Kenny Aladefa MD.MBA.OLY.’s Post

Underfunded No More: Biotech's Impact on Rare Diseases  🌟 Rare diseases, affecting over 300 million people worldwide, often go underfunded and understudied due to their low prevalence. However, biotechnology is transforming the landscape of rare disease treatment, providing new hope through innovative therapies and personalized approaches. Here’s how: Challenges of Rare Diseases - Rare diseases, defined as conditions affecting fewer than 1 in 2,000 people, pose significant challenges due to limited patient populations and the complexity of many conditions. This makes drug development costly and time-consuming, often leading to underfunding Biotech Innovations Addressing Rare Diseases: Gene Therapy 🧬: Gene therapies are designed to address the root causes of genetic disorders. Krystal Biotech, Inc.’s Vyjuvek, a topical gene therapy, treats dystrophic epidermolysis bullosa (DEB), a rare skin disease, by delivering functional copies of the COL7A1 gene to the skin cells. This approach significantly improves patients' quality of life by reducing the severity of their symptoms. RNA-based Therapies 💉: RNA-based therapies, such as those developed by Alltrna, utilize transfer RNA (tRNA) to correct genetic mutations. This innovative approach can potentially treat numerous rare diseases by targeting specific genetic errors, offering a broad spectrum of treatment possibilities. Collaborative Initiatives 🌐: Bespoke Gene Therapy Consortium (BGTC): This initiative accelerates the development of gene therapies tailored to individual rare diseases. By focusing on shared genetic pathways, the BGTC aims to streamline the creation of bespoke treatments, reducing development time and costs. Success Stories and Recent Advances: FDA Approvals: New drug approvals, like Skyclarys for Friedreich’s ataxia, showcase the rapid advancements in rare disease treatments. These drugs not only provide essential therapies but also validate the potential of biotech in addressing previously untreatable conditions. Krystal Biotech’s Vyjuvek: As the first topical gene therapy for DEB, Vyjuvek represents a major milestone in treating genetic skin disorders. Its success underscores the potential of gene therapy to transform patient care. The Future of Rare Disease; Treatment - Biotechnology is breaking down barriers in rare disease treatment, from gene editing to personalized medicine. Continued research and innovation are crucial to developing effective therapies for the millions affected by rare diseases. Collaborative efforts and advancements in biotech promise a brighter future for these patients. Stay tuned for more biotech insights in our 100 Days 100 Learnings series! 🚀 #RareDiseases #GeneTherapy #PersonalizedMedicine #Biotech #MedicalResearch #100Days100Learnings 💯📚

We are entering a highly innovative era in medicine. The fundamental principles of genetic engineering, repair, and editing are currently being translated into novel therapeutic approaches. In addition to these exciting developments, there have been concerns expressed by patients, regulators, and ethicists. New genetic therapies have a high price tag, even with uncertain long-term outcomes. These substantial expenses impede the accessibility of both the therapies themselves and the infrastructure required for their production. The infrastructure that supports the research and development of these genetic medicines benefits only the nations and corporations of the nations where these therapies were developed. Hence, the developing world is deprived of access due to the cost and deprived of the development of infrastructure that can facilitate innovation and entrepreneurship, despite the primary target populations for many of these gene therapy products being located in low- and middle-income countries (LMICs). Genetic therapies would be a one-time treatment that would benefit millions in an ideal world. Nevertheless, novel advancements cannot be so naive as to overlook the inevitable obstacles that arise between clinical triumph in a limited number of clinical trial participants and universal accessibility for millions of individuals worldwide. Although genetic therapies have the potential to alter people's lives for the better, the cost of current therapies and the high potential cost of those still in development will severely limit people's access to these durable treatments and potential cures. Yescarta, a CAR-T cell therapy for non-Hodgkin's lymphoma, is the least expensive cell therapy approved by the US FDA and costs approximately $373,000 per treatment. For factor IX hemophilia (hemophilia B), new approved gene therapies exceed $3 million, whereas the cost for a gene or cell therapy product that could treat or potentially cure sickle cell disease (SCD) is $2.8 million. Since the vast majority of patients with SCD do not have health care insurance, the vast majority of patients who would benefit will be left looking in on the wealthy few. The Global Gene Therapy Initiative (GGTI) was created in 2020 in order to address barriers to LMIC inclusion in gene and cell therapy development, including curative approaches for SCD and HIV. The GGTI brings together researchers, clinicians, regulators, funders, and other diverse stakeholders from various sectors to establish a sustainable pathway for implementing gene and cell therapies in LMICs. We cannot miss this opportunity to build trust between physicians, scientists, and, most importantly, the communities that will benefit the most from advances in gene and cell therapies.

