Putting The Costs And Benefits Of New Gene Therapies Into Perspective

JOSHUA T. COHEN | September 4, 2019 | 28 views

Cell and gene therapies have the potential to revolutionize treatment for a range of severe diseases, but their introductions also force difficult economic conversations. This blog post offers three observations to help the community evaluate the costs and benefits of new—and in many cases, extremely costly—therapies.

Spotlight

Krishidhan Seeds Limited

Our R&D activities and research centers are recognized by the Department of Scientific and Industrial Research (DSIR), Govt. of India. We see ourselves emerging as one of the biggest technology driven Indian agricultural input company with a significant global presence, providing access to latest technologies and all required quality agricultural inputs for the socio economic growth of farmers worldwide.

OTHER ARTICLES
MEDTECH

Expansion of BioPharma: Opportunities and Investments

Article | July 12, 2022

Biopharmaceutical innovations are among the most ingenious and refined achievements of modern medical science. New concepts, techniques, and therapies are emerging, such as the cell therapy Provenge, which can be used to treat cancer, and gene therapies, which provide even more amazing promises of disease remission and regenerative medicine. In addition, the COVID-19 pandemic has caused a huge boom in the pharmaceutical industry. This is because more and more attention is being paid to increasing manufacturing capacity and starting new research on drug development. Biopharma: Leading the Way in the Pharma Sector In the past couple of years, the biopharmaceutical sector has deepened its roots across the medical and pharmaceutical industries, on account of the transformation of pharmaceutical companies towards biotechnology, creating opportunities for growth. Also, growing advancements in technologies such as 3D bioprinting, biosensors, and gene editing, along with the integration of advanced artificial intelligence and virtual and augmented reality are estimated to further create prospects for growth. According to a study, the biopharmaceutical sector makes nearly $163 billion around the world and grows by more than 8% each year, which is twice as fast as the traditional pharma sector. Massive Investments Directed Towards Biopharma Investing in biotech research and development (R&D) has yielded better returns than the pharma industry average. Hence, a number of pharmaceutical companies are shifting their presence toward biopharma to capitalize on the upcoming opportunities by investing in and expanding their biotechnology infrastructure. For instance, Thermo Fisher Scientific Inc., an American manufacturer of scientific instrumentation, reagents and consumables, and software services, announced an investment of $97 million to expand its bioanalytical laboratory operations into three new locations in the U.S. With this investment, the company will add 150,000 square feet of scientific workspace and install the most advanced drug development technologies to produce life-changing medicines for patients in need.

Read More
MEDICAL

2022 U.S. Market Research Report with COVID-19 Forecasts2

Article | July 14, 2022

The global biotechnology market is expected to grow at a compound annual growth rate (CAGR) of 13.9 percent from 2022 to 2030, with a value estimated at USD 1,023.92 billion in 2021. The market is being propelled by strong government support in the form of initiatives aimed at modernizing the regulatory framework, improving approval processes and reimbursement policies, and standardizing clinical studies. The growing presence of personalized medicine and an increasing number of orphan drug formulations are opening up new avenues for biotechnology applications and driving the influx of emerging and innovative biotechnology companies, which is driving market revenue even further. The 2022 Biotech Research and Development Market Research Report is one of the most comprehensive and in-depth assessments of the industry in the United States, containing over 100 data sets spanning the years 2013 to 2026. This Kentley Insights report contains historical and forecasted market size, product lines, profitability, financial ratios, BCG matrix, state statistics, operating expense details, organizational breakdown, consolidation analysis, employee productivity, price inflation, pay bands for the top 20 industry jobs, trend analysis and forecasts on companies, locations, employees, payroll, and much more. Companies in the Biotech Research and Development industry are primarily engaged in biotechnology research and experimental development. Biotechnology research and development entails the investigation of the use of microorganisms and cellular and bimolecular processes to create or modify living or non-living materials. This biotechnology research and development may result in the development of new biotechnology processes or prototypes of new or genetically altered products that can be replicated, used, or implemented by various industries. This report was created using the findings of extensive business surveys and econometrics. The professionals follow reports with accurate and apt information on market sizing, benchmarking, strategic planning, due diligence, cost-cutting, planning, understanding industry dynamics, forecasting, streamlining, gap analysis, and other ana

