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        Global biopharma company PTC Therapeutics has received FDA accelerated approval for Kebilidi (eladocagene exuparvovec-tneq), the first gene therapy approved in the US for direct administration to the brain. The therapy is indicated for the treatment of AADC deficiency in children and adults.
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        The approval is based on an ongoing global clinical trial (PTC-AADC-GT-002) where Kebilidi is administered to the putamen of the brain through stereotactic neurosurgery. The gene therapy delivers a functioning DDC gene to restore dopamine production in patients with AADC deficiency.
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        The company received a Rare Disease Priority Review Voucher along with the approval. PTC has already identified treatment centers and trained surgeons for the therapy's launch. The most common adverse reactions include dyskinesia, pyrexia, hypotension, anemia, and salivary hypersecretion. Gene therapy has already been approved in the EU, UK, and Israel.
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    <a href="https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-ptc-therapeutics-gene-therapy-ultra-rare-disorder-2024-11-13/">
      Reuters
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    <a href="https://pharmaphorum.com/news/ptc-gets-fda-okay-first-brain-delivered-gene-therapy">
      pharmaphorum
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Cell & Gene Therapy

INCUMBENTS_PRESENT

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 <p>PTC Therapeutics, a global biopharma company, has obtained FDA accelerated approval for Kebilidi (eladocagene exuparvovec-tneq), which is the inaugural gene therapy in the US that is approved for direct administration to the brain. This treatment is prescribed for the treatment of AADC deficiency in children and adults.</p>
 <p>This approval hinges upon an ongoing global clinical trial (PTC-AADC-GT-002) in which Kebilidi is given to the putamen of the brain through stereotactic neurosurgery. This gene therapy works by delivering a functional DDC gene to revitalize dopamine production in patients who have AADC deficiency.</p>
 <p>In tandem with the approval, the company has received a Rare Disease Priority Review Voucher. PTC has already pinpointed treatment centers and trained surgeons in preparation for the launch of the therapy. Side effects that are commonly seen include dyskinesia, pyrexia, hypotension, anemia, and salivary hypersecretion. The gene therapy has also been approved in the EU, UK, and Israel.</p>
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PTC Therapeutics receives FDA approval for brain-delivered gene therapy Kebilidi

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 <p>The FDA has given approval to Autolus Therapeutics for its CAR T therapy, Aucatzyl (obecabtagene autoleucel), designed to treat adult patients suffering from B-cell precursor acute lymphoblastic leukemia that has relapsed or proven refractory. This approval is based on the results of the FELIX clinical trial, where 63% of evaluated patients experienced total remission, with 42% achieving this within a three-month period.</p>
 <p>Aucatzyl sets a precedent as the first CAR T therapy approved sans a required Risk Evaluation Mitigation Strategy (REMS) program. The therapy demonstrated minimal cytokine release syndrome levels with a 3% Grade 3 event rate and zero Grade 4 or 5 events. Production for the treatment will occur at the Stevenage, UK facility of Autolus, with Cardinal Health overseeing all US distribution.</p> 
 <p>With a cost of USD 525,000 for each treatment, Aucatzyl will be in direct competition with Gilead's Tecartus, priced at USD 460,000, and Novartis' Kymriah, priced at USD 580,000. The initial plan for Autolus is to begin with 30 treatment centers, tailored to cover 60% of the target demographic, before expanding to 60 centers catering to 90% of patient population within a 12-month timeframe.</p>
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Autolus receives FDA approval for B-ALL therapy Aucatzyl

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 <p>Ocugen, a cell and gene therapy developer, has obtained a credit facility of USD 30 million from Avenue Venture Opportunities Fund, L.P., an Avenue Capital Group fund. This facility has a term of four years.</p>
 <p>The procured funding is scheduled for various usages like general corporate purposes, capital expenditures, working capital, and general and administrative expenses. Additionally, it will provide support for the clinical development of three modifier gene therapies and complete the OCU400 Phase III liMeliGhT clinical trial.</p>
 <p>Ocugen's financial runway, with the help of this new funding and its existing cash reserves, is expected to be extended till the first quarter of 2026.</p>
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Ocugen raises USD 30 million in debt funding from Avenue Capital Group

