Category Archives: Genetic Therapy

Vir and Alnylam Go Ahead with COVID-19 Gene Silencing Therapy

Vir Biotechnology (VIR) announced a new COVID-19 drug candidate, VIR-2703. The company is collaborating with Alnylam Pharmaceuticals (ALNY) for developing this treatment which is an inhalable RNAi therapeutic which works by targeting the SARS-CoV-2 genome for potentially treating and/or preventing the respiratory illness. Both companies are now planning to meet with the FDA for a probable accelerate pathway for progressing to clinical trials stage.

The collaboration also seeks to identify up to three additional drug candidates for treating COVID-19 as well as other coronavirus ailments. Vir recently received infusion of $250 million from GlaxoSmithKline Plc (NYSE:GSK). In March this year, Alnylam synthesized 350 siRNAs targeting the SARS-CoV-2 genome. Alnylam Pharmaceuticals CEO John Maraganore said, "To our knowledge, this is one of the most potent direct-acting antivirals targeting SARS-CoV-2 reported to date. As this pandemic continues to unfold, we are committed to acting with the utmost urgency to broaden and accelerate our efforts to develop investigational RNAi therapeutics against COVID-19, and potentially future coronavirus-mediated diseases." The companies are working closely to generate data for facilitating fast commercialization.

VIR-2703 takes a novel approach for treating coronavirus. It employs RNA interference or RNAi technology for silencing particular genetic material leading to the impact on production of proteins responsible for the disease. Alnylam currently has Givlaari and Onpattro, the FDA approved drugs in its portfolio and these drugs are intended to treat rare genetic diseases. The companies plan to file an Investigational New Drug (IND) or IND equivalent application for the drug candidate by the end of this year. The investigational RNAi therapeutic is being developed as an inhalation formula and would be delivered directly to the patient's lungs.

Alnylam is believed to have synthesized more than 350 small interfering RNAs or siRNAs as the company said that it is focusing on the highly conserved regions of the SARS-CoV-2 genome. VIR-2703 showed to have an effective concentration for 50% inhibition (EC50) of less than 100 picomolar in dose-response assays. It also demonstrated an EC95 of less than one nanomolar in the SARS-CoV-2 live virus model which was used for measuring the inhibition of infectious virion production.

VIR-2703 is also known as ALN-COV and showed 95 percent of its maximal effect at a concentration of one nanomolar in its dose response assays. The drug candidate has been called "one of the most potent direct-acting antivirals targeting SARS-CoV-2 reported to date" by Alnylam CEO John Maraganore. The companies are planning to develop up to three more host factor targeting development candidates for treating COVID-19.

Vir mainly focuses on developing treatments for infectious diseases while Alnylam specializes in developing RNAi treatments. Both the companies are also working on another product candidate VIR-2218, which seeks to treat hepatitis B virus. This drug candidate is an RNAi product and works by inhibiting the production of proteins such as hepatitis B virus surface antigen (HBsAg). VIR-2218 is currently in Phase 2 of development.

BioMarin Pharmaceutical Inc. (BMRN) announced that it has collaborated with a Swiss biotech firm DiNAQOR AG. The deal is mainly concerned with the development of novel gene therapies for treating rare genetic cardiomyopathies. The license initially encompasses DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy. However, later the collaboration will expand to cover various pipeline programs of the Swiss company.

The companies did not disclose the financial terms of the deal but DiNAQOR will be entitled to an undisclosed sum as upfront payment and may also be eligible for biobucks. BioMarin will have the option to expand the license to cover additional programs of DiNAQOR on similar terms. Jean-Jacques Bienaim, chairman and CEO at BioMarin said, " With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology. This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies." BioMarin has its own portfolio of gene therapy work.

It is estimated that nearly half a million people around the world have been diagnosed with HCM. Under the terms and conditions of the deal, DiNAQOR will also receive various other payments tied to the achievement of different development regulatory and commercial milestones in addition to tiered royalties. BioMarin expects the collaboration to help boost its position in the market and provide it global reach.

BioMarin also reiterated its 2020 GAAP net income estimates. The company expects its GAAP net income for the entire year to be in the range of $20 million and $80 million, taking the collaboration into account. DiNAQOR mainly focuses on developing gene therapies which can safely offer treatment to the heart muscle and carry out transduction of the cardiac cells while limiting the exposure of the therapy to other organs.