Future of gene therapy is incredibly promising, extending beyond rare diseases to broader applications like treating common conditions with transformative potential, offering new hope for millions of patients worldwide. Continued investment in research and development, along with thoughtful consideration of the ethical and regulatory landscape, will be key to realizing the full potential of this innovative approach.    At Cipla, we focus on innovation and are excited about these advancements and their implications to provide cutting-edge healthcare solutions to patients. Our strategic investment with Ethris for the development of messenger RNA (mRNA)-based therapies underscores our commitment to pioneering solutions, especially for bringing innovative treatments for patients with respiratory diseases. Inhaled gene therapy stands out as a promising approach for treating a variety of respiratory diseases. By delivering genetic material directly to the lungs, this method opens new possibilities for patients with conditions that have been challenging to treat with conventional therapies. https://lnkd.in/dUYUdSEX   #Transformation #Gene-therapy #Innovation

The Development of New Gene Therapies to Address Unmet Needs in ALS 👉 Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, is a devastating motor neuron disorder characterized by the progressive loss of motor neurons in the brainstem and spinal cord. This results in symptoms such as muscle stiffness, gradual weakening, and eventually the loss of essential functions like walking, speaking, swallowing, and breathing. 👉 With an annual #incidence of 2-3 cases per 100,000 individuals in the USA and Europe, the #prevalence of ALS is comparable to multiple sclerosis and significantly higher than that of Huntington's disease (HD). Despite ongoing research efforts, ALS remains incurable and fatal, with a median survival time of 3 years. However, treatments like Sanofi’s RILUTEK have shown #efficacy in extending the length and quality of life for #patients. 👉 In the field of ALS, over 40 #genes are linked to the disease, with key ones like C9orf72, SOD1, TARDBP, and FUS constituting up to 70% of familial cases. While there are approved #treatments to manage ALS symptoms, there are none for genetic forms. The demand for transformative cell and gene therapies is rising to address #unmet needs and track patients with causative gene mutations. 👉 In June 2024, AB Science's masitinib faced a setback in its application for conditional marketing authorization with EMA, highlighting the continued #challenges in finding effective solutions for ALS patients. 👉 Despite challenges, the past few years have witnessed a remarkable surge in therapeutic development, driven by profound advancements in understanding of ALS disease pathogenesis. Recently, several #investigational products have progressed into phase 2 and 3 RCT trials. (Refer to the key assets in the pipeline section of the carousel) 👉 In early September 2024, a noteworthy development took place as India's Zydus Group concluded a phase 2a 12-week study of usnoflast, a NLRP3 inhibitor for ALS, in India. This inhibitor reportedly achieves the therapeutic concentration in the cerebrospinal fluid of ALS patients. Moving forward, Zydus Life Sciences is gearing up to initiate a more extensive phase 2b trial in collaboration with regulatory authorities. #ALS #LouGehrigsDisease #Neurology #MedicalResearch #FDAApprovals #eosintelligence #perspectives #marketresearch #competitiveintelligence #pharmaceuticals #biotech #healthcare #strategy #leadership #management #marketing #raredisease #rarediseases

🥁 Exciting Updates in Cell and Gene Therapies: Early Fall Outlook 🌟 The cell and gene therapy space continues to thrive with notable breakthroughs: 🎉 #TransCodeTherapeutics: Received a $2 million NCI grant for its RNA therapy TTX-MC138, aimed at advanced solid tumors. This therapy utilizes an innovative approach to target and inhibit tumor growth, emphasizing TransCode's commitment to combating cancer. 🤝 #PrimeMedicine: Partnered with #BristolMyersSquibb on Prime Edited T-cell therapies, securing a $55 million upfront payment. This collaboration aims to leverage Prime's cutting-edge technology to develop therapies for various hematologic malignancies. 🧬 #EstrellaImmunopharma: Reported impressive response rates in patients treated with their CD19-targeted CAR-T therapy for B-cell lymphomas, showcasing potential as a leading treatment option for resistant lymphomas. 