is non-diversified.

iShares US Healthcare ( NYSEArca: IYH ) The investment seeks to track the investment results of an index composed of U.S. equities in the healthcare sector. The fund generally invests at least 90% of its assets in securities of the underlying index and in depositary receipts representing securities of the underlying index. It seeks to track the investment results of the Dow Jones U.S. Health Care Index (the "underlying index"), which measures the performance of the healthcare sector of the U.S. equity market. The fund is non-diversified.

iShares US Healthcare Providers ( NYSEArca: IHF ) The investment seeks to track the investment results of an index composed of U.S. equities in the healthcare providers sector. The fund generally invests at least 90% of its assets in securities of the underlying index and in depositary receipts representing securities of the underlying index. It seeks to track the investment results of the Dow Jones U.S. Select Health Care Providers Index (the "underlying index"), which measures the performance of the healthcare providers sector of the U.S. equity market. The fund is non-diversified.

iShares US Medical Devices ( NYSEArca: IHI ) The investment seeks to track the investment results of an index composed of U.S. equities in the medical devices sector. The fund seeks to track the investment results of the Dow Jones U.S. Select Medical Equipment Index (the "underlying index"), which measures the performance of the medical equipment sector of the U.S. equity market. The underlying index includes medical equipment companies such as manufacturers and distributors of medical devices such as magnetic resonance imaging (MRI) scanners, prosthetics, pacemakers, X-ray machines, and other non-disposable medical devices. The fund is non-diversified.

iShares Nasdaq Biotechnology ( NasdaqGIDS IBB ) The investment seeks to track the investment results of an index composed of biotechnology and pharmaceutical equities listed on the NASDAQ. The fund generally invests at least 90% of its assets in securities of the underlying index and in depositary receipts representing securities of the underlying index. The underlying index contains securities of NASDAQ® listed companies that are classified according to the Industry Classification Benchmark as either biotechnology or pharmaceuticals and that also meet other eligibility criteria determined by the NASDAQ OMX Group, Inc. The fund is non-diversified.

Market Vectors Biotech ETF ( NYSE MKT:BBH ) seeks to replicate as closely as possible, before fees and expenses, the price and yield performance of the Market Vectors® US Listed Biotech 25 Index. The fund normally invests at least 80% of its total assets in securities that comprise the fund's benchmark index. The Biotech Index is comprised of common stocks and depositary receipts of U.S. exchange-listed companies in the biotechnology sector. Such companies may include medium-capitalization companies and foreign companies that are listed on a U.S. exchange. It is non-diversified.

PowerShares Dynamic Biotech & Genome ETF ( NYSEArca: PBE ) The investment seeks investment results that generally correspond (before fees and expenses) to the price and yield of the Dynamic Biotechnology & Genome IntellidexSM Index. The fund generally will invest at least 90% of its total assets in common stocks of biotechnology companies and genome companies that comprise the underlying intellidex. The underlying intellidex was composed of common stocks of 30 U.S. biotechnology and genome companies. These companies are engaged principally in the research, development, manufacture and marketing and distribution of various biotechnological products, services and processes, etc. It is non-diversified.

Biotech/ Pharma Stock Directory


4SC (XETRA: VSC.DE) is an innovative biotech company with a strong focus on clinical development. We discover and develop targeted small molecule drugs with an epigenetic mode of action for the treatment of cancer in indications with a high unmet medical need and major economic potential.

Aastrom Biosciences, Inc. (NasdaqCM: ASTM) is the leader in developing patient-specific expanded cellular therapies for use in the treatment of patients with severe diseases and conditions. Aastrom markets two autologous cell therapy products in the United States for the treatment of cartilage repair and skin replacement. Aastrom is also developing MACI(TM), a third-generation autologous chondrocyte implant for the treatment of cartilage defects in the knee, and ixmyelocel-T, a patient-specific multicellular therapy for the treatment of advanced heart failure due to ischemic dilated cardiomyopathy.