Read More
MEDTECH

Biotech in 2022

Article | July 13, 2022

The robust global channel of more than, 800 gene and cell curatives presently in trials will produce clinical readouts in 2022, revealing what lies ahead for advanced curatives. The impact will be felt in 2022, no matter how you slice it. Eventually, how well industry and non-supervisory bodies unite to produce new frameworks for advanced therapies will shape the year 2022 and further. Pacific Northwest talent will continue to contribute to the advancement of gene and cell curatives in both the short and long term, thanks to its deep pool of ground-breaking scientific developers, entrepreneurial directorial leadership, largely skilled translational scientists, and endured bio manufacturing technicians. We may see continued on-life science fund withdrawal from biotech in 2021, but this can be anticipated as a strong comeback in 2022 by biotech industry, backed by deep-pocketed life science investors who are committed to this sector. A similar investment, combined with pharma's cash-heavy coffers, can result in increased junction and acquisition activity, which will be a challenge for some but an occasion for others. Over the last five years, investment interest in Seattle and the Pacific Northwest has grown exponentially, from Vancouver, British Columbia, to Oregon. The region's explosive portfolio of new biotech companies, innovated out of academic centres, demonstrates the region's growing recognition of scientific invention. This created a belief that continued, especially because Seattle's start-ups and biotech enterprises are delivering on their pledge of clinical and patient impact. Talent and staffing will continue to be difficult to find. It's a CEO's market, but many of these funds' return, and are not rising in proportion to the exorbitant prices they're paying to enter deals. This schism has become particularly pronounced in 2021. Hence, everyone in biotech is concerned about reclamation and retention.

Read More
MEDTECH

Next-Gen Genetics Cancer Therapies Creating Investment Prospects

Article | July 5, 2022

Genetic therapeutics such as genetic engineering and gene therapy are increasingly emerging as one of the most influential and transformed biotechnological solutions around the globe in recent times. These genetic solutions are being assessed across various medical domains, including cancer treatment, neurology, oncology, and ophthalmology. Citing the trend, the genetics industry is estimated to experience a tsunami of approvals, with over 1,000 cell and gene therapy clinical trials currently underway and over 900 companies worldwide focusing on these cutting-edge therapies. Growing Cancer Encourages Advancements in Genetic Technologies With the surging cases of cancers such as leukemias, carcinomas, lymphomas, and others, patients worldwide are increasing their spending on adopting novel therapeutic solutions for non-recurring treatment of the disease, such as gene therapy, genetic engineering, T-cell therapy, and gene editing. As per a study by the Fight Cancer Organization, spending on the treatment of cancer increased to $200.7 billion, and the amount is anticipated to exceed $245 billion by the end of 2030. Growing revenue prospects are encouraging biotechnology and biopharmaceutical companies to develop novel genetic solutions for cancer treatment. For instance, Bristol-Myers Squibb K.K., a Japanese pharmaceutical company, introduced a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, Abecma, for the treatment of relapsed or refractory (R/R) multiple myeloma in 2022. Amid a New Market: Genetics Will Attract Massive Investments Despite several developments and technological advancements, genetics is still considered to be in a nascent stage, providing significant prospects for growth to the companies that are already operating in the domain. Genetics solutions such as gene therapies, gene editing, and T-cell immunotherapy are emerging as highly active treatments across various medical fields, resulting in increasing research and development activities across the domain, drawing significant attention from investors. Given the potential of genetic treatments and the focus on finding new ways to treat cancer and other related diseases, it's easy to understand why companies are investing in the domain. For instance, Pfizer has recently announced an investment of around $800 million to construct development facilities supporting gene therapy manufacturing from initial preclinical research through final commercial-scale production. Due to these advancements, cell and gene therapies are forecast to grow from $4 billion annually to more than $45 billion, exhibiting growth at a 63% CAGR. The Future of Genetics Though there is a significant rise in advancement in genetic technologies and developments, the number of approved genetic treatments remains extremely small. However, with gene transfer and CRISPR solutions emerging as new modalities for cancer treatment, the start-up companies will attract a growing amount and proportion of private and public investments. This is expected present a tremendous opportunity for biopharma and biotechnology investors to help fund and benefit from the medical industry's shift from traditional treatments to cutting-edge genetic therapeutics in the coming years.

Read More

Spotlight

Krishidhan Seeds Limited

Our R&D activities and research centers are recognized by the Department of Scientific and Industrial Research (DSIR), Govt. of India. We see ourselves emerging as one of the biggest technology driven Indian agricultural input company with a significant global presence, providing access to latest technologies and all required quality agricultural inputs for the socio economic growth of farmers worldwide.