💉 #CureVac: Launched Phase 2 trials for its mRNA-based cancer vaccine CV8102, which shows promise in activating anti-tumor immune responses. 🧪 #Moderna: Advanced clinical trials for mRNA-137, targeting cystic fibrosis. This therapy represents a shift toward mRNA applications beyond vaccines. 💰 #AavantiBio: Announced positive Phase 1 results for its AAV gene therapy, demonstrating both safety and efficacy in patients with rare genetic disorders. 🚀 #BlueRockTherapeutics: Major news as BlueRock advances its Parkinson’s therapy, BRT-DA01, into Phase 2 clinical trials, aimed at restoring motor function by regenerating neurons. The company is also collaborating with #bitbio to develop iPSC-derived regulatory T cells (Tregs) for autoimmune diseases, expanding their therapeutic reach. 🔬 #CelyadOncology: Continues its strategic collaboration to innovate in CAR-T cell therapies, focusing on the development of next-generation cell therapies targeting various cancers. 🌍 #OrchardTherapeutics: Expanding its gene therapy pipeline for X-SCID (X-linked Severe Combined Immunodeficiency), aiming to provide effective treatment options for patients with this rare genetic disorder. 📈 Funding Trends: Despite economic pressures, the cell and gene therapy sector accounted for 19% of licensing deals, indicating solid investor interest and confidence in future innovations. 💡 Market Insights: Notable FDA-approved gene therapies like #Casgevy ($2.2 million) and #Lyfgenia ($3.1 million) demonstrate the high cost of these treatments, but their potential for long-term savings in healthcare costs makes them attractive options for managing rare diseases. More exciting innovations are shaping the future of healthcare. Stay tuned for the next wave of advancements! #CellTherapy #GeneTherapy #Biotech #Innovation #Healthcare #Partnerships #CancerResearch #cGMP #FDA

Why Accessibility is an Ethical Imperative In a country as diverse as India, healthcare inequality is a persistent challenge. If gene therapy remains the privilege of the wealthy, it risks exacerbating existing social and economic disparities. Everyone, regardless of their financial background, should have the right to access life-saving treatments. Gene therapy, with its potential to cure or dramatically alleviate chronic conditions, must not become a tool that further entrenches healthcare inequity. What Needs to Be Done 1. Government Subsidies and Pricing Regulations: The Indian government must consider policies that subsidize the cost of gene therapies, similar to how it has provided free or affordable access to essential medicines like antiretrovirals for HIV patients. Pricing regulations could also ensure that pharmaceutical companies do not charge exorbitant rates. 2. Public-Private Partnerships: Collaborative efforts between the government and private industry can help scale production, streamline research, and lower costs. In India, where government-funded health schemes such as Ayushman Bharat are beginning to improve healthcare access, incorporating gene therapy into such frameworks could be a game-changer. 3. Local Research and Development: Encouraging local R&D in gene therapy through increased funding for biotechnology and medical institutions would help reduce dependency on expensive foreign treatments. India’s strength in the generic pharmaceutical industry proves that it has the potential to become a global hub for affordable gene therapies as well. 4. Raising Awareness and Education: Educating both healthcare professionals and the general public about gene therapy’s potential is essential. Widespread understanding can lead to greater acceptance, demand for fair pricing, and pressure on policymakers to ensure broader access. A Call to Action India’s healthcare system is already under strain due to the dual burden of infectious and non-communicable diseases. However, gene therapy presents an opportunity to revolutionize treatment for genetic disorders and chronic illnesses. To fully realize this potential, it is critical that these treatments be accessible to everyone, not just the privileged few. Access to gene therapy should not be a matter of wealth but a matter of human rights. In a country committed to equity and inclusive growth, ensuring universal access to gene therapy can pave the way for a healthier, more equitable future.