ABBOTT LABORATORIES (NYSE: ABT) is a global healthcare company devoted to improving life through the development of products and technologies that span the breadth of healthcare. With a portfolio of leading, science-based offerings in diagnostics, medical devices, nutritionals and branded generic pharmaceuticals, Abbott serves people in more than 150 countries and employs approximately 69,000 people.76

Abeona Therapeutics Inc. (NasdaqCM:ABEO) develops and delivers gene therapy and plasma-based products for severe and life-threatening rare diseases. Abeona's lead programs are AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA). We are also developing ABO-201 (AAV CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. In addition, we are also developing rare plasma protein therapies including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD using our proprietary SDF™ (Salt Diafiltration) ethanol-free process.

Ablynx (Brussels: ABLX.BR) is a biopharmaceutical company engaged in the discovery and development of Nanobodies®, a novel class of therapeutic proteins based on single-domain antibody fragments, for a range of serious and life-threatening human diseases, including inflammation, thrombosis, oncology and pulmonary disease.

Acadia Pharmaceuticals Inc. (NasdaqGS: ACAD) is a biopharmaceutical company focused on the development and commercialization of innovative medicines that address unmet medical needs in neurological and related central nervous system disorders. ACADIA has a pipeline of product candidates led by pimavanserin, for which we have reported positive Phase III trial results in Parkinson's disease psychosis and which has the potential to be the first drug approved in the United States for this disorder. We are currently completing NDA-enabling clinical and manufacturing activities for pimavanserin and are planning to submit an NDA with the FDA near the end of 2014. Pimavanserin is also in Phase II development for Alzheimer's disease psychosis and has successfully completed a Phase II trial in schizophrenia. ACADIA also has clinical-stage programs for chronic pain and glaucoma in collaboration with Allergan, Inc. and two preclinical programs directed at Parkinson's disease and other neurological disorders. All product candidates are small molecules that emanate from internal discoveries.

Access Pharmaceuticals, Inc. (OTC:ACCP) is an emerging biopharmaceutical company that develops and commercializes proprietary products for the treatment and supportive care of cancer patients. Access developed MuGard and ProctiGard and is developing multiple follow-on products. Access also has other advanced drug delivery technologies including CobaCyte™-mediated targeted delivery and CobOral-oral drug delivery, its proprietary nanopolymer delivery technology based on the natural vitamin B12 uptake mechanism.

AcelRx Pharmaceuticals, Inc. (NasdaqGM:ACRX) is a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of moderate-to-severe acute pain. The Company's late-stage pipeline includes ARX-04 (sufentanil sublingual tablet, 30 mcg), designed for the treatment of moderate-to-severe acute pain in medically supervised settings; and Zalviso® (sufentanil sublingual tablet system), designed for the management of moderate-to-severe acute pain in adult patients in the hospital setting. Zalviso delivers 15 mcg sufentanil sublingually through a non-invasive delivery route via a pre-programmed, patient-controlled analgesia device. Zalviso is approved in the EU as well as Norway, Iceland, and Liechtenstein and is investigational and in late-stage development in the U.S. Grunenthal Group holds the rights for Zalviso in Europe and Australia, while AcelRx retains all other world-wide rights.

Acorda Therapeutics, Inc. (NasdaqGS:ACOR) is a biotechnology company focused on developing therapies that improve the lives of people with neurological disorders. Acorda markets three FDA-approved therapies including: AMPYRA (dalfampridine) Extended Release Tablets, 10 mg, a treatment to improve walking in patients with multiple sclerosis (MS); ZANAFLEX CAPSULES® (tizanidine hydrochloride) and Zanaflex tablets, a short-acting drug for the management of spasticity; and QUTENZA® (capsaicin) 8% Patch, for the management of neuropathic pain associated with postherpetic neuralgia. AMPYRA is marketed outside the United States as FAMPYRA® (prolonged-release fampridine tablets) by Biogen Idec under a licensing agreement from Acorda. Acorda has one of the leading pipelines in the industry of novel neurological therapies. The Company is currently developing six clinical-stage therapies and one preclinical stage therapy that address a range of disorders including post-stroke deficits, epilepsy, stroke, peripheral nerve damage, spinal cord injury, neuropathic pain, and heart failure.

Acrux Limited (ASX:ACR.AX) is a dynamic Australian drug delivery business developing and commercialising a range of patient-preferred pharmaceutical products for global markets, using innovative, patented technology to administer drugs through the skin. Fast drying, non-occlusive topical sprays or liquids provide an enhanced transdermal delivery platform with low or no skin irritation, superior cosmetic acceptability, and simple, accurate and flexible dosing.