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 <p>Lyell Immunopharma, a clinical-stage biotechnology company, is set to purchase ImmPACT Bio for USD 30 million in cash and 37.5 million shares of Lyell's common stock. Additional payments based on clinical achievements, which could include up to 12.5 million more shares, have also been outlined in the deal.</p>
 <p>The primary objective of this acquisition is to boost Lyell's current lineup of clinical-stage chimeric antigen receptor (CAR) T-cell therapies. The company has plans to release preliminary findings from IMPT-314's ongoing Phase I–II trial later in the year, with sights on launching a deciding trial in 2025.</p>
 <p>ImmPACT Bio is also a clinical-stage biotech company, with its foremost program, IMPT-314, leading the way. This is a CAR T-cell product candidate that targets CD19/20, and it has showcased potential in treating hematologic malignancies, such as large B-cell lymphoma.</p>
 
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Lyell Immunopharma to acquire ImmPACT Bio for USD 30 million to expand pipeline

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 <p>March Biosciences, a clinical-stage biotechnology company, has garnered USD 28.4 million in a Series A funding round. The round was led by Mission BioCapital and 4BIO Capital, and saw participation from KdT Ventures, Alexandria Venture Investments, and other existing investors. To date, the company's total funds raised amount to over USD 51 million.</p>
 <p>These newfound funds are set to further the Phase II clinical development of MB-105, the company's leading asset. This asset is geared towards the treatment of T-cell lymphoma, with trials projected to launch at the beginning of 2025. In addition, the funds will also aid the continuous advancement of undisclosed products within the company's pipeline.</p>
 <p>The company specializes in the development of chimeric antigen receptor T-cell (CAR-T) therapies, aimed at tackling challenging cancer types. March Biosciences was initially launched as a spinout, stemming from collaborations between the Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital, and Texas Children's Hospital.</p>
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March Biosciences raises USD 28.4 million in Series A funding to advance clinical trials

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 <p>Cell therapy firm AvenCell has managed to secure USD 112 million in a Series B funding round. The round saw participation from lead investor Novo Holdings, F-Prime Capital, Eight Roads Ventures Japan, Piper Heartland Healthcare Capital, NYBC Ventures, and Blackstone Life Sciences.</p>
 <p>The money raised will be channelized to speed up the clinical validity assessment of AvenCell's unique universal switchable CAR-T cell therapy, devised for treating hematologic malignancies and auto-immune diseases. Another important agenda for the fund utilization will be to ensure support for the continuing clinical trials linked to AVC-101 and AVC-201, meant for relapsed/refractory acute myeloid leukemia (AML) treatment.</p>
 <p>The clinical-stage cell therapy company, AvenCell Therapeutics, is renowned for curating switchable CAR-T cell therapies for cancers, that are generally hard to treat. Their unique platform enables precise management of CAR-T cell activity through a two-component system. It includes engineered T cells with a universal receptor that can be activated only when they're bound to a separately administered targeting module. This particular approach allows the CAR-T cells to be controlled in an "on" and "off" mode, post their infusion into the patients, endowing a flexibility to manage potential adverse events efficiently. </p>
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AvenCell raises USD 112 million in Series B funding to accelerate clinical validation

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 <p>Biopharmaceutical firm Tolerance Bio has gathered USD 17.2 million through a seed funding round. The round was led by Columbus Venture Partners and saw participation from Criteria Bio Ventures, Sessa Capital, BioAdvance, Ben Franklin Technology Partners, and several individual investors.</p>
 <p>The raised funding will be utilized to develop an allogeneic thymus induced pluripotent stem cell (iPSC)-based cell therapy platform as well as pharmacological thymus treatments for diseases that are immune-mediated.</p>
 <p>The technologies of Tolerance Bio aim to prevent and delay thymic involution and in case of loss, restore thymic function. The aim is to potentially address immune diseases and enhance longevity. The company's iPSC technologies were originally developed at the University of Colorado and the University of Florida.</p>
 
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Tolerance Bio raises USD 17.2 million in seed funding to advance thymus-based therapies

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 <p>Immatics, an oncology-focused biopharmaceutical company, announced an underwritten public offering of 16.25 million shares at USD 9.25 each and an additional purchase option for 2,437,500 shares. Expected to gross around USD 150 million, the offering is scheduled to close on October 15, 2024, if closing conditions are met.</p>
 <p>The company did not specify an intended use for the raised capital.</p>
 <p>Immatics, a clinical-stage biopharmaceutical company, specializes in the production of T-cell redirecting cancer immunotherapies. It achieves this by pairing the discovery of genuine cancer targets with the development of the appropriate T-cell receptors, triggering a specific T-cell response against these targets. Among their proprietary technology platforms are XPRESIDENT for target discovery and XCEPTOR for T-cell receptor engineering. Immatics' pipeline includes two main therapeutic modalities: Adoptive Cell Therapies (ACT) and T-cell engaging receptor (TCER) bispecifics.</p>
 