📢 𝗧𝗵𝗲 𝗦𝗽𝗮𝗻𝗶𝘀𝗵 𝗣𝗿𝗶𝗰𝗶𝗻𝗴 𝗖𝗼𝗺𝗺𝗶𝘀𝘀𝗶𝗼𝗻 (𝗖𝗜𝗣𝗠) 𝗵𝗮𝘀 𝗽𝗿𝗼𝗽𝗼𝘀𝗲𝗱 𝘁𝗵𝗲 𝘁𝗼𝘁𝗮𝗹 𝗼𝗿 𝗽𝗮𝗿𝘁𝗶𝗮𝗹 𝗿𝗲𝗶𝗺𝗯𝘂𝗿𝘀𝗲𝗺𝗲𝗻𝘁 𝗼𝗳 5 𝗻𝗲𝘄 𝗱𝗿𝘂𝗴𝘀 (1 𝙤𝙛 𝙩𝙝𝙚𝙢 𝙤𝙧𝙥𝙝𝙖𝙣 𝙙𝙧𝙪𝙜𝙨) 𝗮𝗻𝗱 𝗮 𝗻𝗲𝘄 𝗶𝗻𝗱𝗶𝗰𝗮𝘁𝗶𝗼𝗻 𝗳𝗼𝗿 𝗮 𝗱𝗿𝘂𝗴 𝗮𝗹𝗿𝗲𝗮𝗱𝘆 𝗮𝘂𝘁𝗵𝗼𝗿𝗶𝘇𝗲𝗱 𝗮𝗻𝗱 𝗽𝗿𝗲𝘃𝗶𝗼𝘂𝘀𝗹𝘆 𝗳𝗶𝗻𝗮𝗻𝗰𝗲𝗱. ⁣⁣⁣⁣ We are very pleased to share good news for 𝗽𝗮𝘁𝗶𝗲𝗻𝘁𝘀 𝘄𝗶𝘁𝗵 𝗺𝗲𝘁𝗮𝗰𝗵𝗿𝗼𝗺𝗮𝘁𝗶𝗰 𝗹𝗲𝘂𝗸𝗼𝗱𝘆𝘀𝘁𝗿𝗼𝗽𝗵𝘆 (MLD), as a 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆 specifically targeting this 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 will now be available in Spain. S͟u͟m͟m͟a͟r͟y͟ ͟o͟f͟ ͟t͟h͟e͟ ͟d͟r͟u͟g͟s͟ ͟r͟e͟i͟m͟b͟u͟r͟s͟e͟d͟ ͟i͟n͟ ͟t͟h͟i͟s͟ ͟p͟r͟i͟c͟i͟n͟g͟ ͟c͟o͟m͟i͟s͟s͟i͟o͟n͟:͟   • 𝗟𝗶𝗯𝗺𝗲𝗹𝗱𝘆 (atidarsagen autotemcel): Gene therapy for MLD characterized by mutations in the ARSA gene, leading to reduced ARSA enzyme activity:  - In children with late-infantile or early-juvenile forms without clinical manifestations of the disease. - In children with early-juvenile forms showing initial clinical symptoms, who can still walk independently and have not yet experienced cognitive decline.   • 𝗧𝗲𝗽𝗸𝗶𝗻𝗹𝘆 (epcoritamab): Monotherapy for adult patients with relapsed or refractory DLBCL after two or more lines of systemic treatment.   • 𝗧𝗮𝗹𝘇𝗲𝗻𝗻𝗮 (talazoparib): In combination with enzalutamide for adult patients with mCRPC where chemotherapy is not clinically indicated.   • 𝗗𝗲𝘀𝗳𝗹𝘂𝗿𝗮𝗻𝗼 𝗕𝗮𝘅𝘁𝗲𝗿 (desflurane): Inhalation agent for maintaining general anesthesia in hospitalized and outpatient surgeries for adults, adolescents, and intubated children. Its use is restricted to situations where no other anesthetic is medically viable.   • 𝗧𝗲𝘃𝗶𝗺𝗯𝗿𝗮 (tislelizumab): - In combination with pemetrexed and platinum-based chemotherapy for first-line treatment of adult patients with NSCLC expressing PD-L1 in ≥50% of tumor cells, without EGFR or ALK mutations, who have locally advanced NSCLC unsuitable for surgery or platinum-based chemoradiation, or metastatic NSCLC. - In combination with carboplatin and paclitaxel or nab-paclitaxel for first-line treatment of adult patients with squamous NSCLC that is locally advanced or metastatic and unsuitable for surgical resection or platinum-based chemoradiation. - As monotherapy for adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy. Patients with EGFR-mutated or ALK-positive NSCLC must have also received targeted therapies before tislelizumab. - As monotherapy for adult patients with unresectable, locally advanced, or metastatic ESCC after prior platinum-based chemotherapy.   • 𝗩𝘆𝗱𝘂𝗿𝗮 (rimegepant): Acute treatment of migraine, with or without aura, in adults (new indication).   Link: https://lnkd.in/dejK9NjX #Pharmaceuticals #Pricing #MarketAccess #CIPM #Spain