Teva Pharmaceutical Industries (TEVA) announced the commercial availability of its Truxima in the United States. The company has collaborated with Celltrion Healthcare for this purpose. Truxima is the biosimilar to Roche (OTCQX:RHHBY) blockbuster Rituxan for treating rheumatoid arthritis.

The drug is approved to be used for treating rheumatoid arthritis in combination with methotrexate in adult patients suffering from moderately to severely active RA and who have not shown adequate response to one or more TNF antagonist therapies. Brendan O'Grady, Executive Vice President, North America Commercial of Teva said, "We are proud to make TRUXIMA available to patients and providers as a treatment option for these indications, especially as this is the only rituximab biosimilar indicated for rheumatoid arthritis. Following the launch of our other biosimilar earlier this year, we remain focused on our commitment to lower healthcare costs and increase price competition through the availability of biosimilars." Truxima was awarded a pass-through status in the hospital outpatient setting earlier this year. The status was granted by the Centers for Medicare and Medicaid Services.

Under its October 2016 agreement, Teva owns commercialization rights for the drug in the United States and Canada. Celltrion vice chairman Hyoung-Ki Kim said, "We believe that the continued use of biosimilars in the US market will contribute to addressing unmet needs for patients and providers."

It is expected that the drug will be available through primary wholesalers at a Wholesale Acquisition Cost of $845.55 per 100mg vial and $4,227.75 per 500mg vial. However, actual costs to individual patients and providers are likely to be lower as WAC does not take additional rebates and discounts into account. Teva will also provide dedicated patient support services through the CORE program.

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Vir-Alnylam's COVID-19 Therapy, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

Biotech executive, Julia Berretta, Ph.D., is named Chief Executive Officer

Genespire, a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, announced today the successful close of a 16M Series A financing from Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan. The company also announced the appointment of Julia Berretta, Ph.D., as Chief Executive Officer and member of the Board of Directors. Graziano Seghezzi, Managing Partner at Sofinnova Partners, and Lucia Faccio, Ph.D., Partner at Sofinnova Partners, will also join the Board.

Genespire was founded in March 2020 as a spin-off of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), one of the worlds leading cell and gene therapy research institutes spearheaded by gene therapy pioneer Prof. Luigi Naldini. The Company was co-founded by Fondazione Telethon and the San Raffaele Hospital, along with Prof. Naldini and Dr. Alessio Cantore.

The funds will be used to advance Genespires leading-edge platform technologies towards the development of novel gene therapies in two main areas: primary immunodeficiencies and metabolic genetic diseases.

"Our mission has always been to develop breakthrough solutions for genetic diseases," said Prof. Naldini, Genespires co-founder and Director of SR-Tiget. "This financing enables the company to translate our innovative science and early stage programs into clinical development. The appointment of Dr. Berretta as CEO is a major reinforcement of our team."

Sofinnova Partners Dr. Faccio added, "Genespire is an exciting investment with all the key ingredients for success: Outstanding scientists that developed the first ex-vivo gene therapy to market, experienced executives brought in through Sofinnova Partners network and game changing technologies that have the potential to impact the lives of patients with genetic diseases."

"I am thrilled to be joining Genespire and such exceptional scientific founders," said Dr. Berretta. "Genespire was born of decades of experience in the gene therapy field, and is optimally positioned to advance transformative therapies for patients affected by severe inherited diseases."

Dr. Berretta was part of the Executive Committee of Cellectis S.A., a Nasdaq-listed clinical stage gene editing company developing CAR-T cell therapies for cancer, where she led business development as well as strategic planning. She is also an independent Board member of Treefrog Therapeutics, an innovative stem cell company.

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.com

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management.