Related News

CELL AND GENE THERAPY

iTolerance to Collborate with LyGenesis for Joint Research

iTolerance | July 01, 2022

iTolerance, Inc., an early-stage privately held regenerative medicine company developing technology to enable organoid, tissue, and cell therapy, announced entering into a joint collaboration with LyGenesis, Inc., a clinical-stage biotechnology company with an organ regeneration technology platform. The joint collaboration is aimed at evaluating the potential of iTOL-201, a product candidate being developed combining LyGenesis' LYG-LIV-100 liver cell therapy and iTolerance's SA FasL microgel immune tolerance platform to permit the growth of ectopic livers without the requirement for immune suppression. The joint research effort of iTolerance and LyGenesis has produced in vitro data using iTOL-201 and is now moving toward small animal proof of concept work to assess the potential of the combined technology for producing ectopic livers capable of saving the animals from fatal liver disorders without the requirement for immune suppression. With our lead therapy now in the clinic in a Phase 2a trial in patients with End Stage Liver Disease, we have turned our attention toward a second-generation therapy capable of growing ectopic organs without the need for immune suppression. iTolerance's platform holds enormous promise in this respect and we look forward to the results from our joint proof of concept work." Dr. Michael Hufford, Co-Founder and Chief Executive Officer of LyGenesis. While long-term immunosuppression continues to be an obstacle for the use of cell and regenerative therapies, the research being conducted between both LyGenesis and iTolerance could allow for a major advancement in organ regeneration, As we advance our own pipeline of therapies focused on supporting pancreatic islet engraftments, I believe this synergistic collaborative research with LyGenesis has the potential to successfully combine technologies to drive significant value for both biotech companies and importantly, the patients we work to serve." Dr. Anthony Japour, Chief Executive Officer of iTolerance.

Read More

MEDICAL

Anitoa Launches Molecular Test (PCR) for Monkeypox

Anitoa | June 30, 2022

Anitoa Systems, market leader in fast, portable molecular testing, today announced the availability of a CE-marked turn-key PCR solution for testing Monkeypox. This portable solution includes one of a family small foot-print real time PCR instruments called the Maverick qPCR, with an extraction-free multiplex PCR reagent that tests OPXV/MPXV gene-targets in the skin lesion sample from patients suspected of infection with Monkeypox. Anitoa's Monkeypox test is designed to be carried out on-site, near patient, thus avoiding the need to transport patient samples to central labs for testing. The World Health Organization reported that over 2,100 people in 42 countries have been diagnosed with monkeypox as of June 15, nearly all during the past two months. As of June 17, the CDC reports 24 confirmed monkeypox cases in California, US. Although The CDC reports that the threat to the general U.S. population is considered low, it is important for healthcare providers worldwide to have a preparedness plan. Already having a presence in Africa, Anitoa Systems has started deloying its near-patient Monkeypox test in selected clinics in Africa. Anitoa is prepared to deliver its Monkeypox test solution world-wide when the need arises. Anitoa's Maverick line of multiplex real time PCR devices are lab-accurate, fast, portable and calibration-free. They can operate on battery power (including car battery) and takes up very little bench space. Maverick qPCR instruments are built on Anitoa's proprietary CMOS biosensor technology. They have been used widely for on-site testing of food, environment samples and agriculture products due to its rugged all solid-state design, small form factor, and lower operation cost. Anitoa's Monkeypox detection solution is part of an on-going program at Anitoa to develop tests to help combat a series of neglected tropical diseases in low resource settings. These tests currently include IVDR tests for Dengue, Yellow Fever, and Malaria We are closely monitoring the situation of Monkeypox infections around the world and tailoring our test solutions to the needs of the people affected." Zhimin ding, CEO of Anitoa Systems, LLC About Anitoa Anitoa Systems, a medical device technology start-up established in 2013 with headquarter in Sunnyvale, California, develops highly integrated and portable bio-sensor devices for molecular detection, based on its unique CMOS image sensor-based fluorescence and chemiluminescence molecular imaging. By replacing the traditional bulky and costly photon-multiplier tube (PMT) and CCD-based optical sub-systems, Anitoa's ultra-low-light CMOS sensor technology enables a new generation of compact and inexpensive molecular testing platforms targeting infectious, oncology and cardio-vascular markers. Anitoa manufactures its qPCR instruments and test kits in Hangzhou, China and Fremont, California.

