Category Archives: Cell Medicine

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to TRIKAFTA based on in vitro data. SYMDEKO (tezacaftor/ivacaftor and ivacaftor) and KALYDECO (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. These approvals allow more than 600 people with CF not previously eligible for these medicines an opportunity to potentially benefit from treatment that targets the underlying cause of their disease.

The approval for expanded use of three of our CF medicines based on our well-established in vitro model is a testament to the relentless commitment of our scientists to reach our goal of developing treatments for all people with CF, said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. We remain as committed today to reaching every patient who might benefit from our medicines as when we first started out on this journey 20 years ago, and this important milestone now enables hundreds of people with CF access to a treatment option to address the underlying cause of their disease many for the first time.

TRIKAFTA was previously approved for people with at least one F508del mutation and is now approved for 177 additional mutations; SYMDEKO is now approved for 127 additional mutations, for a total of 154 SYMDEKO-responsive mutations; and KALYDECO is now approved for an additional 59 mutations, for a total of 97 KALYDECO-responsive mutations. In addition, for certain people with CF who are currently eligible for KALYDECO, this approval allows them to also be eligible for SYMDEKO or TRIKAFTA; and similarly, for those who are currently eligible for SYMDEKO, this approval allows them to also be eligible for TRIKAFTA.

The full list of mutations for TRIKAFTA, SYMDEKO and KALYDECO can be found within the updated full Prescribing Information for each respective product. In addition, people with CF and their families can search eligibility for Vertex CF medicines through vertextreatments.com.

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO (ivacaftor), SYMDEKO (tezacaftor/ivacaftor and ivacaftor), and TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

What is KALYDECO?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients

age 4 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 4 months of age.

What is SYMDEKO?

SYMDEKO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with SYMDEKO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if SYMDEKO is safe and effective in children under 6 years of age.

What is TRIKAFTA?

TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.

Patients should not take KALYDECO, SYMDEKO, or TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. Johns wort.

Before taking KALYDECO, SYMDEKO, or TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems; have or have had liver problems; are pregnant or plan to become pregnant because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA passes into breast milk. Before taking KALYDECO, patients should tell their doctor if they drink grapefruit juice or eat grapefruit.

KALYDECO, SYMDEKO, or TRIKAFTA may affect the way other medicines work, and other medicines may affect how KALYDECO, SYMDEKO, or TRIKAFTA work. Therefore, the dose of KALYDECO, SYMDEKO, or TRIKAFTA may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

KALYDECO, SYMDEKO, or TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, use machinery, or do anything that needs them to be alert until they know how KALYDECO, SYMDEKO, or TRIKAFTA affects them.

Patients should avoid food or drink containing grapefruit while taking KALYDECO, SYMDEKO or TRIKAFTA.

KALYDECO, SYMDEKO, and TRIKAFTA can cause serious side effects, such as:

High liver enzymes in the blood have been reported in patients receiving KALYDECO, SYMDEKO, or TRIKAFTA. The patient's doctor will do blood tests to check their liver before starting treatment with KALYDECO, SYMDEKO, or TRIKAFTA, every 3 months during the first year of treatment, and every year while on treatment. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.

Abnormality of the eye lens (cataract) in some children and adolescents treated with KALYDECO, SYMDEKO, or TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with KALYDECO, SYMDEKO, or TRIKAFTA to look for cataracts.

The most common side effects of KALYDECO include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

The most common side effects of SYMDEKO include headache, nausea, sinus congestion, and dizziness.

The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.

These are not all the possible side effects of KALYDECO, SYMDEKO, or TRIKAFTA. Please click product link to see the full Prescribing Information for KALYDECO, SYMDEKO or TRIKAFTA.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani in this press release, statements regarding the eligible patient population for TRIKAFTA, SYMDEKO and KALYDECO, our expectations regarding the number of patients newly eligible for TRIKAFTA, SYMDEKO and KALYDECO, and statements regarding the potential benefits of TRIKAFTA, SYMDEKO and KALYDECO. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at http://www.vrtx.com and on the SECs website at http://www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

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Vertex Announces FDA Approvals of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO (tezacaftor/ivacaftor and ivacaftor) and KALYDECO...

REHOVOT, Israel and VANCOUVER, BC, Dec. 10, 2020 /PRNewswire/ -- CollPlant (NASDAQ: CLGN), a regenerative medicine company, and STEMCELL Technologies, Canada's largest privately owned biotechnology company, which develops cell culture media, cell separation systems, instruments, and other reagents for life sciences research, today jointly announced they have entered into aproduct manufacturing and supply agreement. CollPlant will sell its proprietary recombinant human Type I collagen (rhCollagen), the world's first plant-based rhCollagen, to STEMCELL Technologies, which will incorporate CollPlant's product into cell culture media kits.