For more information, please visit: http://www.sofinnovapartners.com

About Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit: http://www.telethon.it

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About Ospedale San Raffaele

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctor to grow professionally. For more information, please visit: http://www.hsr.it

About the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200429005417/en/

Contacts

Julia BerrettaCEO, Genespire S.r.linfo@genespire.com +39 02 83991300

Bommy LeeHead of Communications, Sofinnova Partnersblee@sofinnovapartners.com +33 (0) 6 47 71 38 11

North AmericaRooneyPartners LLCKate Barrettekbarrette@rooneyco.com +1 212 223 0561

FranceStrategiesImage (S&I)Anne Reinanne.rein@strategiesimage.com +33 6 03 35 92 05

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Genespire Secures 16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies - Yahoo Finance

The team that developed Zolgensma is back for round two. A group of former AveXis executives and investors unveiled a new gene therapy company, and theyre wasting no time. With 15 programs, $30 million in seed funding and an unrivaled partnership with UT Southwestern Medical Center, Taysha Gene Therapies plans to be in the clinic by the end of the year.

Its working on adeno-associated vector (AAV) gene therapies for monogenic diseasesthat is, diseases caused by a defect in a single geneof the central nervous system. Tayshas lead program targets GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord, but the companys portfolio includes conditions that are more prevalent.

The company plans to start clinical trials for three more programs by the end of 2021: treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome, Taysha CEO and co-founder R. A. Session II told FierceBiotech.

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And thats not allTaysha has the option to pick up four more prospects from UT Southwestern.

The reason why we can tackle 15 programs with the option to four additional programs is were able to focus on what we do best and were able to allow our collaborators to focus on what they do best, Session said.

Under their partnership, UT Southwestern is working on discovery and preclinical work all the way through IND-enabling studies. It has its own GMP viral manufacturing site to support that work, as well as clinical development, which Taysha will pick up. The company will also take care of regulatory strategy, commercial manufacturing and commercialization. Its a skill set that Tayshas management team honed at AveXis as itdeveloped the gene therapy that eventually became Novartis spinal muscular atrophy treatment Zolgensma.

We essentially flew the plane and built it at the same time when we were developing AveXis We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and were marrying that with a best-in-class academic research institution, Session said.

RELATED: FDA lets Novartis off the hook in Zolgensma data manipulation

Dividing the labor creates this engine for innovation that allows the partners to advance many programs in parallel, Session added. With about 50 people in its gene therapy unit, UT Southwestern can develop a capsidthe protein shell of a virusfor treatment delivery or get a candidate into animal models much more quickly than a biotech going it alone, he said. The same goes for late-stage development and commercialization on Tayshas side.

For its initial programs, Taysha is focusing on AAV gene therapies because the team knows they work.

AAV9 is the best way we have to treat monogenic CNS disease. If its not broke, dont fix it, Session said. We know how to effectively dose AAV9; we know its safe, effective and efficient. And we know its scalable. These are problems we had to solve at our previous company.

But, moving forward, Taysha and UT Southwestern are working on new technologies, including an AAV delivery platform that would allow for the redosing of gene therapies as well as an AAV capsid platform aimed at improving target delivery.

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Taysha Gene Therapies hits the ground running with $30M, 15 programs - FierceBiotech

BOSTON & WALTHAM, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and Affinia Therapeutics announced today that the two companies have entered into a strategic research collaboration to engineer novel adeno-associated virus (AAV) capsids to deliver transformative genetic therapies to people with serious diseases. Affinia Therapeutics proprietary AAVSmartLibrary and associated technology provides capsids for improved tissue tropism, manufacturability and pre-existing immunity. The collaboration will leverage Affinia Therapeutics capsid engineering expertise and Vertexs scientific, clinical and regulatory capabilities to accelerate the development of genetic therapies for people affected by Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and cystic fibrosis (CF).

This collaboration with Affinia Therapeutics will enhance our existing capabilities in discovering and developing transformative therapies for people with serious diseases, said Bastiano Sanna, Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. Affinia Therapeutics innovative approach to the discovery and design of AAV capsids brings yet another tool to our Vertex Cell and Genetic Therapies toolkit, and were excited to partner with them to bring together their technology platform with our research and development expertise.

At Affinia Therapeutics, were setting a new standard in genetic therapy by leveraging our platform to methodically engineer novel AAV vectors that have unique therapeutic properties, said Rick Modi, Chief Executive Officer. Vertex is an established leader in developing transformative medicines for genetic diseases and renowned for its scientific rigor. We are thankful for the scientific validation this partnership brings and look forward to working closely with them to advance life-changing, differentiated genetic therapies and make a meaningful difference to those affected by these diseases.