𝗙𝗶𝗿𝘀𝘁 𝗚𝗲𝗻𝗲-𝗘𝗱𝗶𝘁𝗶𝗻𝗴 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗢𝗳𝗳𝗲𝗿𝘀 𝗛𝗼𝗽𝗲 𝗳𝗼𝗿 𝗕𝗹𝗼𝗼𝗱 𝗗𝗶𝘀𝗼𝗿𝗱𝗲𝗿 𝗣𝗮𝘁𝗶𝗲𝗻𝘁𝘀 In a historic breakthrough, the UK's NICE - National Institute for Health and Care Excellence has approved the first gene-editing therapy for treating transfusion-dependent beta thalassemia (TDT) and sickle cell disease, both of which are severe genetic blood disorders. This therapy, marketed as Casgevy, uses CRISPR technology to modify the patient's bone marrow stem cells, enabling the production of functional hemoglobin and offering a potential cure. 𝗛𝗼𝘄 𝗗𝗼𝗲𝘀 𝗖𝗮𝘀𝗴𝗲𝘃𝘆 𝗪𝗼𝗿𝗸? Casgevy involves collecting stem cells from the patient's blood, editing the faulty gene responsible for the disorder using CRISPR, and then reinfusing the edited cells back into the patient. This process aims to restore healthy hemoglobin production, significantly reducing or eliminating the need for regular blood transfusions. Clinical trials have shown promising results, with most patients experiencing substantial improvements. In trials, 28 out of 29 sickle cell patients were free from severe pain crises, and 39 out of 42 beta thalassemia patients no longer required transfusions for at least a year. The list price for a course of Casgevy is approximately £1.65 million, but it will be available to the NHS at a discounted rate through a commercial agreement. While the treatment represents a significant financial investment, its one-time application and potential to dramatically improve patients' quality of life justify the cost for many healthcare systems Following the UK's approval, the U.S. Food and Drug Administration (FDA) has also authorized the use of Casgevy for patients aged 12 and older with these conditions. This dual approval marks a significant step forward in gene therapy and highlights the potential of CRISPR technology to treat and possibly cure genetic diseases (WUSF). Casgevy's approval is a landmark moment in biotechnology, providing new hope for patients with TDT and sickle cell disease. This advancement underscores the importance of continued investment in gene-editing technologies, promising a future where genetic disorders can be effectively treated or even cured.