The recently signed agreement follows the companies' established business relationship, which started in 2014 when STEMCELL began purchasing and incorporating CollPlant's rhCollagen into some of its cell culture expansion and differentiation media kits. To date, hundreds of companies, as well as research and academic institutes, have used these kits for research and development projects. STEMCELL will distribute the kits globally for use in the regenerative medicine research market.

"Incorporation of rhCollagen into STEMCELL's cell culture applications sold to researchers worldwide is designed to help advance the science in a broad range of dynamic fields including stem cells, immunology, cancer, regenerative medicine, and cellular therapy. We are happy to have entered into this agreement with STEMCELL, which, as Canada's largest biotechnology company, is very well positioned to make rhCollagen-containing cell culture kits widely available in the market," stated Yehiel Tal, Chief Executive Officer of CollPlant. "The cell culture market is just one example of the vast potential of our rhCollagen platform technology in life science applications. We continuously evaluate new fields in which CollPlant's products and technologies have the potential to enable breakthroughs that improve patients' lives."

Dr. Sharon Louis, STEMCELL's Senior Vice President of Research and Development noted that "STEMCELL is pleased to utilize CollPlant's animal component free rhCollagen to promote cell attachment in several products that support the culture of diverse human progenitor cell types. The quality and animal component-free composition of CollPlant's rhCollagen is what first brought this product to STEMCELL's attention, and the robust performance rhCollagen provides with a variety of STEMCELL media is what we want to be able to provide to our customers. Upon entering into this agreement, STEMCELL and CollPlant will together provide high-quality reagents that will be used to further our understanding in life sciences and potentiate regenerative medicine research."

About STEMCELL Technologies

STEMCELL Technologies is Canada's largest biotechnology company. Based in Vancouver, STEMCELL supports life sciences research around the world with more than 2,500 specialized reagents, tools, and services. STEMCELL offers high-quality cell culture media, cell separation technologies, instruments, accessory products, and educational resources that are used by scientists advancing the stem cell, immunology, cancer, regenerative medicine, microbiology, and cellular therapy fields.

Find more information at http://www.stemcell.com

About CollPlant Biotechnologies

CollPlant is a regenerative and aesthetic medicine company focused on 3D bioprinting of tissues and organs, and medical aesthetics. Our products are based on our rhCollagen (recombinant human collagen) that is produced with CollPlant's proprietary plant based genetic engineering technology.

Our products address indications for the diverse fields of tissue repair, aesthetics and organ manufacturing, and, we believe, are ushering in a new era in regenerative and aesthetic medicine.

Our flagship rhCollagen BioInk product line is ideal for 3D bioprinting of tissues and organs. In October 2018, we entered into a licensing agreement with United Therapeutics, whereby United Therapeutics is using CollPlant's BioInks in the manufacture of 3D bioprinted lungs for transplant in humans.Recently, the parties announced the expansion of the collaboration with the exercise by United Therapeutics of its option to cover a second lifesaving organ, human kidneys.

Safe Harbor for Forward-Looking Statements

This press release may include forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to CollPlant's objectives, plans and strategies, as well as statements, other than historical facts, that address activities, events or developments that CollPlant intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as "believes," "hopes," "may," "anticipates," "should," "intends," "plans," "will," "expects," "estimates," "projects," "positioned," "strategy" and similar expressions and are based on assumptions and assessments made in light of management's experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Many factors could cause CollPlant's actual activities or results to differ materially from the activities and results anticipated in forward-looking statements, including, but not limited to, the following: the CollPlant's history of significant losses and its need to raise additional capital and its inability to obtain additional capital on acceptable terms, or at all; CollPlant's expectations regarding the timing and cost of commencing clinical trials; regulatory action with respect to rhCollagen-based products, including but not limited to acceptance of an application for marketing authorization, review and approval of such application, and, if approved, the scope of the approved indication and labeling; commercial success and market acceptance of the CollPlant's rhCollagen-based BioInk; CollPlant's ability to establish sales and marketing capabilities or enter into agreements with third parties and its reliance on third-party distributors and resellers; CollPlant's reliance on third parties to conduct some aspects of its product manufacturing; the scope of protection CollPlant is able to establish and maintain for intellectual property rights and the company's ability to operate its business without infringing the intellectual property rights of others; the overall global economic environment; the impact of competition and new technologies; general market, political, and economic conditions in the countries in which the company operates; projected capital expenditures and liquidity; changes in the company's strategy; and litigation and regulatory proceedings. More detailed information about the risks and uncertainties affecting CollPlant is contained under the heading "Risk Factors" included in CollPlant's most recent annual report on Form 20-F, filed with the SEC, and in other filings that CollPlant has made. The forward-looking statements contained in this press release are made as of the date of this press release and reflect CollPlant's current views with respect to future events, and CollPlant does not undertake, and specifically disclaims, any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact atCollPlant:

Eran RotemDeputy CEO & CFOTel: + 972-73-2325600[emailprotected]

Contact at STEMCELL: Luba Metlitskaia Vice President, Business Development & Licensing [emailprotected]

SOURCE CollPlant

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CollPlant to Supply rhCollagen to STEMCELL Technologies for Use in a Broad Range of Cell Culture Applications - PRNewswire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Faze Medicines, a biotechnology company pioneering therapeutics based on the groundbreaking new science of biomolecular condensates, today announced its launch and Series A financing of $81 million. Faze is founded by leading experts in the emerging field of biomolecular condensates with the mission of leveraging this fundamentally new understanding of cell biology to develop therapies to slow, halt or reverse disease pathology. The Series A was led by Third Rock Ventures with Novartis Venture Fund, Eli Lilly and Company, AbbVie Ventures, Invus, Catalio Capital Management, Casdin Capital and Alexandria Venture Investments participating.

Biomolecular condensates are membrane-less clusters of molecules, such as proteins and nucleic acids, that dynamically organize to perform a wide array of cell functions. Research over the past decade, including seminal work by Fazes founders, has found that disturbances in the behavior of condensates play a causative role in myriad human diseases, including amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. Faze is now poised to deliver medical breakthroughs based on this fundamentally new understanding of cell biology.

The biology of condensates is the kind of science that will rewrite textbooks and, we believe, rewrite medicine, said Cary Pfeffer, M.D., interim chief executive officer of Faze and partner at Third Rock Ventures. Faze is founded by leading experts who have been integral to this field since its very beginnings. Their insights, coupled with the deep expertise of the team we have assembled, will enable us to realize the enormous potential of this new biology.

Cell biology is undergoing a transformation as we come to understand the integral role that biomolecular condensates play within cell processes from DNA repair to intracellular transport, added Rachel Meyers, Ph.D., chief scientific officer of Faze. Faze was founded to translate these exciting discoveries out of the lab and into the clinic, where they could make a real difference in treating diseases that have seen very little therapeutic progress.

The Series A will support Fazes preclinical research in two initial therapeutic focus areas ALS and myotonic dystrophy type 1 (DM1) as well as research to explore condensate biology in other disease areas. In ALS and DM1, a robust body of literature points to a causative role for condensate dysregulation. Leveraging state-of-the-art screening and proteomics techniques, Faze will identify proteins that are key components or regulators of disease-causing condensates, and then employ proprietary assays to discover small molecule drugs targeting these proteins.

Founders and Leadership

Faze is founded by renowned scientific leaders in the field of biomolecular condensates:

Fazes leadership team brings together accomplished biotechnology executives with decades of industry experience and deep scientific, drug discovery and drug development knowledge:

Joining Dr. Pfeffer on the companys inaugural board of directors is:

Faze has additionally established a robust group of expert advisors including those in the areas of drug discovery and clinical development.

About Faze Medicines

Faze Medicines is a biotechnology company harnessing the groundbreaking new science of biomolecular condensates to create medical breakthroughs. Faze was founded by renowned scientific leaders in the field of biomolecular condensates and is supported by a world-class syndicate of investors including Third Rock Ventures, Novartis Venture Fund, Eli Lilly and Company, AbbVie Ventures, Invus, Catalio Capital Management, Casdin Capital and Alexandria Venture Investments. For more information, visit fazemed.com.

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Faze Medicines Launches With $81 Million Series A Financing to Leverage New Biology of Biomolecular Condensates to Treat Disease - Business Wire

EDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease

CAMBRIDGE, Mass., Dec. 09, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced it submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase 1/2 clinical trial of EDIT-301, an experimental CRISPR/Cas12a gene editing medicine in development for the treatment of sickle cell disease. The Company previously received Rare Pediatric Disease designation from the FDA for EDIT-301.

This IND submission is a key milestone for Editas as we continue to advance several ex vivo cell therapy medicines. This submission brings us one step closer to entering the clinic with our potentially best-in-class, transformative, and durable medicine for people living with sickle cell disease, said Cynthia Collins, Chief Executive Officer, Editas Medicine. This moment is very exciting for the Editas team. We know patients are counting on us, and we look forward to next steps for the clinical development of EDIT-301, including dosing sickle cell disease patients.

Editas Medicine continues to prepare for a Phase 1/2 clinical trial evaluating EDIT-301 for the treatment of sickle cell disease. The Company has identified a lead principal investigator and engaged a Clinical Research Organization (CRO). Clinical trial materials are being manufactured by Editas Medicine.

About Sickle Cell DiseaseSickle cell disease is caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS). Fetal hemoglobin (HbF) protects against sickle cell disease by inhibiting HbS polymerization. Individuals with high levels of HbF are protected from sickle cell disease. EDIT-301 is an experimental, autologous cell therapy comprising CD34+ cells genetically modified using a Cas12a ribonucleoprotein (RNP) that targets the HBG1/2 promoter in the beta-globin gene to stimulate HbF production.