See the full stock directory here

Biotech News from Global Newswire

Seeing Is Believing: A Gene Therapy First

Ligandal’s Bioinspired Nanomaterials Deliver CRISPR into Nucleus of a T-Cell

SAN FRANCISCO, May 23, 2018 (GLOBE NEWSWIRE) -- For the first time, a ligand-targeted nanoparticle has been successfully used to deliver CRISPR into the nucleus of a T-cell.

“From the start, CRISPR and other genetic therapies have suffered from the classic ‘last-mile’ problem,” said Andre Watson, founder, chairman and CEO of Ligandal. “For all their power to heal, genetic therapies are ineffective at best if they cannot be delivered to the precise cellular subtypes and locations inside cells.

“Now they can be,” Watson said.

Ligandal’s bioinspired nanomaterials utilize peptide targeting ligands, which bind to precise cellular surface profiles of targeted cell populations, to target CRISPR and other genetic materials into T-cells or other cells. The ligand targeting capacity is an industry first for these gene therapy modalities: Ligandal’s technology can selectively target T-cells with manifold greater efficiency than other blood cell populations.

“In part, the flexibility and ease of this platform is enabled by state-of-the-art peptide synthesis and fluid handling robotics, driven by a back-end machine-learning approach to rapid ligand and nanomaterials discovery,” Watson said. “Of course, none of these robotics or machine learning approaches would be meaningful without our breakthroughs in bioinspired nanomaterials design and synthesis.”

In addition to Ligandal’s breakthroughs in high-throughout biological nanomaterials screening, the company has demonstrated that its first-in-class ligand-targeted particles are efficiently performing gene edits.

In a representative image, Ligandal documented the delivery of CRISPR to a T-cell nucleus via 3D super-resolution microscopy. Such an image has never been shown for a nanoparticle delivering a gene editing tool, and Ligandal is the first to demonstrate ligand-targeting of CRISPR via a non-viral construct. Furthermore, these particles are serum stable, forming the basis of a breakthrough approach to in vivo gene therapy.

“This is just the start,” Watson said. “Our novel imaging pipeline coupled to our leading genomics, peptide synthesis, gene editing and biological nanomaterials expertise will ramp up production by giving us real-time quantification of nanoparticle uptake and gene-editing efficiency for thousands of nanoparticle variants per week. Most importantly, this infrastructure is forming the basis of the first diagnostically-responsive nanomaterials lab the world has ever seen. We are pleased to have brought on leading scientists in the field to drive us forward on our mission of creating curative, scalable, cost-effective genetic therapies for humanity at large.”

Ligandal is now working to program T-cells to target markers of viruses and cancer cells, as well as developing a novel set of nano-immunotherapies with no industry precedent. Current data shows that Ligandal’s approach can differentially target human primary T-cells within mixed blood cell populations, delivering CRISPR and other genetic tools with high efficiency and performing gene edits. The company has also generated compelling data in bone marrow and additional blood models.

Watson gave thanks to Shuailiang Lin, PhD, for his novel genome engineering work; Ryan Spencer, PhD, for his peptide engineering breakthroughs; Matthew Dobbin, PhD’s for his novel computational imaging pipeline; and Kola Awe for his computational robotics and nanomaterials design pipeline. The company also thanks additional team members involved in the project.

Overall, Ligandal is developing a complete platform to unlock the human genome to tackle multiple complex diseases to free humanity from the pain and suffering caused by each disease.

Based in San Francisco, Ligandal is a nanomedicine company developing unique curative genetic therapies with a personalized nanotechnology platform. Visit to learn more.

Contact: Vinh Vuong
+1 814-357-5445

ProQR Appoints Yi-Tao Yu, Ph.D., to Its Scientific Advisory Board

LEIDEN, the Netherlands, May 23, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced the appointment of Dr. Yi-Tao Yu to the company's scientific advisory board.

"Dr. Yu is a pioneer of targeted RNA editing to correct protein translation defects caused by premature termination codons,” said Daniel de Boer, chief executive officer of ProQR. "We welcome Dr. Yu to our scientific advisory board, where his expertise will help guide and inform our development of therapies for rare genetic diseases based on novel RNA editing technologies including our Axiomer® technology."