About the Collaboration

Under the terms of the agreement, Affinia Therapeutics will apply its vector design and engineering technologies to develop novel capsids with improved properties. The agreement provides Vertex an exclusive license under Affinia Therapeutics proprietary technology and intellectual property (IP) in DMD and DM1 with an exclusive option to license rights for CF and an additional undisclosed disease. The scope of the agreement covers all genetic therapy modalities in these diseases. Affinia Therapeutics will be eligible to receive over $1.6 billion in upfront and development, regulatory and commercial milestones, including $80 million in upfront payments and research milestones that will be paid during the research term, plus tiered royalties on future net global sales on any products that result from the collaboration. Affinia Therapeutics will be responsible for the discovery of capsids that meet certain pre-determined criteria. Vertex will be responsible for and will fund the design and manufacturing of genetic therapies incorporating the selected capsids, preclinical and clinical development efforts, and commercialization of any approved products in the licensed diseases.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need. http://www.affiniatx.com.

About Vertex Pharmaceuticals

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, UK. 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 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. 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, Dr. Sannas statements in the second paragraph of the press release, Mr. Modis statements in the third paragraph of the press release, and statements regarding future activities of the parties pursuant to the collaboration. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent Vertex's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, Vertex may not realize the anticipated benefits of the collaboration, and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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Vertex Pharmaceuticals and Affinia Therapeutics Establish Multi-Year Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies -...

In recent years, targeted cancer therapies have increased in use. These individualized treatments are based on the genetic makeup of a patient's disease.

Precision medicine in cancer is characterized by these personalized therapies, and it is tailored to address potential genetic changes in a patients tumor. By directly attacking the tumor, the researchers stress the impact this can have in protecting healthy cells that are also damaged during chemotherapy and radiotherapy, which can lead to severe toxicity in healthy tissues.

The researchers sought to provide an outline of emerging developments in targeted cancer therapy, particularly 2 areas of interest: immune-based therapy and enzyme-/small moleculesbased therapies. They included advancements in cancer targeted therapy, personalized medicine, and cancer combination therapies from research papers published from 2010 to June 2019.

Immune-Based Therapy

As the study authors discuss, immunotherapy-based strategies differ from both chemotherapy and radiotherapy-based treatments. Instead of directly attacking the growth of the cancer, the approach enhances the immune response to the cancer. Several notable innovations have increased the availability of this strategy, including immune checkpoint inhibitors (ICIs), immune cytokines, tumor-targeted superantigens, and ligand targeted therapeutics.

ICIs have served as major advancements in oncology, with several already approved by the FDA. Although their impact in revolutionizing the treatment of cancer has been noted, there have also been reports they could cause serious harm to patients due to their toxicity, with some recipients reporting immune-related adverse events. The researchers highlight that further studies are warranted to gain a detailed understanding of tumors' mechanisms of resistance to checkpoint inhibitors treatment, in order to improve the therapy.

Enzyme-/Small MoleculesBased Therapies

In enzyme-/small moleculesbased therapies, the researchers cited innovations, such as the use of a proteolysis targeting chimera (PROTAC), antibody-drug conjugates, and antibody-directed enzyme prodrug therapy.

The PROTAC strategy has been expanded and utilized in the treatment of many diseases, including ER-positive breast cancer, and to target steroid hormone receptors for ubiquitination and degradation in breast and prostate cancers.

As the researchers note, the approach is inspired by a mechanism in which the cell degrades unwanted proteins and consists of a molecule with 2 independent moieties. Recently, PROTAC was used to degrade the anaplastic lymphoma kinase, which has been associated with several types of human cancer.

The conventional therapeutic paradigm for cancer and other diseases has focused on a single type of intervention for all patients. However, a large literature in oncology supports the therapeutic benefits of a precision medicine approach to therapy as well as combination therapies, said the study authors.

Reference

Bashraheel SS, Domling A, Goda SK. Update on targeted cancer therapies, single or in combination, and their fine tuning for precision medicine [published online February 25, 2020]. Biomed Pharmacother. doi: 10.1016/j.biopha.2020.110009.