About EDIT-301EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.

AboutEditas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companys plans and expectations for EDIT-301. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

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Editas Medicine Announces Submission of IND Application for EDIT-301 with the FDA - GlobeNewswire

Israeli biotech Pluristem is canning its experimental phase 3 critical limb ischemia therapy after an outside review said it was no good.

Haifa, Israel-based Pluristems R&D operation is built upon placenta-derived adherent stromal cells, which the biotech has designed for use in patients of all human leukocyte antigen types. This approach is made possible by the low immunogenicity of the cells. Once inside the body, Pluristem hopes the cells will drive the healing of injured tissue.

But one of its leading contenders using this approach has been judged a failure in phase 3: An independent data monitoring committee (DMC) took a look at the ongoing data for its pivotal phase 3 in patients with critical limb ischemia (CLI), a severe obstruction of the arteries which markedly reduces blood flow to the extremities and can lead to amputation.

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The DMC said the test was unlikely to meet the primary endpoint, and that the CLI study population has experienced a substantial low number of events (major amputation of the index leg or death), different from what is known in clinical medicine for the rate of these events in this patient population. The lower than anticipated event rate in the placebo group reduced the statistical power of the study to meet its primary endpoint.

The biotech is now tossing out the therapy and will instead focus on other pipeline areas, including a long-shot stem cell attempt at treating COVID-19. The biotechs shares fell nearly 40% on the news.

We are deeply disappointed by the outcome of the CLI interim analysis. In light of the DMCs recommendation, we decided that it would be in the best interests of the company and its shareholders to terminate the CLI study and focus our resources and efforts on our other lead indications, said Pluristem CEO and President Yaky Yanay.

We expect to present topline clinical results during calendar year 2021, including our phase 3 study in muscle regeneration following hip fracture, phase 2 studies in Acute Respiratory Distress Syndrome associated with COVID-19 and our phase 1 study in incomplete hematopoietic recovery following hematopoietic cell transplantation. Pluristem is well positioned to advance and support future development of these indications.

Last year, Pluristem penned a deal with NASA to assess its cell therapies against the health problems caused by spending time in space, teaming up with NASAs Ames Research Center for the project, which focuses on using its PLX placenta-derived cell therapies to try to prevent or treat medical conditions that can occur during and after space missions, including conditions that affect the blood, bone, muscle, brain and heart.

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NASA-partnered Pluristem crashes to Earth as it axes leading therapy - FierceBiotech

It takes a lot of poppies to make serious painkillers. Morphine is classed as an essential medicine by the World Health Organization, but it wouldnt exist withoutPapaver somniferum(better known as the opium poppy).Around 100,000 hectares of the flowers are cultivated every year primarily in Australia, Spain, France, India, Turkey and Hungary to meet demand for opioid medication worldwide. Approximately 800 tons of the natural opiates morphine and thebaine are extracted from the harvested straw, some of which are then chemically converted into higher value drugs, such as codeine, oxycodone and hydrocodone.

Although fields of beautiful blooms sound idyllic, industrial poppy farming isnt easy.

Pests, disease and poor weather conditions can dramatically impact the yield. Plus, growing drugs from plants takes a long time and the poppies themselves contain very low levels of the active ingredient. If there is a spike in demand for opioid medicines, the supply chain can struggle to respond fast enough. These issues mean problems for the pharmaceutical industry, which in-turn means drug shortages for doctors and patients.

Medicines derived from nature (as around 40% of our drugs are) are particularly vulnerable in times of uncertainty. During the Covid-19 crisis, weve seen that supply chain problems can have a human cost. In the spring, the US Food and Drug Administration reported a shortage of the opioids needed to safely keep patients on ventilators.

There could be a better way of making the rare compounds found in nature and avoiding similar supply chain issues, says Stanford University adjunct professor Christina Smolke. In 2015, she and a small team of researchers proved that opioids could be produced using genetically-modified yeast.

Smolkes breakthrough involved a process weve known about for centuries: fermentation. Give brewers yeast a little sugar and under the right conditions itll reward you with alcohol and carbon dioxide.

Smolke figured fermentation had the power to speed up drug manufacturing with yields seen in day or weeks, rather than months or years as with crop farming. It would mean supply could be easily increased if there was an unexpected event.

But plant-derived medicines are made from more complex stuff than ethanol or carbon dioxide. If you look at the types of molecules plants make, theyre incredibly complicated from a structural perspective, says Smolke. The way they are able to survive and interact with their environment is through chemistry.

These molecules are almost impossible to replicate in the laboratory using synthetic chemistryso Smolke, who has a chemical engineering background, instead turned to synthetic biology. First, she identifiedwhich plant enzymes are responsible for the chemical reactions that lead to the useful compounds.