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Immatics raises USD 150 million in public funding

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 <p>Purespring Therapeutics, a company specialising in gene therapy, has successfully raised GBP 80 million in a Series B financing round. This was led by Sofinnova Partners and included other participants like Gilde Healthcare, Forbion, British Patient Capital, and Syncona Limited, which was an original investor.</p>
 <p>The funds raised are to be used to support Purespring's pipeline of projects, as well as to initiate a Phase I/II clinical trial for IgA Nephropathy (IgAN), a commonly occurring chronic kidney disease.</p>
 <p>The company focuses on the development of adeno-associated virus (AAV) gene therapies that target a specialised kidney cell, the podocyte. This has a role in about 60% of renal diseases. Purespring's unique platform allows for efficient and specific delivery of functioning gene copies to podocytes. This provides a new method for modifying treatments of various kidney diseases. The company’s primary program, PS-002, aims at IgA Nephropathy (IgAN) and other kidney diseases that are complement-mediated.</p>
 
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Purespring Therapeutics raises GBP 80 million in Series B funding to develop​​ kidney disease pipeline

PROMINENT_CATEGORY

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 <p>Astellas Pharma, a global pharmaceutical company, has made a licensing agreement with gene therapy firm AviadoBio. This agreement gives Astellas exclusive rights to AviadoBio's AVB-101, an AAV-based gene therapy under Phase 1/2 development. Astellas is going to invest USD 20 million in equity and an upfront payment of up to USD 30 million for the licensing option to AVB-101. If Astellas decides to exercise this option, AviadoBio could earn USD 2.18 billion from license fees and milestone payments along with royalties.</p>
 <p>AVB-101 is intended to treat frontotemporal dementia with progranulin mutations (FTD-GRN), a dementia often misdiagnosed or undiagnosed. This alliance plays an important role in speeding up the development and global commercialization of a potential therapy for patients living with FTD-GRN and related neurological disorders.</p>
 
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All Updates

PTC Therapeutics receives FDA approval for brain-delivered gene therapy Kebilidi

Autolus receives FDA approval for B-ALL therapy Aucatzyl

Ocugen raises USD 30 million in debt funding from Avenue Capital Group

Lyell Immunopharma to acquire ImmPACT Bio for USD 30 million to expand pipeline

March Biosciences raises USD 28.4 million in Series A funding to advance clinical trials

AvenCell raises USD 112 million in Series B funding to accelerate clinical validation

Tolerance Bio raises USD 17.2 million in seed funding to advance thymus-based therapies

Immatics raises USD 150 million in public funding

Purespring Therapeutics raises GBP 80 million in Series B funding to develop kidney disease pipeline

AviadoBio partners with Astellas Pharma to develop gene therapy targeting dementia patients

Cell & Gene Therapy

PTC Therapeutics receives FDA approval for brain-delivered gene therapy Kebilidi

Contact us

All Updates

PTC Therapeutics receives FDA approval for brain-delivered gene therapy Kebilidi

Autolus receives FDA approval for B-ALL therapy Aucatzyl

Ocugen raises USD 30 million in debt funding from Avenue Capital Group

Lyell Immunopharma to acquire ImmPACT Bio for USD 30 million to expand pipeline

March Biosciences raises USD 28.4 million in Series A funding to advance clinical trials

AvenCell raises USD 112 million in Series B funding to accelerate clinical validation

Tolerance Bio raises USD 17.2 million in seed funding to advance thymus-based therapies

Immatics raises USD 150 million in public funding

Purespring Therapeutics raises GBP 80 million in Series B funding to develop​​ kidney disease pipeline

AviadoBio partners with Astellas Pharma to develop gene therapy targeting dementia patients

Cell & Gene Therapy

PTC Therapeutics receives FDA approval for brain-delivered gene therapy Kebilidi

Contact us

Purespring Therapeutics raises GBP 80 million in Series B funding to develop kidney disease pipeline