Dr. Yu is a professor of biochemistry and biophysics at the University of Rochester Medical Center. His research focuses on understanding the role of post-transcriptional RNA editing in regulating cellular processes, such as telomere regeneration, pre-mRNA splicing and protein coding capacity of mRNAs. He is the author of over 70 peer-reviewed scientific articles, and he has served as a member or reviewer in a number of scientific, government and patient organizations, including the National Institutes of Health, the National Science Foundation, the Gilbert Family Foundation and the RNA Society. Dr. Yu received his Ph.D. from Case Western Reserve University and conducted his postdoctoral studies at Yale University School of Medicine.

"Targeted RNA editing technology has the potential to develop transformative therapies for genetic conditions," said Dr. Yu. "I look forward to working with ProQR as they continue to develop new therapeutics for patients with rare diseases that do not currently have treatment options."

About Axiomer® Technology Platform

ProQR is pioneering a next-generation RNA technology called Axiomer®, which could potentially yield a new class of medicines for genetic diseases. Axiomer® “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® EONs are designed to recruit an endogenously expressed RNA editing system called ADAR, which it can direct to the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*


This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding our SAB members, and statements regarding our product candidates, including our development and their therapeutic potential. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

ProQR Therapeutics N.V.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431

Fibrocell Reports on Interim Results and Progress of Phase 1/2 Clinical Trial of FCX-007 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

– FCX-007 well-tolerated up to 52 weeks post-administration –

– Continued positive trends noted in wound healing and pharmacology signals, including
type VII collagen expression and evidence of anchoring fibrils –

– Company to Host Conference Call & Webcast Today at 8:30 am EDT–

EXTON, Pa., May 21, 2018 (GLOBE NEWSWIRE) -- Fibrocell Science, Inc. (NASDAQ:FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today provided an update on the interim results and progress of its Phase 1/2 clinical trial of FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).  The results were presented by M. Peter Marinkovich, MD, the trial’s Study Director at the Stanford University site and Associate Professor of Dermatology at the Stanford University School of Medicine, at the 7th International Investigative Dermatology meeting on May 19, 2018.   

Four adult patients (n=7 wounds) aged 20 to 37 have been dosed with FCX-007 in the margins of and across targeted wounds, as well as in separate intact skin sites.  Three patients received a single intradermal injection session at baseline.  One patient received a second injection session in the remaining unhealed areas of wounds at 25 weeks post-administration, as allowed by the clinical trial protocol.    

Safety data from these patients show FCX-007 was well-tolerated up to 52 weeks post-administration. There were no serious adverse events and no product related adverse events reported.  No type VII collagen (COL7) autoantibody response was noted.

Various COL7 expression signals were detected throughout the data set using either immunofluorescence (IF) or immunoelectron microscopy (IEM) up to 52 weeks post-administration.  Anchoring fibril structures have also been observed using IEM.

Wounds were evaluated during a monitoring period prior to dosing and they were observed to be open for up to eight months. Compared to the baseline measurement collected at Day 0 before the administration of FCX-007, the percentage of dosed wounds healing > 50% when compared to baseline were observed as follows:

- 100% (7/7) at 4 weeks post-administration
- 86% (6/7) at 12 weeks
- 67% (2/3) at 25/32 weeks
- 100% (1/1) at 52 weeks

A similar trend was also observed for treated wounds healing > 75% when compared to baseline.  Untreated wounds of similar size to the treated wounds were selected and monitored as controls on each patient.  The percentage of untreated control wounds healing > 50% when compared to baseline were observed as follows:

- 14% (1/7) at 4 weeks post-administration
- 17% (1/6) at 12 weeks
- 0% (0/2) at 25/32 weeks
- 0% (0/1) at 52 weeks

“The Phase 1 portion of the trial of FCX-007 continues to be encouraging and reinforces the potential for treating RDEB patients,” said Alfred Lane, MD, Chief Medical Advisor of Fibrocell and Professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine.  “As we move into Phase 2 of the trial, I am looking forward to incorporating these learnings into the trial and determining the impact on patient outcomes.”

There is one patient enrolled in the Phase 2 portion of the trial, with three additional screening visits scheduled prior to the end of June 2018.  Enrollment of six patients is expected to be completed in the third quarter of 2018.