Then its a case of making the genetic recipe for these chemicals and translating it into a language the yeast can understand. Synthetic DNA is then inserted into the yeast, which tells it to make the desired compounds out of the sugar and amino acids the scientists feed to it.

To coax the yeast to make the opioid hydrocodone, the team translated 23 proteins from the poppy into the microbe. After her initial breakthrough, Smolke formed start-up synthetic biology company Antheia to focus on creasing ever-more complex medicines using yeast.

Its not the first time a microbe has been used to make medicine. In 1982, Eli Lily introduced synthetic insulin Humulin to the market, a breakthrough drug for people with diabetes. Before, insulin had to be extracted from the pancreases of cows or pigs. This caused allergic reactions in some patients as animal insulin isnt an exact match for the hormone produced in healthy human pancreases.

Humulin is made by introducing human DNA into a bacterial host cell. As the bacteria multiply, the medicine is produced. Human insulin can now also be made using yeast cells and fermentation. And since 2014, Sanofi has also used yeast to produce artemisinic acid to make the anti-malarial artemisinin, which is usually sourced from the sweet wormwood plant.

In August 2020, Smolke and colleagues announced inNaturethat they had successfully produced neuromuscular blockers used in Parkinsons disease and intestinal disorders. These chemicals were the tropane alkaloids hyoscyamine and scopolamine, which are naturally found in the nightshade family of plants.

As with the poppy, cultivating nightshades is subject to global supply risks, such as environmental disasters and the ongoing pandemic. Shortages of alkaloid-based medicines are unfortunately common.

To achieve tropane alkaloid synthesis in yeast, Smolke and her team had to overcome several challenges. As before, they first needed to work out which enzymes the nightshade plant uses to make the compounds and how to get those to work in a yeast host.

But the biggest hurdle was realising the critical chemical reactions required to make tropane alkaloids are segregated across different subcellular compartments, cells or tissues in the plant. It would be tough to recapitulate this in single-cell yeast.

In many cases, its not enough just to get the protein made because it wont have the correct kind of biochemical environment for it to actually perform its reaction, says Smolke. A lot of times where these types of projects fail you can identify the enzyme, but you cant get the yeast to make it in a way that the reaction actually happens.

After a gruelling coding process involving 26 genes from ten different organisms, they were able to create six different subcellular compartments within the yeast cell to make sure the right chemical reaction would occur at the right time.

What we also did beyond distributing the enzymes was incorporating other proteins like transporters that could be localised to the membranes of these compartments, Smolke explains.

This allowed for more efficient routing of the metabolites from one set of chemistries to the next. In doing so, the team transformed the yeast into a chemical factory, in which different synthesis steps are conducted in separate reactors to ensure each reaction has the optimum conditions.

TheNaturepaper is further proof that brewers yeast can be a platform for making some of natures most valuable and complex molecules. The next step for Smolke will be convincing the pharmaceutical industry that this could be the future of drug manufacturing. She and her team will need to prove the technology can mass-produce molecules faster, cheaper and more reliably than the original farming methods.

In the latest work, only a few milligrams of tropane alkaloids per litre of yeast culture were produced not yet a competitive alternative to production through plant extraction. However, Smolke says Antheia is constantly tinkering with the yeast to improve its efficiency so a larger amount of compound over a given period time is produced.

As these examples scale, well see there are new ways of structuring supply chains that will bring more resiliency and control, Smolke says. This is going to open up the pharmaceutical industry.

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Brewprint: using yeast to make plant-based drugs - pharmaceutical-technology.com

Event will review the companys novel approach to inducing fetal hemoglobin

Live webcast on December 15, 2020 at 8:30am ET

CAMBRIDGE, Mass., Dec. 09, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.(Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it will host a Key Opinion Leader (KOL) meeting on Tuesday, December 15, 2020 from 8:30am 10:00am ET to discuss the companys program with FTX-6058 for select hemoglobinopathies, including sickle cell disease and beta-thalassemia.

Dr. Maureen Achebe and Dr. Gerd Blobel will join senior executives from Fulcrum in presenting and discussing sickle cell disease, the treatment landscape and the FTX-6058 program followed by a Question and Answer session. Maureen Achebe, MD is currently Clinical Director, Non-Malignant Hematology Clinic, Assistant Director, Brigham and Womens Hospital Outpatient Infusion Center, Director, Brigham and Womens Hospital Sickle Cell Program and Assistant Professor of Medicine, Harvard Medical School. Gerd Blobel, MD, PhD is currently Frank E. Weise III professor of pediatrics, University of Pennsylvania and Co-director Epigenetics Institute. He also holds the Frank E. Weise III Endowed Chair of Pediatrics at The Childrens Hospital of Philadelphia and the Perelman School of Medicine.