Sickle cell disease (SCD) is a group of inherited blood disorders characterized by abnormal hemoglobin production, primarily affecting individuals of African descent. It's crucial to understand the types, diagnosis, and treatment options available. Types include Sickle Cell Anemia (HbSS), Sickle Hemoglobin-C Disease (HbSC), Sickle Beta Thalassemia (HbSBeta), and Sickle Cell Trait (HbAS). Diagnosis involves blood tests, newborn screening, and genetic testing. Treatment options range from pain management to gene therapy, with approaches like Hydroxyurea, blood transfusions, and bone marrow transplantation. Gene therapy shows promise in modifying patients' stem cells for normal hemoglobin production. In regions with high SCD prevalence like Nigeria, Unity Health Pharmaceuticals can play a vital role by focusing on affordable Hydroxyurea production, pain management solutions, blood transfusion safety, community health education, and gene therapy research. Collaborative efforts can ensure accessibility to essential treatments and advance healthcare for African patients in need.

𝐃𝐢𝐬𝐫𝐮𝐩𝐭𝐢𝐨𝐧𝐬 𝐢𝐧 𝐭𝐡𝐞 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭 𝐨𝐟 𝐂𝐞𝐥𝐥 𝐚𝐧𝐝 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬   Cell and gene therapies (known as CGTs) hold immense potential for highly effective treatments across various diseases. They offer innovative therapeutic solutions tailored to specific needs. However, the commercial production of CGTs involves multiple steps and rigorous checks, and any challenges or failures during these processes can lead to significant disruptions in their development.   Such disruptions may encompass delayed approval, trial failures or even the discontinuation of entire trials due to public safety concerns and regulatory issues. It’s important to explore the factors contributing to these disruptions in CGT development, shedding light on the complexities and challenges involved in realizing their full potential.   𝐂𝐞𝐥𝐥 𝐚𝐧𝐝 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 Cell and gene therapies represent state-of-the-art approaches in the field of medicine. Cell therapy involves the use of living cells to treat diseases, such as stem cell transplantation for regenerative purposes. Gene therapy, on the other hand, aims to modify or replace faulty genes to treat genetic disorders or introduce therapeutic proteins.   These therapies hold great importance in treating various diseases, due to their potential to address underlying causes at the cellular and genetic level. Cell therapies offer the possibility of tissue repair, organ regeneration and immune modulation, while gene therapies provide opportunities for targeted interventions in genetic diseases. A second category of cell therapies falls under the classification of gene-modified cell therapies. These therapies are primarily focused on oncology applications and utilize gene modification techniques to enable immune cells to effectively target both blood and solid tumor cancers.   These therapies offer personalized treatment options and hold promise for conditions with limited therapeutic alternatives. With ongoing advancements and research, cell and gene therapies continue to pave the way for innovative and potentially transformative treatment approaches in modern medicine. The global #cellandgenetherapy market size was estimated at USD 18.13 billion in 2023 and is projected to hit around USD 97.33 billion by 2033, growing at a CAGR of 18.3% during the forecast period from 2024 to 2033. 𝐓𝐡𝐞 𝐅𝐮𝐥𝐥 𝐒𝐭𝐮𝐝𝐲 𝐢𝐬 𝐑𝐞𝐚𝐝𝐢𝐥𝐲 𝐀𝐯𝐚𝐢𝐥𝐚𝐛𝐥𝐞 | 𝐃𝐨𝐰𝐧𝐥𝐨𝐚𝐝 𝐭𝐡𝐞 𝐒𝐚𝐦𝐩𝐥𝐞 𝐏𝐚𝐠𝐞𝐬 𝐨𝐟 𝐭𝐡𝐢𝐬 𝐑𝐞𝐩𝐨𝐫𝐭@ https://lnkd.in/dwcyhcSw