Read More

MEDICAL

Hillstream BioPharma to Announce Collaborating with Sapien Biosciences

Hillstream | June 30, 2022

Hillstream BioPharma, Inc., a U.S.-based biotechnology company developing novel therapeutic candidates targeting ferroptosis for cancer treatment, announced a collaboration with an Indian pioneer biobank and peonalized medicine company that leverages Saarum Innovation's global life sciences research expertise, Sapien Biosciences. The collaboration is aimed at assessing the compatibility of HSB-1216 with immune checkpoint inhibitors. HSB-1216, Hillstream’s most advanced candidate, is an IMCD modulator that targets various types of solid tumors. In an exhaustive study in Germany, the active drug in HSB-1216 was found to be efficacious in a clinical pilot for devastating cancers, including epithelial carcinomas and triple-negative breast cancer. Hillstream aims to build an early clinical proof-of-concept by beginning a clinical investigation with HSB-1216 in 2022. With this collaboration, Hillstream will be able to use Sapien’s patient samples and rich datasets to validate its HSB-1216 for the development of novel cancer therapeutics. We are initiating studies, in collaboration with Sapien Biosciences, to determine the synergy between HSB-1216, our ferroptosis inducer and nivolumab, an anti-PD1 antibody.” He stated, “In addition, we will also study the potential synergistic effects of HSB-1216 with the recently approved Bristol Myers Squibb drug Opdualag® (nivolumab + relatlimab), a fixed dose combination of an anti-PD1 MAb + anti-LAG3 MAb, to demonstrate that targeting a ferroptosis-associated metabolism in tumors may improve the efficacy of cancer immunotherapy.” Randy Milby, Hillstream’s Chief Executive Officer. We are excited to extend our collaboration with Hillstream Biopharma to evaluate potential synergy between their HSB-1216 molecule and ICIs using our expertise in establishing patient tissue-derived cell models in oncology, immunology and Immune-oncology Previously, Sapien demonstrated potent anti-cancer activity of HSB-1216 in primary triple negative breast cancer cells which were presented at AACR 2020.” Dr. Jugnu Jain, CEO, Sapien Biosciences.

Read More

CELL AND GENE THERAPY

iTolerance to Collborate with LyGenesis for Joint Research

iTolerance | July 01, 2022

iTolerance, Inc., an early-stage privately held regenerative medicine company developing technology to enable organoid, tissue, and cell therapy, announced entering into a joint collaboration with LyGenesis, Inc., a clinical-stage biotechnology company with an organ regeneration technology platform. The joint collaboration is aimed at evaluating the potential of iTOL-201, a product candidate being developed combining LyGenesis' LYG-LIV-100 liver cell therapy and iTolerance's SA FasL microgel immune tolerance platform to permit the growth of ectopic livers without the requirement for immune suppression. The joint research effort of iTolerance and LyGenesis has produced in vitro data using iTOL-201 and is now moving toward small animal proof of concept work to assess the potential of the combined technology for producing ectopic livers capable of saving the animals from fatal liver disorders without the requirement for immune suppression. With our lead therapy now in the clinic in a Phase 2a trial in patients with End Stage Liver Disease, we have turned our attention toward a second-generation therapy capable of growing ectopic organs without the need for immune suppression. iTolerance's platform holds enormous promise in this respect and we look forward to the results from our joint proof of concept work." Dr. Michael Hufford, Co-Founder and Chief Executive Officer of LyGenesis. While long-term immunosuppression continues to be an obstacle for the use of cell and regenerative therapies, the research being conducted between both LyGenesis and iTolerance could allow for a major advancement in organ regeneration, As we advance our own pipeline of therapies focused on supporting pancreatic islet engraftments, I believe this synergistic collaborative research with LyGenesis has the potential to successfully combine technologies to drive significant value for both biotech companies and importantly, the patients we work to serve." Dr. Anthony Japour, Chief Executive Officer of iTolerance.