The live webcast will be accessible through the Investor Relations section of the companys website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available on the website for up to 90 days.

About FTX-6058FTX-6058 is a highly potent small molecule inhibitor of EED capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrums proprietary Product Engine. The companys composition of matter patent covering FTX-6058 and related structures has been granted. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy volunteers.

About Fulcrum TherapeuticsFulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrums proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia, into Phase 1 clinical development.

Please visit http://www.fulcrumtx.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Companys product candidates and the potential advantages and therapeutic potential of the Companys product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Companys strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrums ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Companys product candidates; replicate in later clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the Risk Factors section, as well as discussions of potential risks, uncertainties and other important factors, in the Companys most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof and should not be relied upon as representing the Companys views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

Contact:

Investors:Christi WaarichDirector, Investor Relations andCorporate Communications 617-651-8664cwaarich@fulcrumtx.com

Stephanie Ascher Stern Investor Relations, Inc.stephanie.ascher@sternir.com212-362-1200

Media: Kaitlin GallagherBerry & Company Public Relationskgallagher@berrypr.com212-253-8881

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Fulcrum Therapeutics to Host Virtual Key Opinion Leader Event Featuring FTX-6058 for Sickle Cell Disease - GlobeNewswire

JERUSALEM, Dec. 8, 2020 /PRNewswire/ --NeuroGenesis, a clinical-stage biopharmaceutical company advancing innovative cell therapies to combat myelin-related neurodegenerative diseases, and Hadassah Medical Center announced today highly positive results from a placebo-controlled Phase 2 clinical trial, headed by Prof. Dimitrios Karussis, together with Dr. Petrou Panayiota and Dr. Ibrahim Kassis from Hadassah Medical Center in Jerusalem, assessing the impact of NG-01 autologous proprietary subpopulation of mesenchymal stem cells (MSCs) on patients with progressive multiple sclerosis (MS).

The results, recently published in Brain, a prestigious peer-reviewed journal published by Oxford University, and highlighted in the "Editor's Choice", show that:

"The treatment was well tolerated and the trial met all of its primary endpoints," said Professor Dimitrios Karussis, lead principle investigator and Director of MS Center at Hadassah Medical Center, Jerusalem. "The patients' improvement was in many cases quite remarkable and included regain of motor function and noticeable effects on their cognitive abilities."

Prof Karussis added, "Although we currently have several good treatment options for relapsing remitting MS, we fall short in providing effective treatment for progressive MS that could substantially suppress the progression of disability. This trial provides encouraging results and suggests a potential for a new approach that may not only slow down the progression of the disease but even induce improvement and promote repair mechanisms in progressive MS."

The technology is now further developed by NeuroGenesis, following a license from Hadasit, Hadassah Medical Center Technology Transfer Company.

Neurogenesis' technology entails collecting bone marrow from the patient. Then by utilizing a proprietary process, a unique subpopulation of bone marrow cells is identified, cultured and enhanced towards remyelinating biofactory cells (NG-01) that also possess neurotrophic immunolatory and neuroprotective properties. The NG-01 cell population is injected directly into the central nervoussystem (through the cerebrospinal fluid), where the cells home-in on the damaged area, take up residence and produce significant amounts of neurotrophic factors.

"Progressive MS is a chronic, debilitating disease with no satisfactory treatment to improve or reverse established disability," said Tal Gilat, CEO of NeuroGenesis. "We are therefore extremely pleased to witness the significant positive effect of our NG-01 cells. Following recent interactions with the FDA, we look forward to confirming and expanding these findings in a large multi-center MS trial, and continuing advanced studies in additional indications such as ALS."

About the Phase 2 trial of NG-01

The Phase 2, randomized, double-blind, placebo-controlled, clinical trial assessed the safety, tolerability and efficacy of transplantation of NG-01 in people with progressive MS. The study enrolled 48 participants with progressive MS which were randomized into 3 groups, receiving either an intrathecal or intravenous NG-01 injection, or a placebo injection.

The two predetermined primary endpoints of the trial were: (i) the safety of the intrathecal and intravenous NG-01 treatments assessed by incidence of adverse events versus those in the placebo-treated group; and (ii) the differences among the three groups in the Expanded Disability StatusScale(EDSS) score changes and the proportion of patients with treatment failure, as evidenced by an increase in EDSS (disease progression) score, at 6 and 12 months. Overall, the study duration was 14 months.