“We are pleased with the continued progress of our FCX-007 program that offers promise to be transformative for RDEB patients,” said John Maslowski, President and Chief Executive Officer of Fibrocell. “Based on safety, pharmacology and wound healing data, we plan to continue exploring dose range and administration for future patients. We are looking forward to moving ahead with this protocol that advances the clinical trial of FCX-007 and positions us to achieve future milestones for the program.”

Fibrocell is developing FCX-007 in collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon Corporation (NYSE:XON), a leader in synthetic biology.

Fibrocell Conference Call & Webcast

On Monday, May 21, 2018 at 8:30 a.m. EDT, Fibrocell will host a conference call and webcast to discuss the interim results and progress of the Phase 1/2 clinical trial of FCX-007.  A slide presentation summarizing the data will be referenced during the call and is posted in the Investors section of the Company’s website under the event, “Fibrocell FCX-007 Conference Call & Webcast.”  Following the presentation, there will be a question-and-answer session with John Maslowski, President and CEO of Fibrocell, and Alfred Lane, MD, Chief Medical Advisor of Fibrocell and Professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine.

To participate on the live call, please dial 866-548-4713 (domestic) or +1-323-794-2093 (international) and provide the conference code 5629866. The conference call will also be webcast live from the Investors section of Fibrocell's website at  under the event, “Fibrocell FCX-007 Conference Call & Webcast,” and will be archived there for 30 days.

About FCX-007

FCX-007 is Fibrocell's clinical-stage, gene therapy product candidate for the treatment of RDEB, a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7 and is being developed in collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon Corporation. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treating wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution. FCX-007 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track Designations by the U.S. Food and Drug Administration. 

About the Phase 1/2 Clinical Trial

The primary objective of this open-label clinical trial is to evaluate the safety of FCX-007 in RDEB patients.  Additionally, the trial is assessing wound healing and pharmacology at 4, 12, 25 and 52 weeks post-administration.  Six patients ages seven and older are targeted to be treated with FCX-007 in the Phase 2 portion of the trial. To learn more about the clinical trial, please visit and search the identifier NCT02810951.

About Fibrocell  

Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue.  Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase 1/2 clinical trial for the treatment of RDEB. Fibrocell is also developing FCX-013, the Company’s product candidate for the treatment of moderate to severe localized scleroderma.  Fibrocell’s gene therapy portfolio is being developed in collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon Corporation (NYSE:XON), a leader in synthetic biology.  For more information, visit or follow Fibrocell on Twitter at @Fibrocell.


Fibrocell, the Fibrocell logo, and Fibrocell Science are trademarks of Fibrocell Science, Inc. and/or its affiliates.  All other names may be trademarks of their respective owners.

Forward-Looking Statements

This press release contains, and our officers and representatives may from time to time make, statements that are “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. All statements that are not historical facts are hereby identified as forward-looking statements for this purpose and include, among others, statements relating to: Fibrocell’s expectations regarding the exploration of strategic alternatives; the timing of dosing and reporting of interim data and trial updates for its Phase 1/2 clinical trial of FCX-007; the completion of enrollment in the Phase 2 portion of its Phase 1/2 clinical trial of FCX-007; the potential for FCX-007 to receive Priority Review Vouchers upon market authorization; the potential advantages of Fibrocell’s product candidates; and other statements regarding Fibrocell’s future operations, financial performance and financial position, prospects, strategies, objectives and other future events.

Forward-looking statements are based upon management’s current expectations and assumptions and are subject to a number of risks, uncertainties and other factors that could cause actual results and events to differ materially and adversely from those indicated herein including, among others: the impact of the announcement of the Board of Directors’ review of strategic alternatives, as well as any strategic transaction or alternative that may be pursued, on the Company's business, including its financial and operating results and its employees; that interim clinical trial results are not necessarily indicative of final clinical results and final clinical trial results may not be positive with regard to safety or efficacy of FCX-007; uncertainties and delays relating to the initiation, enrollment and completion of pre-clinical studies and clinical trials; whether pre-clinical study and clinical trial results will validate and support the safety and efficacy of Fibrocell’s product candidates; unanticipated or excess costs relating to the development of Fibrocell’s gene therapy product candidates; Fibrocell’s ability to obtain additional capital to continue to fund operations; Fibrocell’s ability to maintain its collaboration with Precigen, Inc., a wholly owned subsidiary of  Intrexon Corporation; and the risks, uncertainties and other factors discussed under the caption “Item 1A. Risk Factors” in Fibrocell’s most recent Form 10-K filing and Form 10-Q filings. As a result, you are cautioned not to place undue reliance on any forward-looking statements. While Fibrocell may update certain forward-looking statements from time to time, Fibrocell specifically disclaims any obligation to do so, whether as a result of new information, future developments or otherwise.