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Updates on Single, Combination Precision Medicine in Targeted Cancer Therapy - AJMC.com Managed Markets Network

You can't lump all clinical-stage biotechs together. TakeCara Therapeutics (NASDAQ:CARA) andSangamo Therapeutics (NASDAQ:SGMO), for example. The two biotechs are about as different as night and day. One thing that both Cara and Sangamo have in common, though, are promising pipeline candidates.

Investors have placed a greater value on Sangamo's pipeline based on the company's higher market cap. But which of these biotech stocks is the better pick for long-term investors? Here's how Cara and Sangamo stack up against each other.

Image source: Getty Images.

All of Cara Therapeutics' hopes ride on one drug -- Korsuva. But that one drug has multiple formulations and multiple opportunities.

The most promising of these opportunities is for Korsuva injection in treating chronic kidney disease-associated pruritis (CKD-aP) in patients on hemodialysis. The itching (pruritis) experienced by many patients with CKD is very problematic. So far, there haven't been any approved treatments for CKD-aP.

That could soon change. Last week, Cara and its partner, Vifor Fresenius Medical Care Renal Pharma, announced positive results from a global late-stage study of Korsuva injection in treating CKD-aP in patients on hemodialysis. These results reinforced positive results reported last year from a U.S. study. The two companies plan to file for U.S. regulatory approval for Korsuva injection in the second half of 2020, followed closely by filing for European approval of the drug.

Cara anticipates completing an interim statistical analysis within the next couple of months for a phase 2 study evaluating an oral version of Korsuva in treating pruritis in patients with atopic dermatitis. The biotech is also conducting another phase 2 study of oral Korsuva in treatingpatients with pruritus and hepatic impairment due to primary biliary cholangitis (PBC).

Oral Korsuva should advance to a late-stage study in treating CKD-aP in patients who aren't on hemodialysis later this year. Cara reported results from a phase 2 study in December with the drug meeting its primary endpoint but failing to meet two secondary endpoints.

Korsuva injection could achieve peak annual sales topping $500 million. An oral version of the drug could boost that number considerably. With Cara Therapeutics' market cap currently below $750 million, the stock should have plenty of room to run if Korsuva wins regulatory approvals and reaches its commercial potential.

Unlike Cara Therapeutics, Sangamo isn't hanging its hat on only one candidate. The biotech's pipeline includes five different therapies in clinical testing. However, none of these programs are yet in late-stage testing.

Sangamo specializes in gene editing using a technique known as zinc finger nuclease (ZFN) technology and in developing gene therapies. The company's approach has captured attention from several big drugmakers.

Pfizeris partnering with Sangamo on developing SB-525, a gene therapy targeting hemophilia A. Sanofiteamed up with Sangamo on two gene-edited cell therapies in clinical studies. ST-400 targets treatment of rare blood disease beta-thalassemia, while BIVV-003 targets treatment of sickle cell disease.

Sangamo also has two wholly owned programs in phase 1/2 studies. Gene therapy ST-920 targets Fabry disease. ZFN gene-editing therapy SB-913 targets rare genetic diseasemucopolysaccharidosis type II (MPS II).

In addition to its clinical programs, Sangamo has 11 therapies in preclinical testing. Gilead Sciencesis collaborating with Sangamo on two of these preclinical programs. Pfizer and Japanese drugmaker Takeda have teamed up with the biotech on one preclinical program each.

In February, Sangamo announced a dealwith Biogen to develop gene therapies targeting neurological diseases. Three of Sangamo's preclinical programs are included in this agreement.

Sangamo's market cap stands at a little under $1 billion. While the company has a long way to go before any of its candidates could potentially win approval, it also has multiple shots on goal. Successes for only one or two of its experimental therapies would enable this small biotech to become much larger.

I think that both of these stocks could be big winners over the long run. However, my view is that Cara Therapeutics offers a better risk-reward proposition.

Although it's not a slam dunk that Cara wins FDA and EU approvals for Korvuva injection, I think the odds appear to be pretty good. Assuming those approvals are secured, Cara should be set to begin pulling in some solid revenue by late 2021.

Having said that, it wouldn't surprise me if Sangamo achieves greater success by the end of this decade. I think that investors should keep the biotech on their radar screens.

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Better Buy: Cara Therapeutics vs. Sangamo Therapeutics - Motley Fool