About Multiple Sclerosis

Multiple sclerosis (MS) is an autoimmune disease that causes damage in the myelin and the nerve cells of the central nervous system (demyelinating plaques in brain and spinal cord), resulting in cumulating neurological disability. The destruction of the myelin (the covering that protects nerves and promotes the efficient transmission of nerve impulses) causes secondary damage to the nerve cells and progressive atrophy. MS often causes sensory disturbances in the limbs, including a prickling or tingling sensation (paresthesia), numbness, pain, and itching. Motor problems are common in people with MS. Affected individuals may have tremors, muscle stiffness (spasticity), exaggerated reflexes (hyperreflexia), weakness or paralysis of the muscles of the limbs, difficulty in walking, and poor sphincter control. The condition is also associated with visual problems, such as blurred or double vision or partial or complete vision loss. There is no known cure for multiple sclerosis.The existing treatments are mostly aimed to reduce the incidence of relapses of the disease and slow down the rate of neurological deterioration.

About NeuroGenesis

Neurogenesis is developing cell therapy for neurodegenerative diseases based on a unique approach for sustained delivery of high levels of remyelinating growth factors using the patient's own stem cells. The technology for this unique approach was licensed from Hadasit, theTechnology TransferCompany of Hadassah Medical Organization in Jerusalem, Israel. The Company's lead product is NG-01 for the treatment of progressive Multiple Sclerosis, (in which a placebo-controlled Phase 2 study has been completed and recently published). NG-01 were also tested in two successful Phase 2a trials in ALS patients. Up to today, more than 150 progressive MS and ALS patients from around the world have been treated with Neurogenesis'products via clinical trials (Phase 1 and Phase 2) and compassionate use treatments.

About Hadassah and Hadasit

For more than a century, Hadassah has set the standard of excellence for medical care and research in Israel. Our doctors and scientists are on the frontlines, uniquely positioned to pinpoint ever-evolving medical needs. Their experience and ingenuity have yielded new ideas with huge potential in all areas of medicine, including therapeutics, diagnostic medical devices, and digital health. Hadasit is the technology transfer company of Hadassah Medical Center in Jerusalem. We transform the cutting-edge research coming out of Hadassah into marketable medical technologies. We turn ground-breaking ideas into viable products and services that can change the world and better humanity.

NeuroGenesiscontact:Tsipi HaitovskyGlobal Media LiaisonNeuroGenesis+972-52-5989-892[emailprotected]

Hadassah contact:Hadar ElboimspokeswomanHadassah Medical Organization+ 972- 2-6776079[emailprotected]

SOURCE NeuroGenesis

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Hadassah Medical Center and Neurogenesis Announce Groundbreaking Results from a Phase 2 Study in Progressive Multiple Sclerosis treated with NG-01...

Further understanding is needed of the redox change called reductive stress and its impact on the onset and progression of neurodegeneration.

Further understanding is needed of the redox change called reductive stress and its impact on the onset and progression of neurodegeneration.Cells require a balance among oxidation-reduction reactions, or redox homeostasis. Loss of that balance to create oxidative stress is often associated with neurodegeneration. Less is known about how loss of that balance at the other end of the spectrum reductive stress, or RS may affect neurons.

Now Rajasekaran Namakkal-Soorappan, Ph.D., associate professor in the University of Alabama at Birmingham Department of Pathology, Division of Molecular and Cellular Pathology, and colleagues in the United States and India have shown for the first time that reductive stress promotes protein aggregation in neuroblastoma cells and impairs neurogenesis.

Our data suggest that, despite the association of oxidative stress and neuronal damage, RS can play a crucial role in promoting proteotoxicity, and thereby lead to neurodegeneration, Namakkal-Soorappan said. Moreover, this study adds to the emerging view that the regulation of redox homeostasis, and its impact on diverse diseases, is part of a complex process in which appropriate doses of antioxidants are required only in response to an oxidative or toxic challenge in cells or organisms.

Namakkal-Soorappan and colleagues have previously found that RS is pathogenic in a mouse-model of heart disease, and that RS impairs the regeneration of skeletal muscle in cultured mouse myoblast cells.

In the current study, the researchers used sulforaphane to establish RS in proliferating and differentiating Neuro 2a neuroblastoma cells grown in culture. Sulforaphane activates Nrf2/ARE signaling, leading to antioxidant augmentation. Specifically, they found that sulforaphane-mediated Nrf2 activation diminished reactive oxygen species in a dose-dependent manner leading to RS. The resulting RS abrogated oxidant signaling and impaired endoplasmic reticulum function, which promoted protein aggregation and proteotoxicity, and impaired neurogenesis. This included elevated Tau and -synuclein and their co-localization with other protein aggregates in the cells.

Namakkal-Soorappan says they were also surprised to see that acute RS impaired neurogenesis, as measured by reduced neurite outgrowth and length, and that maintaining the cells in sustained RS conditions for five consecutive generations dramatically reduced differentiation and prevented the formation of axons.

This impairment of neurogenesis occurs through activation of the pathogenic GSK3/Tau cascade to promote phosphorylation of Tau and create proteotoxicity.