Investor & Media Relations Contact:
Karen Casey

Kannalife Sciences to be Featured in Dateline NBC Segment Covering Company’s Research on CBD-Like Molecules as Treatments for HE, CTE

NEW YORK, May 18, 2018 (GLOBE NEWSWIRE) -- Kannalife Sciences, Inc. (“Kannalife”), a bio-pharmaceutical and phyto-medical company, is excited to announce that the Company will be featured in a Dateline NBC segment discussing how their research on cannabidiol (CBD)-like molecules could potentially change the landscape for various ailments including chronic traumatic encephalopathy (CTE) and the opioid epidemic.

The Kannalife-centric segment will air at 7:00pm EDT, during the Sunday, May 20th, episode of Dateline NBC. The segment will take an in-depth look into Kannalife Sciences and their groundbreaking research investigating whether the Company’s proprietary CBD-like molecules can help change the medical landscape and offer several new treatments for specific ailments.

“We are excited to be a part of this Dateline NBC special, dedicated to the therapeutic benefits of cannabinoids derived from the cannabis plant, cannabinoid-like molecules and how companies like Kannalife are working to conduct the necessary research to bring new drugs to market,” said Kannalife Sciences CEO Dean Petkanas. “This kind of exposure helps us communicate the health and wellness benefits of CBD and CBD-like molecules, as well as new potential treatments for CTE.”

A good portion of this news piece will focus on the problem of CTE, a degenerative neurological disease caused by repeated head trauma that can lead to symptoms like violent mood swings, depression and other cognitive difficulties. Currently, CTE can only be definitively diagnosed by direct tissue examination after death. Because there is not much known about this debilitating disease, treatment and prevention options are bleak.

Kannalife Sciences currently holds two licenses, one of them exclusive, with the National Institutes of Health (NIH) for the Commercialization of U.S. Patent #6630507, “Cannabinoids as Antioxidants and Neuroprotectants”, to research Hepatic Encephalopathy (HE) and CTE.

Additionally, with over 20,000 deaths a year related to prescription opioids, Kannalife discusses the potential benefits of using cannabis as a replacement for opioids to not only help those suffering from addiction but as an alternative to opioids to treat chronic pain.

About Kannalife Sciences
Kannalife Sciences, Inc. is a bio-pharmaceutical and phyto-medical company involved in the research and development of novel new therapeutic agents designed to reduce oxidative stress, and act as immuno-modulators and neuroprotectants. Kannalife Sciences currently holds an exclusive license with National Institutes of Health – Office of Technology Transfer (“NIH-OTT”) for the Commercialization of U.S. Patent #6630507, “Cannabinoids as Antioxidants and Neuroprotectants”. Kannalife is currently conducting research and development at the Bucks County Pennsylvania Biotechnology Center in Doylestown, PA, for a target drug candidate to treat HE and CTE. HE and CTE are oxidative stress related diseases that affect the cognitive and behavioral functions, and the wellness of the brain.

This press release may contain certain forward-looking statements and information, as defined within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, and is subject to the Safe Harbor created by those sections. This material contains statements about expected future events and/or financial results that are forward-looking in nature and subject to risks and uncertainties. Such forward-looking statements by definition involve risks, uncertainties. The statements in this press release have not been evaluated by the FDA and are not intended to diagnose, treat or cure any disease. The Company does not sell or distribute any products that are in violation of the United States Controlled Substances Act.