Read More

MEDICAL

Anitoa Launches Molecular Test (PCR) for Monkeypox

Anitoa | June 30, 2022

Anitoa Systems, market leader in fast, portable molecular testing, today announced the availability of a CE-marked turn-key PCR solution for testing Monkeypox. This portable solution includes one of a family small foot-print real time PCR instruments called the Maverick qPCR, with an extraction-free multiplex PCR reagent that tests OPXV/MPXV gene-targets in the skin lesion sample from patients suspected of infection with Monkeypox. Anitoa's Monkeypox test is designed to be carried out on-site, near patient, thus avoiding the need to transport patient samples to central labs for testing. The World Health Organization reported that over 2,100 people in 42 countries have been diagnosed with monkeypox as of June 15, nearly all during the past two months. As of June 17, the CDC reports 24 confirmed monkeypox cases in California, US. Although The CDC reports that the threat to the general U.S. population is considered low, it is important for healthcare providers worldwide to have a preparedness plan. Already having a presence in Africa, Anitoa Systems has started deloying its near-patient Monkeypox test in selected clinics in Africa. Anitoa is prepared to deliver its Monkeypox test solution world-wide when the need arises. Anitoa's Maverick line of multiplex real time PCR devices are lab-accurate, fast, portable and calibration-free. They can operate on battery power (including car battery) and takes up very little bench space. Maverick qPCR instruments are built on Anitoa's proprietary CMOS biosensor technology. They have been used widely for on-site testing of food, environment samples and agriculture products due to its rugged all solid-state design, small form factor, and lower operation cost. Anitoa's Monkeypox detection solution is part of an on-going program at Anitoa to develop tests to help combat a series of neglected tropical diseases in low resource settings. These tests currently include IVDR tests for Dengue, Yellow Fever, and Malaria We are closely monitoring the situation of Monkeypox infections around the world and tailoring our test solutions to the needs of the people affected." Zhimin ding, CEO of Anitoa Systems, LLC About Anitoa Anitoa Systems, a medical device technology start-up established in 2013 with headquarter in Sunnyvale, California, develops highly integrated and portable bio-sensor devices for molecular detection, based on its unique CMOS image sensor-based fluorescence and chemiluminescence molecular imaging. By replacing the traditional bulky and costly photon-multiplier tube (PMT) and CCD-based optical sub-systems, Anitoa's ultra-low-light CMOS sensor technology enables a new generation of compact and inexpensive molecular testing platforms targeting infectious, oncology and cardio-vascular markers. Anitoa manufactures its qPCR instruments and test kits in Hangzhou, China and Fremont, California.

Read More

MEDICAL

Hillstream BioPharma to Announce Collaborating with Sapien Biosciences

Hillstream | June 30, 2022

Hillstream BioPharma, Inc., a U.S.-based biotechnology company developing novel therapeutic candidates targeting ferroptosis for cancer treatment, announced a collaboration with an Indian pioneer biobank and peonalized medicine company that leverages Saarum Innovation's global life sciences research expertise, Sapien Biosciences. The collaboration is aimed at assessing the compatibility of HSB-1216 with immune checkpoint inhibitors. HSB-1216, Hillstream’s most advanced candidate, is an IMCD modulator that targets various types of solid tumors. In an exhaustive study in Germany, the active drug in HSB-1216 was found to be efficacious in a clinical pilot for devastating cancers, including epithelial carcinomas and triple-negative breast cancer. Hillstream aims to build an early clinical proof-of-concept by beginning a clinical investigation with HSB-1216 in 2022. With this collaboration, Hillstream will be able to use Sapien’s patient samples and rich datasets to validate its HSB-1216 for the development of novel cancer therapeutics. We are initiating studies, in collaboration with Sapien Biosciences, to determine the synergy between HSB-1216, our ferroptosis inducer and nivolumab, an anti-PD1 antibody.” He stated, “In addition, we will also study the potential synergistic effects of HSB-1216 with the recently approved Bristol Myers Squibb drug Opdualag® (nivolumab + relatlimab), a fixed dose combination of an anti-PD1 MAb + anti-LAG3 MAb, to demonstrate that targeting a ferroptosis-associated metabolism in tumors may improve the efficacy of cancer immunotherapy.” Randy Milby, Hillstream’s Chief Executive Officer. We are excited to extend our collaboration with Hillstream Biopharma to evaluate potential synergy between their HSB-1216 molecule and ICIs using our expertise in establishing patient tissue-derived cell models in oncology, immunology and Immune-oncology Previously, Sapien demonstrated potent anti-cancer activity of HSB-1216 in primary triple negative breast cancer cells which were presented at AACR 2020.” Dr. Jugnu Jain, CEO, Sapien Biosciences.

Read More

Events