Intriguingly, there have been reports of increased levels of enzymes that can promote RS, both in the brains of Alzheimers patients and in the post-mortem brains of Alzheimers and Parkinsons patients. Also, attempts to promote neurogenesis in neurodegenerative diseases using small molecule antioxidants have had poor outcomes.

Rajasekaran Namakkal-Soorappan, Ph.D.Therefore, clinical evidence warrants a closer investigation and further understanding of redox changes and their impact at the onset and progression of neurodegeneration, Namakkal-Soorappan said.

Neurodegenerative diseases, including Alzheimers, Parkinsons and Huntingtons, are a major health problem in aging populations throughout the world.

Co-authors with Namakkal-Soorappan in the study, Reductive stress promotes protein aggregation and impairs neurogenesis, published in the journal Redox Biology, are Kishore Kumar S. Narasimhan, UAB Department of Pathology; Asokan Devarajan, David Geffen School of Medicine, University of California, Los Angeles; Goutam Karan, University of Utah; Sandhya Sundaram, Sri Ramachandra Medical University & Research Institute, Chennai, India; Qin Wang and Thomas van Groen, UAB Department of Cell, Developmental and Integrative Biology; and Federica del Monte, Medical University of South Carolina, Charleston.

Support came from National Institutes of Health grants 2HL118067, HL118067, 2HL118067-7S and AG042860; American Heart Association grant BGIA 0865015F; and grants from the University of Utah and UAB.

In the three studies on RS published in 2020, Namakkal-Soorappans name is listed as Namakkal S. Rajasekaran.

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Reductive stress in neuroblastoma cells aggregates protein and impairs neurogenesis - The Mix

As the precision medicine field evolves and the science behind personalized therapies for complex conditions surges ahead, reimbursement models are racing to catch up. Precision medicine treatments, like cell and gene therapies, tend to have high price tags and novel delivery mechanisms. This makes creating effective payment models for these therapies a challenge, but drug developers and payers are working together to create out-of-the-box solutions.

Determining prices for breakthrough cell and gene therapies is a complicated process, said Laura Okpala, director of reimbursement policy at Gilead Sciences, at the MedCity INVEST Precision Medicine conference. Though there is a strong belief that the pricing process needs to be driven by value value means different things to different people. Biopharmaceutical companies, like Gilead Sciences, must consult with various stakeholders, including patients, caregivers and payers, who all have different perspectives on value.

Part of why the pricing is so difficult is because of the inherent complexities in the healthcare system, Okpala said. When we think of traditionally how drugs are paid for, were thinking about chronic treatment, were thinking about treatment over a long, extended period, treatment over and over again, reimbursement every single time, and that adds up.

But when you think about cell and gene therapies, all those costs and all of that treatment happens upfront, she added. And then you get that durable response, up to four years at this point. And that is really a paradigm shift when you think about [a] healthcare system that really isnt set up to deal with that upfront cost and that value delivered over time.

But the upfront payment is just one of many challenges. Mark Trusheim, strategic director of the NEWDIGS initiative at the MIT Center for Biomedical Innovation, said at the virtual conference that there are two more key challenges that arise: the performance uncertainty regarding these therapies, particularly around their durability, and the actuarial uncertainty it causes for payers. Most of these therapies are for rare conditions, so a single high-cost therapy in any given month can have a negative impact on payers income statements.

To combat these challenges, several innovative reimbursement models have been developed.

One is a model based on treatment milestones. Per this model, a certain amount of money is paid upfront, and if the therapy doesnt show the intended effects in certain predetermined timeframes, the drug developer pays back a portion of the initial payment.

[The model allows] some risk sharing between the developer and the payer, so they dont have to argue quite so much up front, Trusheim said. And the actual product performance [resolves] how much [is] finally the net reimbursement or the net price for that therapy.

This model helps manage the different expectations and fears of both parties, he added.

Another is a subscription-based model, which includes a fixed fee for unlimited access to certain therapies, Trusheim explained. Cigna has an insurance product that offers this reimbursement model, where plan members contribute a certain amount each month that is used to pay for therapies as needed. Cigna takes on the risk, guaranteeing that they will provide as much therapy as the members require.

This model is a great example of how payers can manage the actuarial fluctuation that occurs when funding cell and gene therapies, Trusheim said. But it comes with its challenges, because in some cases, its difficult to ascertain the eligible population for a particular therapy especially if there are alternate therapies already available.

But Trusheim is confident that innovation in reimbursement will catch up to clinical innovation in the precision medicine arena.

Were now in an era where innovation in payment structures and approaches are beginning to match the kind of innovation we have in the transformative science for patients, he said. Successfully providing patient access and benefit requires both kinds of innovation, not just scientific innovation. The creativity is there we are going to succeed. Just as the science has succeeded, the payment innovation is also moving forward and having success.

Photo credit: Devrimb, Getty Images

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Innovative payment models to support cell and gene therapies on the rise - MedCity News