Public Relations Contact:
Andrew Hard
Chief Executive Officer
CMW Media
P: 888-829-0070

Adverum Biotechnologies Presents Additional Long-term Preclinical Data on ADVM-022 in Wet AMD at ASGCT 21st Annual Meeting

-- Single Intravitreal Administration of Gene Therapy ADVM-022 Provides
Long-term Protection in Nonhuman Primate Model of Wet AMD --

MENLO PARK, Calif., May 17, 2018 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq:ADVM), a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases, announced the presentation of additional long-term efficacy data from a preclinical study of ADVM-022 in wet age-related macular degeneration (wAMD) in a poster session today at the ASGCT 21st Annual Meeting in Chicago, IL.

“It is exciting to see that a single administration of ADVM-022 has the potential to provide long-term protection against choroidal neovascularization, which is associated with vision loss in wAMD,” said Mehdi Gasmi, Ph.D., chief science and technology officer of Adverum Biotechnologies. “Our poster presentation at this year’s ASGCT Annual Meeting includes the most robust set of data to date on ADVM-022. Based on the results from these studies and our ongoing Investigational New Drug-enabling studies, we are on track to submit an IND Application for ADVM-022 in the second half of 2018. We are eager to get ADVM-022 into the clinic to advance this gene therapy that may offer convenient, long-term protection for patients living with wAMD.”

Preclinical data from the ADVM-022 poster presentation at ASGCT include:

  • After 13 months, a single intravitreal injection of ADVM-022 was found to be safe and statistically significant (p<0.0001) in preventing the development of Grade IV lesions compared to the vehicle control group. The efficacy at 13 months was consistent with earlier-reported data, demonstrating that ADVM-022 induced long-term efficacy that was comparable to aflibercept, an anti-Vascular Endothelial Growth Factor (VEGF) standard-of-care therapy. Additionally, mean CNV complex areas were significantly smaller (p<0.0001) in the ADVM-022 and aflibercept groups, compared with vehicle, when analyzed by spectral domain optical coherence tomography (SD-OCT).
  • ADVM-022 induced sustained intraocular expression of aflibercept for up to 16 months following a single intravitreal injection. Robust levels of aflibercept protein were detected up to 16 months in aqueous and vitreous humor and, more importantly, in retina and choroid tissues, where neovascularization occurs in wAMD.
  • A separate pharmacokinetics study revealed that levels of vector-derived aflibercept measured in the vitreous and the retina 56 days post ADVM-022 injection match the levels of aflibercept recombinant protein 3-4 weeks post bolus of protein injection, which is well within the therapeutic window of the standard of care. In addition, the levels of vector-derived aflibercept at 56 days were consistent with those observed in the long-term efficacy study at 16 months. Taken together, these data suggest that a single injection of ADVM-022 administered intravitreally can generate stable levels of aflibercept found to be within the therapeutic window of the standard-of-care recombinant protein.
  • ADVM-022 was well tolerated, with no serious adverse events.  ADVM-022 had mild effects on aqueous and vitreous cell infiltrates, keratic precipitates, and intraocular pressure (IOP). Treatment-related side effects were limited to mild inflammatory responses and related transient IOP decrease. Longitudinal OCT studies, fundoscopy, and fluorescein angiography did not identify changes in retinal morphology, optic nerve head and vascular integrity.

About ADVM-022 Gene Therapy for wAMD
Adverum’s gene therapy candidate ADVM-022 utilizes a proprietary vector capsid (AAV.7m8) carrying an aflibercept coding sequence under the control of a proprietary expression cassette and is administered as a single intravitreal injection. Vascular endothelial growth factor (VEGF) activity is associated with wAMD progression and vision loss. Anti-VEGF standard-of-care therapies administered every 4-8 weeks have shown the potential to prevent disease progression and preserve or even improve patients’ vision.  Treatment with ADVM-022 is designed to minimize the burden of frequent anti-VEGF injections, the current standard-of-care treatment for wAMD.

About Adverum Biotechnologies, Inc.
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products for ophthalmic diseases and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development. For more information please visit

Adverum’s Forward-Looking Statements
Statements contained in this press release regarding Adverum’s intention to file an IND application for ADVM-022 in the second half of 2018 and potential for further development of ADVM-022 are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties described in Adverum’s periodic reports filed with the Securities and Exchange Commission (SEC), in particular under the caption “Risk Factors” in its Form 10-Q filed with the SEC on May 9, 2018. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

CONTACT: Contact for Adverum: 
Leone Patterson
Interim President and Chief Executive Officer

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